Rare Disease clinical trials

Explore recruiting rare disease clinical trials.

Recruiting rare disease studies right now

Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record
Study details & eligibility ClinicalTrials.gov record

Also recruiting in Europe

Rare Disease trials registered in the EU Clinical Trials Information System (CTIS). These are outside the "near me" matcher above - open a study for full details.

A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Disease.

Italy, Germany

Phase I and Phase II (Integrated)- First administration to humans EU CTIS Study details

BeCALM - Brentuximab vedotin in CutAneous T-cell Lymphomas (CTCL): post-allogeneic hematopoietic stem cell transplant Maintenance

France

Therapeutic confirmatory (Phase III) EU CTIS Study details

A first-in-human, two-part, open-label, clinical study to assess the safety, tolerability, and activity of intravenous doses of ICT01 as monotherapy and in combination with an immune checkpoint inhibitor, in patients with advanced-stage, relapsed/refractory cancer (EVICTION Study)

Belgium, France, Spain, Germany

Phase I and Phase II (Integrated)- First administration to humans EU CTIS Study details

A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects with Late Infantile Metachromatic Leukodystrophy

Germany, France, Belgium, Greece, Spain, Italy

Therapeutic exploratory (Phase II) EU CTIS Study details

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alpha (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease

Czechia, Italy, Norway, Belgium, Denmark

Therapeutic confirmatory (Phase III) EU CTIS Study details

The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants with Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Hungary, France, Sweden, Italy, Spain

Therapeutic confirmatory (Phase III) EU CTIS Study details

Rare Disease trials by city

Rare Disease in Toronto Rare Disease in Vancouver Rare Disease in Montreal Rare Disease in Calgary Rare Disease in Edmonton Rare Disease in Ottawa Rare Disease in Winnipeg Rare Disease in Quebec City Rare Disease in Hamilton Rare Disease in Kitchener Rare Disease in London Rare Disease in Halifax Rare Disease in New York Rare Disease in Los Angeles Rare Disease in Chicago Rare Disease in Houston

Other conditions

Obesity Type 2 diabetes Type 1 diabetes Prediabetes Weight loss Metabolic syndrome High cholesterol High triglycerides High blood pressure PCOS Hypothyroidism Thyroid disease Gout Fatty liver disease (NAFLD/NASH) Heart failure Coronary artery disease Atrial fibrillation Stroke Stroke recovery Peripheral artery disease Pulmonary hypertension Deep vein thrombosis Chronic kidney disease Diabetic kidney disease