Clinical trials: Rare Disease
Relapsed/Refractory Waldenstrom Macroglobulinemia, Relapsed/Refractory Richter Transformation
Study of Brexucabtagene Autoleucel in Adults With Rare B-cell Malignancies
Master protocol: The goal of this master clinical study is to test how well the study drug, brexucabtagene autoleucel, works in participants with rare B-cell malignancies: relapsed/refractory Waldenstrom macroglobulinemia (r/r…
- Conditions
- Relapsed/Refractory Waldenstrom Macroglobulinemia, Relapsed/Refractory Richter Transformation, Relapsed/Refractory Burkitt Lymphoma, Relapsed/Refractory Hairy Cell Leukemia
- Trial name
- ZUMA-25 (NCT05537766)
- Testing
- Brexucabtagene Autoleucel, Cyclophosphamide, Fludarabine
- Where
- 26 sites in United States, Austria and 6 more
- Volunteers
- 19 people
- Sponsor
- Kite, A Gilead Company
Cystinosis
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
The purpose of this study is to gather information about the effectiveness (how well it works to treat cystinosis) and safety of a new form of cysteamine bitartrate called RP103, compared to the already-approved drug cystinosis…
- Trial name
- NCT01733316
- Testing
- RP103, Cystagon®
- Where
- 14 sites in United States, Belgium and 4 more
- Volunteers
- 41 people
- Sponsor
- Amgen
Canavan Disease
Natural History Study of Patients With Canavan Disease (CANinform Study)
This study uses medical records that allow retrospective data extraction of critical milestone and motor function data. In addition, prospective assessments collect data relevant to the natural history of Canavan disease in…
- Trial name
- NCT04126005
- Where
- 3 sites in United States, Germany
- Volunteers
- 67 people
- Sponsor
- Aspa Therapeutics
Pregnancy Related
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period [LMP]).
- Trial name
- NCT07556913
- Testing
- Elafibranor
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Ipsen
Idiopathic Pulmonary Fibrosis
23andMe IPF Research Study
The long term goal of this study is to increase genetic understanding of IPF to enable the development of an effective drug for IPF that can improve the lives of those living with the condition.
- Trial name
- IPF (NCT05028894)
- Testing
- No intervention
- Where
- United States
- Volunteers
- 690 people
- Sponsor
- 23andMe, Inc.
Myelodysplastic Syndrome, Thalassemia
This Study Will Evaluate Efficacy and Safety of Deferasirox in Patients With Myelodysplastic Syndromes (MDS), Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
This study will evaluate the efficacy and safety of deferasirox in patients with MDS, thalassemia and rare anemia patients with transfusion iron overload.
- Trial name
- NCT01250951
- Testing
- Deferasirox
- Where
- 2 sites in Russia
- Volunteers
- 111 people
- Sponsor
- Novartis Pharmaceuticals
Staphylococcal Skin Infections, Wounds and Injuries
A Study for Patients With Complicated Skin and Skin Structure Infections
Two Phase III trials to demonstrate the efficacy and safety of oritavancin in the treatment of complicated skin and skin structure infections (cSSSI) have been completed.
- Conditions
- Staphylococcal Skin Infections, Wounds and Injuries, Abscess, Cellulitis
- Trial name
- SIMPLIFI (NCT00514527)
- Testing
- oritavancin
- Where
- 66 sites in United States, Australia and 4 more
- Volunteers
- 294 people
- Sponsor
- Targanta Therapeutics Corporation
MPS 7, Sly Syndrome
A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003.
- Conditions
- MPS 7, Sly Syndrome, Mucopolysaccharidosis, MPS VII
- Trial name
- NCT02230566
- Testing
- UX003
- Where
- 4 sites in United States
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Homozygous Familial Hypercholesterolemia (HoFH)
Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients
This study is designed to help identify patients with HoFH due to mutations in the LDLR as confirmed by genotyping.
- Trial name
- NCT04148001
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- REGENXBIO Inc.
Hypertension
COOL-BP Study: Continuous Versus Occasional Blood Pressure Study
The COOL-BP study is part of the Remote Hypertension Program and will investigate the data provided by Aktiia Bracelet (a cuffless Blood Pressure monitor at the wrist) when integrated into the Remote Hypertension Program.
- Trial name
- NCT05211648
- Testing
- Aktiia Bracelet
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- Aktiia SA
Ewing Sarcoma, Ewing Family of Tumors
A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide
The goal of this clinical trial was to compare participants with first relapse or refractory Ewing's sarcoma when treated with investigational product (Vigil) in addition to the standard treatment of irinotecan and temozolomide…
- Conditions
- Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic
- Trial name
- VITA (NCT03495921)
- Testing
- Vigil, Irinotecan, Temozolomide
- Where
- 15 sites in United States
- Volunteers
- 32 people
- Sponsor
- Gradalis, Inc.
Leukemia, Hematologic Malignancy
Solving Riddles Through Sequencing
During the last decades hematologists have excelled at improving and refining the classification, diagnosis, and thus ultimately the therapeutic decision-making process for their patients.
- Conditions
- Leukemia, Hematologic Malignancy, Rare Diseases, Refractory Leukemia
- Trial name
- SIRIUS (NCT05046444)
- Testing
- Next Generation Sequencing
- Where
- Germany
- Volunteers
- 100 people
- Sponsor
- Munich Leukemia Laboratory
Leukodystrophy, Globoid Cell
Study of Safety, Tolerability and Efficacy of PBKR03 in Pediatric Subjects With Early Infantile Krabbe Disease
PBKR03 is a gene therapy for Krabbe Disease (Globoid cell leukodystrophy) intended to deliver a functional copy of the GALC gene to the brain and peripheral tissues.
- Trial name
- GALax-C (NCT04771416)
- Testing
- PBKR03
- Where
- 9 sites in United States, Brazil and 4 more
- Volunteers
- 24 people
- Sponsor
- Gemma Biotherapeutics
Hereditary Transthyretin-mediated Amyloidosis With Polyneropathy, Pregnancy
TEGSEDI Pregnancy Surveillance Program
This is a worldwide safety surveillance study of pregnancy outcomes in women with hATTR-PN who may have been exposed or were not exposed to TEGSEDI prior to or during the pregnancy and of pediatric outcomes up to 1 year of age.
- Trial name
- NCT04270058
- Testing
- inotersen
- Volunteers
- 20 people
- Sponsor
- Akcea Therapeutics
Mitochondrial Diseases, Pearson Syndrome
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Mitochondrial diseases are a genetically heterogeneous group of disorders caused by mutations or deletions in mitochondrial DNA (mtDNA) displaying a wide range of severity and phenotypes.
- Trial name
- NCT03384420
- Testing
- CD34+ cells enriched with MNV-BLD
- Where
- Israel
- Volunteers
- 7 people
- Sponsor
- Minovia Therapeutics Ltd.
Asthma
A Study to Determine the Effect of Montelukast Sodium as an Episode Modifier in the Treatment of Infrequent Episodic Asthma in Children (0476-165)
A 53-week study to determine the effect of montelukast sodium when given to children (with infrequent episodic asthma) at the earliest symptoms of an acute episode of asthma.
- Trial name
- NCT00140881
- Testing
- MK0476; montelukast sodium
- Volunteers
- 220 people
- Sponsor
- Organon and Co
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Pregnancy Related
Odevixibat Pregnancy and Lactation Surveillance Program: A Study to Evaluate the Safety of Odevixibat During Pregnancy and/or Lactation
The participants of this study will be of any age who are exposed to at least 1 dose of odevixibat at any time during pregnancy (from 1 day prior to conception to pregnancy outcome) and/or at any time during lactation (up to 12…
- Trial name
- NCT06258902
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Ipsen
Carotid Artery Disease
CAPTURE 2 Post-Marketing Registry
The purposes of the registry are: 1) To provide an ongoing post-market surveillance mechanism to document clinical outcomes. 2) To provide additional information that the RX ACCULINK™ and RX ACCUNET™ can be used safely by a wide…
- Trial name
- CAPTURE 2 (NCT00302237)
- Testing
- RX ACCULINK, RX ACCUNET Embolic Protection System
- Where
- United States
- Volunteers
- 6,426 people
- Sponsor
- Abbott Medical Devices
Amyotrophic Lateral Sclerosis
RAPA-501 Therapy for ALS
RAPA-501-ALS is a phase 2/3 expansion cohort study of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS).
- Trial name
- NCT04220190
- Testing
- RAPA-501 Autologous T stem cells
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Rapa Therapeutics LLC