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rare disease orphan drug trials recruiting
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“rare disease orphan drug trials recruiting”
NCT06219421
Recruiting
Automatic Phenotyping of Patients on 2D Photography
N/A
Dysmorphia
Orphan Diseases
Dysmorphies Craniofaciales
Clinical data reuse
Imagine Institute
22,000 participants
1 site · France
Completes Mar 2028
More detail
CT.gov
Full analysis →
NCT05136976
Recruiting
Rituximab Therapy in Anti-Myelin Associated Glycoprotein Patients With Characteristics of Good Responders
Phase 3
Anti-MAG Neuropathy
Rituximab infusion
Placebo infusion
Premedications
Centre Hospitalier Universitaire de Saint Etienne
90 participants
15 sites · France
Completes Dec 2027
More detail
CT.gov
Full analysis →
NCT06065852
Recruiting
National Registry of Rare Kidney Diseases
N/A
Adenine Phosphoribosyltransferase Deficiency
AH Amyloidosis
AHL Amyloidosis
UK Kidney Association
35,000 participants
1 site · United Kingdom
Completes Dec 2039
More detail
CT.gov
Full analysis →
NCT06845501
Recruiting
Purine Supplementation in Patients With AICA-Ribosiduria
N/A
AICA-ribosiduria Due to ATIC Deficiency
purine-rich diet
Centre Hospitalier Universitaire de Saint Etienne
10 participants
1 site · France
Completes Mar 2027
More detail
CT.gov
Full analysis →
NCT01793168
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
N/A
Rare Disorders
Undiagnosed Disorders
Disorders of Unknown Prevalence
Sanford Health
20,000 participants
2 sites · United States, Australia
Completes Dec 2100
More detail
CT.gov
Full analysis →
NCT03087253
Recruiting
The LD Lync Study - Natural History Study of Lipodystrophy Syndromes
N/A
Lipodystrophy (Genetic or Acquired, Non HIV)
University of Michigan
500 participants
3 sites · United States, Turkey (Türkiye)
Completes Mar 2031
More detail
CT.gov
Full analysis →
NCT04220190
Recruiting
RAPA-501 Therapy for ALS
Phase 2
Phase 3
Amyotrophic Lateral Sclerosis
RAPA-501 Autologous T stem cells
Rapa Therapeutics LLC
41 participants
1 site · United States
Completes Jul 2026
More detail
CT.gov
Full analysis →
NCT04656184
Recruiting
A Trial Comparing the Efficacy and Safety of Anakinra Versus Intravenous Immunoglobulin (IVIG) Retreatment, in Patients With Kawasaki Disease Who Failed to Respond to Initial Standard IVIG Treatment
Phase 3
Kawasaki Disease
ANAKINRA
Intravenous immunoglobulin
Assistance Publique - Hôpitaux de Paris
84 participants
1 site · France
Completes Mar 2027
More detail
CT.gov
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