Clinical trials: Rare Disease
Breast Cancer, Lung Cancer
Ontario-wide Cancer TArgeted Nucleic Acid Evaluation
Substantial progress has been made in the treatment of cancer through the use of targeted therapies, but what works for one patient might not work for another patient.
- Conditions
- Breast Cancer, Lung Cancer, Colorectal Cancer, Melanoma
- Trial name
- OCTANE (NCT02906943)
- Where
- 7 sites in Canada
- Volunteers
- 10,000 people
- Sponsor
- University Health Network, Toronto
Amyotrophic Lateral Sclerosis
RAPA-501 Therapy of ALS Expanded Access Protocol
RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 T stem cells in patients living with amyotrophic lateral sclerosis (pwALS).
- Trial name
- NCT06169176
- Testing
- RAPA-501 Autologous T stem cells
- Where
- 10 sites in United States
- Sponsor
- Rapa Therapeutics LLC
Rare Genetic Disease, Undiagnosed Diseases
Research for Individualized Therapeutics in Rare Genetic Disease
The purpose of this research study is to identify individuals that have a rare genetic disease without an adequate therapeutic strategy that might be treatable with drug developed to target the disease-causing genetic alteration.
- Trial name
- NCT05236595
- Testing
- Individualized drug matching per genetic disease
- Where
- 3 sites in United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Mayo Clinic
Tay-Sachs Disease, Sandhoff Disease
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period…
- Trial name
- AMETHIST (NCT04221451)
- Testing
- venglustat GZ402671
- Where
- 23 sites in United States, Argentina and 11 more
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Myocarditis, Giant Cell Myocarditis
Phase II Randomized Study of Muromonab-CD3, Cyclosporine, Methylprednisolone, and Prednisone in Patients With Giant Cell Myocarditis
OBJECTIVES: I. Assess the effect of immunosuppression with muromonab-CD3, cyclosporine, methylprednisolone, and prednisone versus standard care in terms of death, heart transplantation, or left ventricular assistive device…
- Trial name
- NCT00004482
- Testing
- Cyclosporine, methylprednisolone, Muromonab-CD3
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Mayo Clinic
Lung Cancer
Personalized Sequence for Thoracic, Esophageal, and H&N Cancer
The next generation of personalized medical treatment according to the type of personal genetic information are evolving rapidly.
- Trial name
- NCT02299622
- Testing
- Genetic test
- Where
- South Korea
- Volunteers
- 200 people
- Sponsor
- Samsung Medical Center
Lung Cancer
Osimertinib Plus Chemotherapy in Uncommon EGFRm NSCLC
This is an open-label, single-arm, multicenter, exploratory Phase II study sponsored by Astrazeneca Investment (China) Co., LTD.
- Trial name
- MINOVA (NCT05215951)
- Testing
- osimertinib
- Where
- 6 sites in China
- Volunteers
- 4 people
- Sponsor
- AstraZeneca
Prostate Cancer Metastatic to Bone
Rhenium-188-HEDP vs. Radium-223-chloride in Patients With Advanced Prostate Cancer Refractory to Hormonal Therapy
Radium-223 chloride is an alpha-emitting radiopharmaceutical with proven survival benefit in patients with castration-resistant prostate cancer metastatic to bone.
- Trial name
- RaRe (NCT03458559)
- Testing
- Radium-223 chloride, Rhenium-188-HEDP
- Where
- Netherlands
- Volunteers
- 402 people
- Sponsor
- Amsterdam UMC, location VUmc
Rare Diseases, Aortic Stiffness
Aortic Stiffness in Patients With Genetic Aortopathies
The goal of this observational study is to study arterial stiffness in patients with thoracic ascending aortic aneurysms (TAA), either syndromic or non-syndromic.
- Conditions
- Rare Diseases, Aortic Stiffness, Connective Tissue Defect
- Trial name
- PULSEWAVE (NCT05715203)
- Testing
- Pulsotonometry
- Where
- Italy
- Volunteers
- 250 people
- Sponsor
- IRCCS Policlinico S. Donato
Obesity
Nutritious Eating With Soul at Rare Variety Cafe
To date, few dissemination and implementation (D\&I) studies exist of successful healthy eating interventions delivered through restaurant settings.
- Trial name
- NEWSoul@RV (NCT04513769)
- Testing
- Vegan diet
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- University of South Carolina
Spinal Cord Injury, Quadriplegia
Electrical Activation of The Diaphragm for Ventilatory Assist
OBJECTIVES: The objectives of this study are to test the Diaphragm Pacing Stimulation (DPS) System for treating chronic ventilatory insufficiency in persons with respiratory muscle paralysis.
- Trial name
- NCT00010374
- Testing
- NeuRx DPS
- Where
- 5 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Synapse Biomedical
Oligometastatic Disease
Prospective Observational Basket Study of Stereotactic Body Radiation Therapy for OligoMetastatic Patients From Rare Tumors
This prospective observational study aims to investigate the effectiveness and safety of SBRT in the management of oligometastases from rare tumors.
- Trial name
- PROMPT (NCT05903261)
- Testing
- SBRT
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Istituto Clinico Humanitas
Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura
Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center
Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic organ damage due to microvascular thrombosis.
- Conditions
- Thrombotic Thrombocytopenic Purpura (TTP), Immune Thrombotic Thrombocytopenic Purpura, Thrombotic Microangiopathies, Microangiopathy, Thromboic
- Trial name
- TWILIGHT (NCT07205861)
- Where
- France
- Volunteers
- 1,200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Disability Evaluation, Child of Impaired Parents
Motherhood With MOtor Impairment Due to MAlady (Rare Disease)
Background Women with rare diseases resulting in motor disabilities wishing to become mother face a major challenge. The investigators hypothesize that provided with adequate support, they are able to achieve a successful…
- Trial name
- MMOMA (NCT02727010)
- Where
- France
- Volunteers
- 29 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Systemic Lupus, Autoimmune Diseases
Biocollection of Rare Pediatric-onset of Autoimmune and Autoinflammatory Diseases
Rare diseases are defined as those that affect one person in 2,000, or around three million people in France. The majority of rare diseases are caused by genetics and tend to be severe when they begin in childhood.
- Conditions
- Systemic Lupus, Autoimmune Diseases, Autoinflammatory Disease, Genetic Disease
- Trial name
- GENIALII (NCT06435468)
- Testing
- Blood sample for genetic analysis, Blood sample for immunological response assessments, Blood sample to identify relevant biomarker of the disease
- Where
- 13 sites in France
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Multiple Sclerosis
Phase III Randomized, Double-Blind, Placebo-Controlled Study of Copolymer 1 for Relapsing-Remitting Multiple Sclerosis
OBJECTIVES: I. Compare tolerance to and therapeutic impact of copolymer 1, a mixture of synthetic polypeptides, with placebo in patients with relapsing-remitting multiple sclerosis.
- Trial name
- NCT00004814
- Testing
- copolymer 1
- Volunteers
- 250 people
- Sponsor
- National Center for Research Resources (NCRR)
Porphyria
Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic Attacks
OBJECTIVES: Assess whether chronic administration of gonadotropin-releasing hormone analogues is safe and effective for the prevention of cyclic attacks of acute porphyria in women.
- Trial name
- NCT00004330
- Testing
- luteinizing hormone-releasing factor
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Duchenne Muscular Dystrophy (DMD)
A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD
The primary objective of Parts 1 and 2 of the study were to establish the histologic effects of givinostat administered chronically at the selected daily dose.
- Trial name
- NCT01761292
- Testing
- Givinostat
- Where
- 4 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Italfarmaco
Glucosephosphate Dehydrogenase Deficiency, Hyperbilirubinemia
Phase II Randomized Study of Tin Mesoporphyrin for Neonatal Hyperbilirubinemia
OBJECTIVES: I. Compare the efficacy of preventive vs. therapeutic tin mesoporphyrin in direct Coombs' test-positive ABO hemolytic disease of the newborn and glucose-6-phosphate dehydrogenase deficiency in infants living in…
- Conditions
- Glucosephosphate Dehydrogenase Deficiency, Hyperbilirubinemia, Hemolytic Disease of Newborn
- Trial name
- NCT00004381
- Testing
- tin mesoporphyrin
- Where
- 2 sites in United States
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Solid Advanced Tumor, Lymphoma
Dose-finding and Dose Expansion Study of OSE-279 in Subjects With Advanced Solid Tumors or Lymphomas
This is a phase 1/2, multicenter, dose-finding and dose expansion study of OSE-279, a PD-1 blocking monoclonal antibody, in subjects with advanced solid tumors or lymphomas.
- Conditions
- Solid Advanced Tumor, Lymphoma, NSCLC (Non-small Cell Lung Cancer)
- Trial name
- NCT05751798
- Testing
- Part A: OSE-279 100mg, Part A: OSE-279 300mg, Part A: OSE-279 600mg
- Where
- 11 sites in Belgium, France and 1 more
- Volunteers
- 41 people
- Sponsor
- OSE Immunotherapeutics