Search Results Within Category "Rare Diseases"
MT2023-29: Long-term Follow-up of Subjects With Sickle Cell Disease Treated With ExVivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
The purpose of this study is to evaluate the long-term safety and ability of a transplant with gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. Participants must have received investigational gene therapy with bb1111 in a clinical study sponsored by bluebird bio. There is no additional treatment associated with this study as this is a long-term follow-up study.
• 2 to 53 years old
• treated with a clinical product to Sickle Cell Disease (SCD) in clinical study sponsored by bluebird bio-
• there are no exclusion criteria for this study
Phase 2b Multicentre, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Dose-Ranging Study to Assess the Efficacy, Safety, and Tolerability of AZD2373 in Participants with APOL1-Mediated Kidney Disease (APPRECIATE) (APPRECIATE)
This study is for adults with APOL1-Mediated Kidney Disease (AMKD). Researchers are testing a study medicine called AZD2373 to learn if it may help treat this type of kidney disease and how safe it is to take.
• 18 to 70 years old
• of African descent, including Black, African American, Afro-Caribbean, Afro-Latino, West African, or other African diaspora heritage
• have a high-risk APOL1 genetic type (G1/G1, G1/G2, or G2/G2)
• diagnosis of kidney disease with increased protein in the urine and reduced kidney function
• see link to clinicaltrials.gov for complete inclusion criteria
• body Mass Index (BMI) greater than 45 kg/m²
• diagnosis of type 1 diabetes or certain concerns related to type 2 diabetes, serious heart conditions, high blood pressure, abnormal heart test results, certain types of kidney disease not related to APOL1, or a bleeding disorder
• receiving dialysis or have had a kidney or other organ transplant
• see link to clinicaltrials.gov for complete exclusion criteria
REMIND: An Investigator Initiated Study Testing the Feasibility of Maintenance Radiotherapy for Patients with Newly Diagnosed Glioblastoma
This study is testing whether a single additional treatment of radiation therapy, called maintenance radiotherapy, is safe and feasible for adults who have recently been diagnosed with glioblastoma (GBM). Participants will first receive their usual treatment for GBM, including surgery, radiation therapy, and the chemotherapy drug temozolomide. About one month after completing standard radiation therapy, participants will receive one additional treatment of radiation therapy. The study will also look at changes in certain immune cells before and after the additional radiation treatment. Participants will continue their usual follow-up care every 2 to 4 months for up to 5 years after the additional treatment.
• 18 years of age or older
• newly diagnosed with glioblastoma (GBM) and planning to receive standard treatment, including surgery, radiation therapy, and temozolomide (TMZ)
• currently pregnant or breastfeeding
• cancer involves the brainstem within the area planned for radiation
• previous radiation therapy to the brain
• unable to safely have MRI or CT scans
MT2025-20: A Phase II, multi-part, five-year, randomized, open-label, assessor-blinded, active-controlled, multicenter study to evaluate the efficacy and safety of rapcabtagene autoleucel versus rituximab treatment in participants with severe refractory diffuse cutaneous systemic sclerosis
The goal of this study is to find out if the experimental drug rapcabtagene autoleucel (herein referred to as YTB323), an investigational new therapy, is safe and effective (can help) for people who have severe refractory diffuse cutaneous systemic sclerosis (dcSSc). YTB323 is a chimeric antigen receptor T cell (CAR-T cell) therapy, which is a type of gene therapy or immunotherapy. CAR-T involves collecting and using a patient’s own immune cells, specifically their T cells, to treat their condition.
• 18 to 70 years old
• diagnosis of systemic sclerosis and meet the criteria for diffuse cutaneous (dcSSc)
• Disease onset from the first symptoms attributable to SSc (e.g., puffy hands, scleroderma, digital ulcers, arthralgia, dyspnea) within past 7 years
• severe, progressive systemic sclerosis disease
• see link to clinicaltrials.gov for complete Inclusion criteria
• Rheumatic disease other than dcSSc
• pulmonary hypertension
• significant kidney disease
• uncontrolled hypertension
• see link to clinicaltrials.gov for complete Exclusion criteria
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
MT2026-02: A Phase III, Randomized Study of Daratumumab, Cyclophosphamide, Bortezomib and Dexamethasone (Dara-VCD) Induction Followed by Autologous Stem Cell Transplant or Dara-VCD Consolidation and Daratumumab Maintenance in Patients with Newly Diagnosed AL Amyloidosis (SWOG S2213)
The purpose of this study is to compare two treatment approaches for people with amyloidosis: daratumumab, cyclophosphamide, bortezomib, and dexamethasone (Dara-VCD) chemotherapy followed by an autologous stem cell transplant versus Dara-VCD followed by daratumumab maintenance therapy without a stem cell transplant.
• diagnosis of systemic amyloid light chain (AL) amyloidosis
• may receive up to one cycle (or 28 days) of therapy prior to starting the study
• may be receiving chronic corticosteroids if they are being given for other disorders
• must be willing to undergo high dose chemotherapy and autologous stem cell transplantation
• see link to clinicaltrials.gov for complete inclusion criteria
• women who are pregnant or breastfeeding
• other uncontrolled illnesses including diabetes, hypertension, heart or lung disease
• see link to clinicaltrials.gov for complete exclusion criteria