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Search Results Within Category "Rare Diseases"
Global Registry For Novel Therapies In Rare Bone & Endocrine Conditions
This research study is for creating a registry of all ages with conditions in endocrine and both health. Registries are used very often these days by doctors and scientists to collect information and use to perform research into rare conditions. This registry will be part of a global registry, called "GloBE-Reg" with the University of Glasgow (Scotland) and with the University of MInnesota.
• for this study is not for any specific diagnosis
• any child receiving human growth hormone treatment
MT2022-41 A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial) (BEACON)
BEAM-101 is an experimental new therapy being developed for treating people with SCD and vaso-occlusive crises. The goal of this study is to see if BEAM-101 is safe and effective for people in the study. The study sponsor and study doctors would also like to see if individuals who are treated with BEAM-101 require fewer blood transfusions and experience fewer vasoocclusive crises requiring hospitalization, compared to before they received BEAM-101. This study will also measure the levels of fetal hemoglobin along with measures that assess quality of life and ability to function following treatment with BEAM-101.
• 18 to 35 years old
• documented diagnosis of sickle cell disease with specific genotypes (study staff will review)
• disease is severe
• HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
• previous transplant
• history of an overt stroke
MT2023-31: A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT) (HURCULES)
This research study is designed to compare a new gene therapy, known as OTL-203 (study drug), with a standard treatment called “allogeneic hematopoietic stem cell transplant” (allo-HSCT), to find out which is better for the treatment of MPS-IH.
• at least 28 days old to no more than 30 months old
• confirmed laboratory diagnosis of MPS-IH
• evidence of altered GAG metabolism
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• previous allo-HSCT or gene therapy
• diagnosis of HIV, Hepatitis B, Hepatitis C, or Mycoplasma
• history of uncontrolled seizures
• contraindications for MRI scans
• study staff will review additional exclusion criteria
VX21-522-001: A Phase 1 Multiple Dose Escalation Study Evaluating the Safety and Tolerability of VX-522 in Subjects 18 Years of Age and Older With Cystic Fibrosis and a CFTR Genotype Not Responsive to CFTR Modulator Therapy
This is a clinical research study exploring the safety and tolerability of a single dose of VX-522 for people with cystic fibrosis (CF) who are not expected to benefit from CFTR modulators.
• 18 to 65 years old
• Stable cystic fibrosis disease
• FEV1 at least 40%
• Specific CFTR gene mutations
• Uncontrolled asthma in the last year
• Oxygen saturation without oxygen therapy is >94%
• Severe liver disease
A multicenter, single-arm, open label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult atypical hemolytic uremic syndrome (aHUS) patients who are naive to complement inhibitor therapy (APPELHUS)
To evaluate the efficacy and safety of iptacopan at a dose of 200 mg twice a day for 52 weeks in patients with atypical hemolytic uremic syndrome (aHUS).
• at least 18 years old
• evidence of active Thrombotic Microangiopathy (TMA)
• previous or ongoing treatment with complement inhibitors, including anti-C5 antibody
• ADAMTS13 deficiency
• positive test for Shiga toxin * direct Coombs test
• had a bone marrow transplant or hematopoietic stem cell transplant, or a heart, lung, small bowel, pancreas or live transplant
Efficacy and safety of GLP-1 agonist therapy in overweight and obese subjects with cystic fibrosis-related diabetes: a pilot study
In this study we will be looking at the safety and effectiveness of the medication GLP-1 (Semaglutide) in patients who are overweight and have been diagnosed with Cystic Fibrosis Related Diabetes (CFRD).
• diagnosis of cystic fibrosis
• diabetic using insulin
• BMI 26 kg/m2 or greater
• able to read & speak English
• personal or family history of medullary thyroid cancer
• chronic GI problems requiring hospitalization in the past year
• history of suicide attempts or active ideas of suicide
SPR001-205 A Phase 2 Study to Evaluate the Safety, Pharmacokinetics,;and Exploratory Pharmacodynamics of SPR001 (Tildacerfont) in Children: Aged 6 to 17 Years with Congenital Adrenal Hyperplasia
The goal of this study is to test the safety and effectiveness of tildacerfont in children with congenital adrenal hyperplasia (CAH). When a child is enrolled in the study, in addition to taking the study drug (tildacerfont), he or she will continue to take his or her standard glucocorticoid doses. A part of the study will be to test different doses of the study drug and to measure adrenal hormones at each visit. Children will be in the study for 18 weeks and will have to visit the study clinic 5 times.
• age 2 to 17 years
• childhood diagnosis of classic congenital adrenal hyperplasia (CAH) a genetic mutation in CYP21A2
• currently taking steroids to treat CAH and on a stable dose for 1 month or more
• clinically significant unstable medical or mental health condition (study staff will review)
• females who are pregnant or nursing
• unable to swallow medications
MT2021-24: A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
The purpose of the study is to determine the safety and effectiveness of a new procedure to treat Mucopolysaccharidosis Type I Hurler-Scheie and Scheie (MPS I). This procedure involves collecting some white blood cells (termed “B cells”) and growing them outside of the body in a laboratory. While the cells are in the lab, the B cells will be changed to produce more of the IDUA that is missing. This process is called “genetic modification.” The newly modified B cells are then infused back into the participant.
• diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome
• creatinine clearance, calculated or measured directly, that is greater than 60ml/min/1.73m2
• ejection fraction at least 40% by echocardiogram
• must agree to stay <45-minute drive from the study site for a minimum of 5 days after cell infusion.
• must commit to traveling to the study site for the necessary follow-up evaluations.
• known family inherited cancer syndrome
• had a previous hematopoietic stem cell transplant (HSCT)
• any medical condition likely to interfere with assessment of safety or efficacy of the study treatment (study staff will review)
An International, Phase 3, Randomized, Multicenter, Open label Study of Ripretinib vs Sunitinib in Patients with Advanced Gastrointestinal Stromal Tumor (GIST) with KIT Exon 11 and Co occurring KIT Exons 17 and/or 18 Mutations Who Were Previously Treated with Imatinib (INSIGHT) (INSIGHT)
This study is being done to learn how well ripretinib works against cancer as compared to sunitinib in patients with a specific GIST-gene mutation who have received imatinib. We will also learn more about the safety of ripretinib and look at how ripretinib may affect your body. The choice of whether you will be given ripretinib or sunitinib will be assigned by a computer, by chance, like the flip of a coin. You will have a 2 out of 3 chances of receiving ripretinib. You will know if you are receiving ripretinib or sunitinib.
• diagnosis of GIST with co-occurring KIT exons 11+17/18 mutations confirmed by ctDNA sample
• disease progression on imatinib treatment, confirmed by scan
• ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
• participants of reproductive potential must agree to follow contraception requirements
• contact study staff for additional inclusion criteria
• known active central nervous system metastases
• heart disease, myocardial infarction within 6 months of starting the study, active ischemia or any other uncontrolled cardiac condition such as angina, significant cardiac arrhythmia requiring therapy, uncontrolled hypertension, or congestive heart failure
• Gastrointestinal abnormalities such as inability to take oral medication, malabsorption syndromes, requirement for intravenous alimentation
• additional exclusions apply malabsorption syndromes requirement for intravenous alimentation
MT2023-06: A CLINICAL STUDY TO ASSESS THE EFFICACY AND SAFETY OF LERIGLITAZONE IN ADULT MALE SUBJECTS WITH CEREBRAL ADRENOLEUKODYSTROPHY (CALYX)
This study has 2 parts: a double-blind period and an open-label extension. In the double-blind period of this study, the study medicine will be compared to a placebo. A placebo is a treatment that looks and tastes exactly like the study medicine but does not contain any active ingredient. In this study, you will receive leriglitazone or placebo. Whether you receive leriglitazone or placebo will be decided randomly (by chance, like flipping a coin). In this study, 1 out of every 2 subjects (50%) will receive leriglitazone and 1 out of every 2 subjects (50%) will receive placebo. To make this study fair, you and the study doctor will not be told which treatment you will receive, this is called “blinding”. In the open-label extension, all subjects will receive leriglitazone.
• diagnosis of progressive cerebral adrenoleukodystrophy (cALD), defined as GdE with brain lesions
• bone marrow transplantation (HSCT) is not recommended patient is not willing to undergo HSCT
• no major cognitive impairment
• see link to clinicaltrials.gov for additional inclusion criteria
• or treatment with ex-vivo gene therapy (eli-Cel).
• known type 1 or type 2 diabetes
• see link to clinicaltrials.gov for additional exclusion criteria
HEALEY ALS Platform Trial
The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. A regimen is a specific course of treatment, each with a different study drug. We are doing this research to find out if different treatments have an effect on Amyotrophic Lateral Sclerosis (ALS). We also want to find out if these treatments are safe to take without causing too many side effects.
• 18 years or older
• diagnosed with sporadic or familial ALS
• weakness started no more than 36 months ago -able to to swallow pills and liquids
• unstable medical or mental health condition
• limitations on prior or current use of certain medications (study staff will review)
• women who are pregnant or breast feeding
A randomized, open-label, multi-center, comparative trial, to assess the efficacy and safety of pritelivir versus foscarnet for the treatment of acyclovir-resistant mucocutaneous HSV infections in immunocompromised subjects (PRIOH-1) (PRIOH-1)
The purpose of this research study is to look at the safety and effectiveness of pritelivir given orally (by mouth for a maximum of 42 days) for people with an impaired immune system who have recurrent lesions caused by the form of HSV that does respond to treatment with acyclovir.
• at least 16 years old
• immunocompromised or body is unable to fight off infection
• have lesions that can been seen in order to determine if they are healing
• willing to use highly effective birth control
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
• history or current evidence of gastrointestinal malabsorption
• on hemodialysis for any reason and end stage renal disease (ESRD)
• women who are pregnant or breastfeeding
• unable to communicate with study staff
A seamless, Phase 1b/2 multiple ascending dose/proof of concept study of XTMAB-16 in patients with pulmonary sarcoidosis with or without extrapulmonary manifestations
XTMAB-16 is a new, experimental drug and is part of a group of drugs known as tumor necrosis factor alpha (TNF α) inhibitors. TNF is a protein in your body that causes inflammation. TNF α inhibitors work by suppressing part of the immune response along with reducing inflammation. We are conducting this research study to see if XTMAB-16 will help in the treatment of pulmonary sarcoidosis.
• Diagnosis of pulmonary sarcoidosis with some respiratory symptoms
• Must be taking 7.5 to 25 mg of prednisone daily and will to taper down dose
• Must be on an additional medication for the treatment of sarcoidosis
• Please contact umnsarc@umn.edu if you have any questions
• Pregnancy or breast-feeding
MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
Syn-Sleep Study
The purpose of the study is to evaluate the use of the Syn-One Test for identifying patients with idiopathic rapid eye movement sleep disorder (iRBD) that will progress to synucleinopathies, such as Parkinson’s disease, dementia with Lewy bodies, pure autonomic failure and multiple system atrophy. Your participation will last approximately 2 years.
• age 18 to 65
• diagnosis of Rapid Eye Movement (RBD) sleep disorder
• have repeated episodes of sleep related vocalizations and/or complex motor behaviors
• documented by sleep study to occur during REM sleep
• diagnosis of Parkinson's disease, dementia, multiple system atrophy or any other cause
• unable to have a skin biopsy because of poor wound healing, use of anticoagulants other than aspirin or Plavix, or severe vascular disease
MT2022-45 Primary Immune Regulatory Disorders (PIRD): Longitudinal Study of Clinical Presentation, Treatment and Outcomes
Primary Immune Regulatory Disorders (PIRD disorders) are a group of diseases that cause the immune system to function abnormally and cause infections, autoimmunity or inflammation that can begin early in life. PIRD is usually caused by changes in genes in DNA. Researchers are trying to learn what types of medical problems patients with PIRD have and how these respond to treatment. Researchers also want to learn which genes cause PIRD and how it can cause the medical problems of PIRD.
• age 0 to 99 years
• diagnosis of immune-mediated bowel disease affecting at least one segment of the bowel
• evidence of interstitial lung disease (ground-glass opacities) or pulmonary nodules/cysts
• decreased lung function
• additional inclusion and exclusion criteria apply (study staff will review)
• also enrolling parent, sibling, or child of eligible participants
• documented HIV infection
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
MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
• patient's parent/ legal guardians are unable to provide informed consent.
MT2022-30: A double-blind, randomized, placebo-controlled, interventional, multicenter, phase llI clinical trial to investigate the safety and efficacy of ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs) on epidermolysis bullosa (EB)", allo-APZ2-EB-III
This is a study of an investigational drug (also known as the “study drug”) called allo-APZ2-OTS as a possible treatment for RDEB. The study drug contains allogeneic dermal ABCB5-positive mesenchymal stromal cells (ABCB5+ MSCs). These cells suppress inflammation and produce proteins that are important for wound healing. The study drug is expected to counteract local inflammation, improve wound healing and skin strength, and speed up wound closure.
• confirmed diagnosis of RDEB or JEB
• wound meeting the following criteria: 5-50 cm, present for at least 21 days and less than 9 months without signs of acute infection
• women of childbearing potential, male participants, and their partner must be willing to use highly effective contraceptive methods during the course of the entire clinical trial
• current cancer diagnosis including squamous cell carcinoma and basal cell carcinoma
• women who are pregnant or breast feeding
• clinically significant or unstable medical or mental health diagnosis (study staff will review)
Study of Nutraceutical Intervention with High Phenolic Extra Virgin Olive Oil and Curcumin for Neurofibromatosis, type 1 (NF1)
This is a single center, open label, Phase I clinical trial of bioactive curcumin with high phenolic extra virgin olive oil (HP-EVOO) to treat cutaneous neurofibromas (cNF) in Neurofibromatosis, type 1 (NF1) patients (aged 18 years or older).
• clinical diagnosis of Neurofibromatosis type 1 and/or genetic testing
• measurable skin neurofibromas
• treatment with selumetinib or other MAPK, MEK or mTOR inhibitors, other targeted therapies, chemotherapy or radiation (study staff will review)
• swallowing difficulties or strong gag reflex that make it difficult to take study treatment
• supplement with high phenolic olive oil or curcumin within six months
• women who are pregnant or anticipate becoming pregnant
• history of other physical or mental health issues (study staff will review)
MT2019-01: Adrenoleukodystrophy National Registry Study (ALD) and Biobank
The purpose of this research to enhance our understanding of adrenoleukodystrophy ALD and study biospecimens such assaliva, blood, urine and stool to identify potential biomarkers for early identification of dise. We invite people who have or are at risk to have ALD, including females who are known or at risk carriers of the mutation for ALD, to help us learn more.
• age 0 to 100
• patient or family member diagnosed with ALD (confirmed by positive VLCFA testing and/or genetic mutation
• patient or family member with known or presumed mutation with ALD based on pedigree or confirmed mutation in ABCD1 gene
• living in the United States and territories
• have undergone BMT or other cellular therapy
• not fluent in English who are unable to consent in-person
• people who are unable to read or write
A multicenter, single arm, open-label trial to evaluate efficacy and safety of oral, twice daily iptacopan in adult PNH patients who have Hb >=10 g/dL in response to anti-C5 antibody and switch to iptacopan
The purpose of the study is to find out if iptacopan is effective and safe in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who switch from their current standard of care treatment (eculizumab or ravulizumab) to iptacopan.
• diagnosis of Paroxysmal Nocturnal Hemoglobinuria PNH)
• hemoglobin level at least 10 g/dL
• on a stable regimen (dose and intervals) of anti-C5 antibody treatment (either eculizumab or ravulizumab) for at least 6 months
• needed red blood cell transfusion in the past 6 months
• history of stem cell transplant or solid organ transplant
• Human immunodeficiency virus (HIV) infection (known history of HIV or test positive for HIV antibody)
• history of cancer of any part of the body within the past 5 years
A Phase 3 open-label, controlled, randomised, multi-centre trial comparing imlifidase and standard-of-care with standard-of-care alone in the treatment of severe anti-GBM antibody disease (Goodpasture disease) (GOOD-IDES-02)
The purpose of the trial is to evaluate the effect and safety of imlifidase when given to participants with antiGBM disease (also called Goodpasture disease). We will study if the addition of imlifidase to the standard of care treatment results in a better effect without causing unacceptable side effects compared to standard of care alone.
• Anti-GBM antibodies constituting an indication for Plasma exchange (PLEX)
• presence of blood or sediment in urine
• diagnosis of anti-GBM disease made more than 14 days ago
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
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
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
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
A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated with Lonapegsomatropin (SkyPASS) (SkyPASS)
The purpose of this study is to evaluate the long-term safety and effectiveness of Skytrofa treatment in children growth hormone deficiency. Patient care will follow the normal treatment practices at the clinic. No additional visits will be performed beyond the usual clinical practice.
• 1 to 18 years old
• on treatment with SKYTROFA (lonapegsomatropin)
• participating in any interventional clinical study for short stature
Monoclonal Gammopathy and Amyloidosis Registry and Biobank (MGARB)
The purpose of this study is to establish a registry and biobank to study monoclonal gammopathy and amyloidosis. We aim to understand better the changes these diseases cause, the symptoms they present, and the clinical progression to be able to better prevent them and treat them in the future.
• diagnosis of monoclonal gammopathy or amyloidosis
• unable to speak English
ALX-HPP-501: An Observational,Longitudinal Prospective, Long-term Registry of Patients with Hypophosphatasia
This is a long-term registry is designed to collect data on hypophosphatasia (HPP) to better understand the condition and learn more about the disease, how patients feel about living with HPP and effect of HPP on the patients wellbeing and health. The study will look at participant’s medical records and health questionnaires about the health status of patients. This study collects observational data from clinical care and does not involve any treatment for HPP or administration of medication for HPP.
• confirmed diagnosis of HPP.
• documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.
• able to read and speak English
• currently participating in an Alexion-sponsored clinical trial
MAYFLOWERS-0B-20: A Prospective Study Evaluating Maternal and FetaL Outcomes in the ERa of ModulatorS (MAYFLOWERS) (MAYFLOWERS)
This study is being done to look at how pregnancy affects the health of women with cystic fibrosis (CF) and to look at the health of babies born to women with CF. It is expected that most but not all women will be taking the newest CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) modulator medication called TRIKAFTA®. It is not known how this medication will affect the health of mother and infant.
• at least 16 years old
• pregnant, intending to continue pregnancy
• enrolled in the Cystic Fibrosis Foundation Patient Registry (CFFPR)
• none