Search Results
COG ARST2032: A Prospective Phase 3 Study of Patients with Newly Diagnosed Very Low-risk and Low-risk Fusion Negative Rhabdomyosarcoma
Rhabdomyosarcoma is a type of cancer that occurs in the soft tissues in the body. This phase III trial aims to maintain excellent outcomes in patients with very low risk rhabdomyosarcoma (VLR-RMS) while decreasing the burden of therapy using treatment with 24 weeks of vincristine and dactinomycin (VA) and examines the use of centralized molecular risk stratification in the treatment of rhabdomyosarcoma. Another aim of the study it to find out how well patients with low risk rhabdomyosarcoma (LR-RMS) respond to standard chemotherapy when patients with VLR-RMS and patients who have rhabdomyosarcoma with DNA mutations get separate treatment. Finally, this study examines the effect of therapy intensification in patients who have RMS cancer with DNA mutations to see if their outcomes can be improved.
• 21 or younger at time of enrollment
• newly diagnosed embryonal rhabdomyosarcoma (ERMS), spindle cell/sclerosing RMS, or FOXO1 fusion negative alveolar rhabdomyosarcoma
• must be enrolled in APEC14B1 (NCT02402244) prior to enrollment and treatment on ARST2032 (this trial)
• contact study team for more detailed criteria
• received prior chemotherapy and/or radiation therapy for cancer prior to enrollment
• unable to undergo radiation therapy
• Females who are pregnant
A Phase 3 Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of BBP-418 (ribitol) in Patients with Limb Girdle Muscular Dystrophy 21 (LGMD21) (Fortify)
This study will use BBP-418 study drug in patients with LGMD to assess the clinical biomarkers, efficacy and safety of BBP-418 during the 36 months treatment phase.
• 12 to 60 years of age
• genetically confirmed diagnosis of limb girdle muscular dystrophy
• have clinical symptoms of weakness
• weight at least 30 kg (66 lbs.)
• willing to use a highly effective method of birth control until 12 weeks after last dose of study medication
• any significant medical or mental health diagnosis including abnormal lab values (study staff will review)
• surgery for scoliosis or other indication planned during the time of the study
• use of ribose or other sugar alcohol-containing supplement within 90 days of staring the study
• use of a systemic corticosteroid for the treatment of muscular dystrophy within 90 days of starting the study
A Phase 1/2 Study of the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Relatlimab Plus Nivolumab in Pediatric and Young Adult Participants with Recurrent or Refractory Classical Hodgkin Lymphoma and Non-Hodgkin Lymphoma Protocol Number: CA224069 (RELATIVITY-069)
CA224069 is an open-label, Phase 1/2 clinical trial of relatlimab + nivolumab in children, adolescents and young adults with Recurrent or Refractory Classical Hodgkin Lymphoma (R/R cHL) and Non-Hodgkin Lymphoma (NHL). Part A will encompass safety and dose determination of relatlimab + nivolumab. Part B will be composed of an expansion cohort of cHL (Cohort 1) and an exploratory assessment in NHL (Cohort 2).
• up to 30 years old
• pathologically confirmed high-risk recurrent/relapsed or refractory (R/R) classical Hodgkin lymphoma (cHL), after non-response to or failure of first-line standard therapy prior to a definitive therapy e.g.high-dose chemotherapy/autologous stem cell transplant (HDCT/ASCT)
• participants with pathologically confirmed R/R NHL after failure or non-response to second line therapy, including but not limited to primary mediastinal B-cell lymphoma, diffuse large B-cell lymphoma (DLBCL), mediastinal gray zone lymphoma (MGZL), anaplastic large cell lymphoma (ALCL), or peripheral T-cell lymphoma (PTCL)
• aggressive B-cell lymphomas subtypes including Burkitt lymphoma (BL), lymphoblastic lymphoma, and NK/T-cell lymphoma/leukemia
• prior autologous stem cell transplantation (HDCT/ASCT)
• see link to clinicaltrials.gov for additional exclusion criteria
COG AALL1621 - A Phase 2 Study of Inotuzumab Ozogamicin (NSC# 772518, IND#133494) in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)
This phase II trial studies how well inotuzumab ozogamicin works in treating younger patients (≥1 year and < 22 years ) with CD22 positive B acute lymphoblastic leukemia that has come back or does not respond to treatment. Immunotoxins, such as inotuzumab ozogamicin, are antibodies linked to a toxic substance and may help find cancer cells that express CD22 and kill them without harming normal cells.
• 1 to 21 years old
• must have B Acute Lymphoblastic Leukemia (B-ALL), or previously diagnosed B lymphoblastic lymphoma (B-LL)
• Patients with one of the following: Second or greater relapse; Primary refractory disease with at least 2 prior induction attempts; First relapse refractory to at least one prior re-induction attempt; OR Any relapse after HSCT (Cohort 1 ONLY)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• currently receiving another investigational drug
• currently receiving or plan to receive other anti-cancer agents (except hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy, and intrathecal chemotherapy)
MT2022-27: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Primary Purpose: To identify risk factors and mechanisms of lung injury, test novel diagnostic strategies and treatments to reduce morbidity and mortality from lung injury after transplant.
• up to 24 years old
• undergoing allogeneic or autologous HSCT
Cochlear Implantation in Children with Asymmetric Hearing Loss or Single-Sided Deafness Clinical Trial
• Parents and child fluent in English
• Parents desire functional hearing in both ears for their child
• Severe to profound sensorineural hearing loss in one ear and normal hearing in the other ear
• If older than 5 years, documentation of progressive hearing loss (i.e. passed newborn hearing screening, or significant change in hearing)
• Medical condition that contraindicates a cochlear implant, including abnormal hearing nerve
• Already using a cochlear implant
• Inability to complete study procedures
• Unrealistic expectations related to the benefits and limitations of cochlear implantation
• Unwillingness or inability to comply with all investigational requirement
MT2013-06C : Treatment of graft Failure after HSCT
COG ACNS1931 - A Phase 3 Study of Selumetinib (NSC# 748727, IND# 77782) or Selumetinib in Combination with Vinblastine for non-NF1, non-TSC Patients with Recurrent or Progressive Low-Grade Gliomas (LGGs) Lacking BRAFV600E or IDH1 Mutations
This phase III trial investigates the best dose of vinblastine in combination with selumetinib and the benefit of adding vinblastine to selumetinib compared to selumetinib alone in treating children and young adults with low-grade glioma (a common type of brain cancer) that has come back after prior treatment (recurrent) or does not respond to therapy (progressive). Selumetinib is a drug that works by blocking a protein that lets tumor cells grow without stopping. Vinblastine blocks cell growth by stopping cell division and may kill cancer cells. Giving selumetinib in combination with vinblastine may work better than selumetinib alone in treating recurrent or progressive low-grade glioma.
• ages 2 to 21 at time of starting the study
• diagnosis of low-grade glioma or low-grade astrocytoma brain tumor
• tumor size increased or returned after treatment with at least one cancer therapy
• high blood pressure (hypertension) must be under control
• must be able to swallow whole capsules
• contact study staff for additional criteria
• treatment for another tumor in the past year
• any serious medical or mental health diagnosis, including substance use disorders or ophthalmological conditions (study staff will review)
• women who are pregnant or breast feeding
A Double-Blind, Placebo-Controlled, Dose Escalation Study to Assess the Efficacy, Safety and Pharmacokinetics of Voclosporin in Adolescents with Lupus Nephritis (VOCAL)
The aim of this study is to investigate whether voclosporin, added to standard treatment, is able to reduce activity of lupus nephritis over a study treatment period of 24 weeks, and to determine its safety as well as the best dose for treatment of lupus nephritis in children or adolescents.
• 12 to 17 years old
• diagnosis of systemic lupus erythematosus (SLE)
• active lupus nephritis confirmed by a kidney biopsy
• currently need dialysis
• clinically significant active medical or mental health conditions (study staff will review)
• certain medications, including: immunosuppression biologic agents, cyclophosphamide, calcineurin inhibitors (CNIs), start or change dose of ACE inhibitors/ARBs within 4 weeks prior to starting study, IV corticosteroids and IV immunoglobulin within 2 weeks of starting study
COG AALL1732: A Phase 3 Randomized Trial of Inotuzumab Ozogamicin (IND#:133494, NSC#: 772518) for Newly Diagnosed High-Risk B-ALL; Risk-Adapted Post-Induction Therapy for High-Risk B-ALL, Mixed Phenotype Acute Leukemia, and Disseminated B-LLy
• patients must be > 365 days and < 25 years of age
• participant has newly diagnosed B-ALL or MPAL with ?25% blasts on a bone marrow (BM) aspirate or newly diagnosed B-LLy
• see link to clinicaltrials.gov for complete inclusion criteria
• patients with Down syndrome are not eligible
• patients with acute undifferentiated leukemia (AUL) are not eligible
• female patients who are pregnant, since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required for female patients of childbearing potential
• lactating women who plan to breastfeed their infants while on study and for 2 months after the last dose of inotuzumab ozogamicin.
• see link to clinicaltrials.gov for complete exclusion criteria
MT-2018-20: COG AALL1631 - International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones
This randomized phase III trial studies how well imatinib mesylate and combination chemotherapy work in treating patients (> 1 year and < 21 years) with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia. Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imatinib mesylate and combination chemotherapy may work better in treating patients with Philadelphia chromosome positive acute lymphoblastic leukemia.
• >= 1 year (365 days) and =< 21 years at ALL diagnosis
• Diagnosis: Ph+ (BCR-ABL1 fusion): newly diagnosed de novo ALL (B-ALL or T-ALL) or mixed phenotypic acute leukemia (MPAL meeting 2016 WHO definition) with definitive evidence of BCR-ABL1 fusion by karyotype, FISH and/or molecular methodologies. OR
• Diagnosis: ABL-class fusion: newly diagnosed B-ALL with definitive evidence of ABLclass fusions. ABL-class fusions are defined as those involving the following genes: ABL1, ABL2, CSF1R, PDGFRB, PDGFRA.
• see link to clinicaltrials.gov for complete Inclusion Criteria
• known history of chronic myelogenous leukemia (CML)
• ALL developing after a previous cancer treated with cytotoxic chemotherapy
• Down syndrome
• patients with congenital long QT syndrome, history of ventricular arrhythmias or heart block
2020IS043; MT2020-06; A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF JSP191 FOR HEMATOPOIETIC CELL TRANSPLANTATION CONDITIONING TO ACHIEVE ENGRAFTMENT AND IMMUNE RECONSTITUTION IN SUBJECTS WITH SCID
This study is looking at whether giving a new type of experimental medicine, called JSP191, can prepare the body to help the stem cell transplant work better, so the immune system can grow and fight infections. The study doctor and Sponsor also want to see how safe and well tolerated this experimental medicine is. They will study whether it is safe to give to patients and look at how much medication to give and what side effects may occur. During this study, the optimal dose of JSP191 will be determined and additional patients will be enrolled in this study using that dose level.
• at least 3 months old
• diagnosis of typical Severe Combined Immunodeficiency (SCID)
• patient with human leukocyte antigen (HLA) matched related or unrelated donors
• acute or uncontrolled infections
• patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• patients with active malignancies
• active Graft-versus-host disease (GVHD) within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD
COG AREN1921 - Treatment of Newly Diagnosed Diffuse Anaplastic Wilms Tumors (DAWT) and Relapsed Favorable Histology Wilms Tumors (FHWT)
This phase II trial studies how well combination chemotherapy works in treating patients (≤ 30 years old) with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) or favorable histology Wilms tumors (FHWT) that have come back (relapsed).This trial may help doctors find out what effects, good and/or bad, regimen UH-3 (vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan) has on patients with newly diagnosed DAWT and standard risk relapsed FHWT (those treated with only 2 drugs for the initial WT)and regimen ICE/Cyclo/Topo (ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan) has on patients with high and very high risk relapsed FHWT (those treated with 3 or more drugs for the initial WT).
COG AGCT1532 - A Randomized Phase 3 Trial of Accelerated versus Standard BEP Chemotherapy for Patients with Intermediate and Poor-risk Metastatic Germ Cell Tumors (P3BEP)
This trial is an open label, randomized, stratified 2-arm Australian-led multicenter phase 3 clinical trial undertaken in two stages. Participants (age >= 11 years and <= 45 years) with intermediate and poor-risk metastatic germ cell tumors will be randomized into either a “standard BEP” group or “accelerated BEP” group. Participants will be assigned to the two treatment arms in a 1:1 ratio and evaluated weekly, and then for 5 years after completing the study to assess the long-term effects of the chemotherapy. Bleomycin, Etoposide, Cisplatin (BEP) administered 3-weekly x 4 remains standard 1st line chemotherapy for intermediate- and poor-risk metastatic germ cell tumours (GCTs). BEP is accelerated by cycling Cisplatin and etoposide 2-weekly instead of 3-weekly. The aim of this study is to determine if accelerated BEP is superior to standard BEP as first-line chemotherapy for intermediate and poor risk metastatic GCTs.
• between 11 years and 45 years old
• confirmed germ cell tumour (non-seminoma or seminoma) OR Exceptionally raised tumour markers (AFP equal or greater than 1000ng/mL and/or HCG equal or greater than 5000 IU/L)
• primary arising in testis, ovary, retro-peritoneum, or mediastinum
• metastatic disease or non-testicular primary
• see link to clinicaltrials.gov for completed Inclusion/Exclusion criteria
• other primary malignancy (EXCEPT adequately treated non-melanomatous carcinoma of the skin, germ cell tumour, or other malignancy treated at least 5 years previously with no evidence of recurrence)
• significant cardiac disease resulting in inability to tolerate IV fluid hydration for cisplatin
• peripheral neuropathy equal or greater than grade 2 or clinically significant sensorineural hearing loss or tinnitus
A US Multi-center, Prospective, Non-interventional, Long-term, Effectiveness and Safety Study of Patients Treated with SKYTROFA (lonapegsomatropin) (SkybriGHt) (SkybriGHt)
Skytrofa is approved in the U.S. for sale and use in children with growth hormone deficiency (GHD). This study is being done to find out how safe and useful Skytrofa is for long-term treatment. A child’s care will follow the normal treatment practices at the clinic. There is no new treatment or medicine involved and no additional visits will be performed.
• 1 to 18 years old
• on treatment with SKYTROFA (lonapegsomatropin)
• participating in any interventional clinical study
MT2015-25: Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants with Type 2 Gaucher Disease
PR001A is designed to deliver a normal GBA1 gene copy into the body to increase the activity of GCase, which is low in Type 2 Gaucher Disease (GD2) patients. The new GBA1 gene will remain a child’s body cells for many years and possibly for the rest of their life. A participant will need one surgery during which the study drug will be given and will stay in the hospital for at least 48 hours following the surgery.
• 0 to 24 months of age
• clinical diagnosis on Gaucher disease, Type 2 (GD2)
• Bi-allelic GBA1 mutation
• child has a reliable caregiver (i.e., parent/legal guardian) who is willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities
• diagnosis of a significant CNS disease other than GD2
• able to walk independently
• any other significant medical diagnosis (study staff will review)
• significant laboratory test result abnormalities
• unable to tolerate diagnostic imaging (MRI, CT scan) or unable to tolerate contrast agent
• unable to have sedation or anesthesia
MT2016-11 :Autologous Stem Cell Transplant In Patients with Hodgkin Lymphoma (HL) and Non-Hodgkin Lymphomas (NHL)
The treatment (chemotherapy and transplant procedures) is considered standard clinical care that are usually given to the patients with this disease. The research aspect of this study is to collect data on the patients who are being treated on this plan. Patients will be followed throughout the course of their clinical care and for three years after their transplant.
• up to 75 years of age
• diagnosis of Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Mature B cell Lymphoma, or Mature T cell Lymphoma
• at least 4 weeks from previous chemotherapy; 6 weeks from nitrosoureas
• women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment
• see link to clinicaltrials.gov for complete criteria
• women who are pregnant or breastfeeding
• chemotherapy resistant disease
• unrelated active infection
COG ARST2031: A Randomized Phase 3 Trial of Vinorelbine, Dactinomycin, and Cyclophosphamide (VINO-AC) Plus Maintenance Chemotherapy with Vinorelbine and Oral Cyclophosphamide (VINO-CPO) vs Vincristine, Dactinomycin and Cyclophosphamide (VAC) plus VINO-CPO Maintenance in Patients with High Risk Rhabdomyosarcoma (HR-RMS)
his phase III trial compares the safety and effect of adding vinorelbine to vincristine, dactinomycin, and cyclophosphamide (VAC) for the treatment of patients with high risk rhabdomyosarcoma (RMS). High risk refers to cancer that is likely to recur (come back) after treatment or spread to other parts of the body. This study will also examine if adding maintenance therapy after VAC therapy, with or without vinorelbine, will help get rid of the cancer and/or lower the chance that the cancer comes back. Vinorelbine and vincristine are in a class of medications called vinca alkaloids. Dactinomycin is a type of antibiotic that is only used in cancer chemotherapy. Cyclophosphamide is in a class of medications called alkylating agents. Vinorelbine, vincristine, dactinomycin and cyclophosphamide are chemotherapy medications that work by slowing or stopping the growth of cancer cells in the body. This trial may have the potential to eliminate rhabdomyosarcoma for a long time or for the rest of patient's life.
ANHL2121: Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory). Langerhans cell histiocytosis is a type of disease that occurs when the body makes too many immature Langerhans cells (a type of white blood cell). When these cells build up, they can form tumors in certain tissues and organs including bones, skin, lungs and pituitary gland and can damage them. This tumor is more common in children and young adults. DAY101 may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Using DAY101 may be effective in treating patients with relapsed or refractory Langerhans cell histiocytosis.
• 180 days to < 22 years (at time of study enrollment)
• patients with multifocal progressive, relapsed, or recurrent LCH with measurable disease at study entry
• participant must be able to take an enteral dose and formulation of medication. Study medication is only available as an oral suspension or tablet, which may be taken by mouth or other enteral route such as nasogastric, jejunostomy, or gastric tube
• see link to clinicaltrials.gov for complete inclusion criteria
• LCH arising along with other hematologic malignancy (e.g. mixed LCH with acute lymphoblastic leukemia) or any history of non-histiocytic malignancy
• history of solid organ or hematopoietic bone marrow transplantation
• female patients who are pregnant are ineligible. A pregnancy test is required for female patients of childbearing potential
• lactating females who plan to breastfeed their infants are ineligible
• see link to clinicaltrials.gov for complete exclusion criteria
AHOD2131: A Randomized Phase 3 Interim Response Adapted Trial Comparing Standard Therapy with Immuno-oncology Therapy for Children and Adults with Newly Diagnosed Stage I and II Classic Hodgkin Lymphoma
COG AALL1731 - A Phase 3 Trial Investigating Blinatumomab (IND# 117467, NSC# 765986) in Combination with Chemotherapy in Patients with Newly Diagnosed Standard Risk or Down syndrome B-Lymphoblastic Leukemia (B-ALL) and the Treatment of Patients with Localized B-Lymphoblastic Lymphoma (B-LLy)
This phase III trial studies how well blinatumomab works in combination with chemotherapy in treating patients (365 Days to 31 Years) with newly diagnosed, standard risk B-lymphoblastic leukemia or B-lymphoblastic lymphoma with or without Down syndrome. Monoclonal antibodies, such as blinatumomab, may induce changes in body's immune system and may interfere with the ability of cancer cells to grow and spread. Drugs used in chemotherapy, such as vincristine, dexamethasone, prednisone, prednisolone, pegaspargase, methotrexate, cytarabine, mercaptopurine, doxorubicin, cyclophosphamide, and thioguanine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Leucovorin decreases the toxic effects of methotrexate. Giving monoclonal antibody therapy with chemotherapy may kill more cancer cells. Giving blinatumomab and combination chemotherapy may work better then combination chemotherapy alone in treating patients with B-ALL. This trial also assigns patients into different chemotherapy treatment regimens based on risk (the chance of cancer returning after treatment). Treating patients with chemotherapy based on risk may help doctors decide which patients can best benefit from which chemotherapy treatment regimens.
• Age: patients must be > 365 days and < 10 years of age (B-ALL patients without Down Syndrome-DS) OR no more than 31 years of age (B-ALL patients with DS) OR no more than 31 years of age (B-LLy patients with or without DS)
• Diagnosis: Patient has newly diagnosed B-cell ALL, with or without Down syndrome: > 25% blasts on a BM aspirate; OR if a BM aspirate is not obtained or is not diagnostic of B-ALL, the diagnosis can be established by a pathologic diagnosis of B-ALL on a BM biopsy OR a complete blood count (CBC) documenting the presence of at least 1,000 circulating leukemic cells;
• OR Patient has newly diagnosed B-cell LLy Murphy Stages I or II, with or without Down syndrome
• White Blood Cell Count (WBC) Criteria: B-ALL patients without DS must have an initial white blood cell count < 50,000
• B-ALL patients with DS are eligible regardless of the presenting WBC
• Patient must not have secondary ALL that developed after treatment of a prior malignancy with cytotoxic chemotherapy
• Patients must not have received any prior cytotoxic chemotherapy for either the current diagnosis of B-ALL or B-LLy or for any cancer diagnosed prior to initiation of protocol therapy on AALL1731
• Patients requiring radiation at diagnosis
• Female patients who are pregnant or breastfeeding their infants
MT2022-44 Analysis of Autoinflammation in Chronic Granulomatous Disease Patients Undergoing Hematopoietic Cell Transplantation or Gene Therapy (PIDTC 6908)
CGD disorders are a group of diseases that cause the immune system to function abnormally, leading to infections, autoimmunity and/or inflammation that can begin early in life. CGD is usually caused by changes in certain genes in DNA. Researchers are trying to learn what types of medical problems patients with CGD have and how these respond to treatment, including bone marrow transplantation and gene therapy. Researchers also want to learn which genes cause CGD and how specific gene abnormalities lead to medical problems.
• confirmed diagnosis of Chronic Granulomatous Disease (CGD)
• to be scheduled to undergo HCT or GT within one year of enrollment
• additional inclusion criteria (study staff will review)
• .presence of other primary immunodeficiency syndromes that do not meet the clinical and laboratory criteria for CGD
• HIV infection
• past or current medical problems or findings from physical examination or laboratory testing that are not listed above which, in the opinion of the investigator, may pose additional risks from participation
An Open-label, Single Arm, Multicenter, Phase III Study on the Efficacy, Safety,and Pharmacokinetics of FP-001 42 mg Controlled Release in Patients with Central (Gonadotropin-Dependent) Precocious Puberty
The study drug FP-001 (Leuprolide mesylate) is being developed for children that are suffering from central (gonadotropin-dependent) precocious puberty (CPP). Leuprolide has been approved in the United States (US) and the European Union (EU) as treatment for prostate cancer already, and other forms of Leuprolide from other companies have been approved for the treatment of CPP. In this clinical study, Leuprolide will be used in the form of a 6-month depot injection.
• females aged 2 to 8 years or males aged 2 to 9 years old
• diagnosis of Central Precocious Puberty (CPP) within 12 months
• additional inclusion criteria (study staff will review)
• major medical or psychiatric illness that could interfere with study visits
• history of seizures, epilepsy, and/or central nervous system disorders that may be associated with seizures or convulsions
• specific prior treatments (study staff will review)
AAML18P1: Stopping Tyrosine Kinase Inhibitors (TKI) to Assess Treatment-Free Remission (TFR) in Pediatric Chronic Myeloid Leukemia - Chronic Phase (CML-CP)
This phase II trial studies how stopping tyrosine kinase inhibitors will affect treatment-free remission in patients with chronic myeloid leukemia in chronic phase. When the level of disease is very low, it's called molecular remission. TKIs are a type of medication that help keep this level low. However, after being in molecular remission for a specific amount of time, it may not be necessary to take tyrosine kinase inhibitors. It is not yet known whether stopping tyrosine kinase inhibitors will help patients with chronic myeloid leukemia in chronic phase continue or re-achieve molecular remission.
• < 25 years old
• diagnosis of CML-CP before age 18
• patient must be in molecular remission (MR) for ? 2 consecutive years at the time of enrollment
• patient must have received any TKI for a minimum of 3 consecutive years and agree to stop using TKI therapy
• see link to clinicaltrials.gov for complete criteria
• known T3151 mutation
• history of accelerated phase or blast crisis CML
• women who are pregnant
• if breast feeding, must agree to stop
Neurofeedback and Neural Plasticity of Self-Processing and Affect Regulation Circuits in Suicide Attempting Adolescents
The purpose of this study is to examine a new, experimental treatment for adolescents at risk for suicide attempts called neurofeedback training. In neurofeedback training, you are trying to control your brain function on purpose. In this study, your child will see their brain activity (displayed like a thermometer). He/she will recall positive memories to try to change the levels of their brain activity shown on the visual thermometer inside a scanner.
• any gender identity
• 11-17 years old
• past suicide attempt and/or current suicide ideation
• Autism Spectrum Disorder
• Cognitive Developmental Delay (IQ < 75 i.e.intellectual disability)
• diagnosis of Schizophrenia
A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in idiopathic immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN). (APPARENT)
To evaluate the efficacy and safety of iptacopan compared to placebo in patients with idiopathic immune complex mediated membranoproliferative glomerulonephritis (IC-MPGN)
• 12 to 60 years old
• diagnosis of idiopathic IC-MPGN as confirmed by kidney biopsy within 12 months prior to starting the study for adults and within 3 years for adolescents
• must have a vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection prior to the start of study treatment
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• have had cell or a solid organ transplantation, including kidney transplantation
• Body mass index (BMI) >38 kg/m2
• Body weight <35 kg (77 pounds)
• additional criteria that study staff will review
COG ALTE1631 - A Randomized Web-based Physical Activity Intervention among Children and Adolescents with Cancer
Regular participation in physical activity helps maintain a healthy weight, improves energy levels and overall health. Children and teenagers who have received treatment for cancer are often less active, may gain weight and have more health problems as compared to children and teenagers who have not received treatment for cancer. This study looks at physical activity and its effect on your health. This study will use a variety of interventions to see if they affect how active you are over time.
• between 8 and 16 years old
• diagnosed with cancer that is now in remission (ICD-0 histologic behavior code of 2 - carcinoma in situ or 3 - malignant only)
• completed treatment within the past 12 months
• able to walk and do self care with some assistance
• report less than 420 minutes of moderate to vigorous physical activity over the last week
• child and at least one parent/guardian are able to read and write English, Spanish, or French
• eligibility will be assessed at first study visit
• contact study team with any questions
• previous hematopoietic Stem Cell Transplant (HSCT)
• significant additional medical, mental health, or social issue that would compromise safety, participating in the study or follow-up
• pregnant or unwilling to use effective birth control for the time of the study (abstinence included)
• cognitive, motor, visual, or auditory problem that prevents use of a computer
COG AGCT1531 - A Phase 3 Study of Active Surveillance for Low Risk and a Randomized Trial of Carboplatin vs. Cisplatin for Standard Risk Pediatric and Adult Patients with Germ Cell Tumors
This partially randomized phase III trial studies how well active surveillance, bleomycin, carboplatin, etoposide, or cisplatin work in treating pediatric and adult patients with germ cell tumors. Active surveillance may help doctors to monitor subjects with low risk germ cell tumors after their tumor is removed. Drugs used in chemotherapy, such as bleomycin, carboplatin, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.
• newly diagnosed with a Stage I germ cell tumor or metastatic germ cell tumor
• see link to clinicaltrials.gov for detailed inclusion criteria
• patients must have had no prior systemic therapy for the current cancer diagnosis
• patients must have had no prior radiation therapy (exception of CNS irradiation of brain metastases for standard risk 1 patients)
• female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs
• lactating females who plan to breastfeed their infants
• there are additional exclusion criteria (study staff will review)
Lifestyle Counseling and Medication for Adolescent Weight Management (QUEST)
This study will compare the effectiveness and durability of intensive behavioral counseling vs. medical management plus low-intensity behavioral counseling on BMI, body fat, cardiometabolic risk factors, and quality of life in adolescents with severe obesity. We hypothesize that Wegovy (semaglutide) plus low-intensity behavioral counseling will elicit superior reductions in BMI and body fat and greater improvement in cardiometabolic factors and quality of life compared to intensive behavioral counseling at 56 weeks.
• ages 12-17
• BMI greater than or equal to the 95th percentile for age and sex
• Type 1 or Type 2 diabetes
• use of medications for obesity in the past 6 months
• any treatment with growth hormone
• bariatric surgery -major mental health diagnosis (study staff will review)
• pregnant or plan to become pregnant
• significant medical diagnosis (study staff will review)