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Explore/rare disease orphan drug

Status

Phase

Pre-filtered to show active, later-stage trials with the strongest clinical signal.

12 results for “rare disease orphan drug” · Recruiting

Recruiting

The LD Lync Study - Natural History Study of Lipodystrophy Syndromes

N/ALipodystrophy (Genetic or Acquired, Non HIV)

University of Michigan500 participants4 sites · United States, Brazil, Turkey (Türkiye)Completes Mar 2034

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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 3Kawasaki Disease

ANAKINRAIntravenous immunoglobulin

Assistance Publique - Hôpitaux de Paris84 participants1 site · FranceCompletes Mar 2027

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Recruiting

Efficacy and Safety of Envafolimab Plus Doxorubicin and Ifosfamide for Advanced Soft Tissue Sarcoma

Phase 2Soft Tissue Sarcoma

Envafolimab combined with an AI regimen (doxorubicin and ifosfamide)

Second Affiliated Hospital, Zhejiang University, School of Medicine25 participants1 site · ChinaCompletes Dec 2026

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Recruiting

Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study

Phase 4C3 Glomerulopathy (C3G)Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Pegcetacoplan

Swedish Orphan Biovitrum150 participants101 sites · Australia, Austria, BelgiumCompletes Dec 2031

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Recruiting

Purine Supplementation in Patients With AICA-Ribosiduria

N/AAICA-ribosiduria Due to ATIC Deficiency

purine-rich diet

Centre Hospitalier Universitaire de Saint Etienne10 participants1 site · FranceCompletes Mar 2027

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Recruiting

RAPA-501 Therapy for ALS

Phase 2Phase 3Amyotrophic Lateral Sclerosis

RAPA-501 Autologous T stem cells

Rapa Therapeutics LLC41 participants1 site · United StatesCompletes Jul 2027

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Recruiting

Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome

Phase 2Fragile X Syndrome (FXS)Fragile X Syndrome

CTH120CTH120 placebo hard capsules

Connecta Therapeutics, S.L.30 participants2 sites · SpainCompletes May 2027

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Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

N/ARare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence

Sanford Health20,000 participants2 sites · United States, AustraliaCompletes Dec 2100

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Recruiting

Automatic Phenotyping of Patients on 2D Photography

N/ADysmorphiaOrphan DiseasesDysmorphies Craniofaciales

Clinical data reuse

Imagine Institute22,000 participants1 site · FranceCompletes Mar 2028

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Recruiting

National Registry of Rare Kidney Diseases

N/AAdenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis

UK Kidney Association35,000 participants1 site · United KingdomCompletes Dec 2039

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Recruiting

Rituximab Therapy in Anti-Myelin Associated Glycoprotein Patients With Characteristics of Good Responders

Phase 3Anti-MAG Neuropathy

Rituximab infusionPlacebo infusionPremedications

Centre Hospitalier Universitaire de Saint Etienne90 participants15 sites · FranceCompletes Dec 2027

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Recruiting

A Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders

N/ACystic Fibrosis (CF)

Istituto Giannina Gaslini165 participants3 sites · ItalyCompletes Dec 2026

Compare CT.gov Full analysis →

1–12 of 12

Explore/rare disease orphan drug

Status

Phase

Pre-filtered to show active, later-stage trials with the strongest clinical signal.

12 results for “rare disease orphan drug” · Recruiting

Recruiting

The LD Lync Study - Natural History Study of Lipodystrophy Syndromes

N/ALipodystrophy (Genetic or Acquired, Non HIV)

University of Michigan500 participants4 sites · United States, Brazil, Turkey (Türkiye)Completes Mar 2034

Compare CT.gov Full analysis →

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 3Kawasaki Disease

ANAKINRAIntravenous immunoglobulin

Assistance Publique - Hôpitaux de Paris84 participants1 site · FranceCompletes Mar 2027

Compare CT.gov Full analysis →

Recruiting

Efficacy and Safety of Envafolimab Plus Doxorubicin and Ifosfamide for Advanced Soft Tissue Sarcoma

Phase 2Soft Tissue Sarcoma

Envafolimab combined with an AI regimen (doxorubicin and ifosfamide)

Second Affiliated Hospital, Zhejiang University, School of Medicine25 participants1 site · ChinaCompletes Dec 2026

Compare CT.gov Full analysis →

Recruiting

Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study

Phase 4C3 Glomerulopathy (C3G)Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)

Pegcetacoplan

Swedish Orphan Biovitrum150 participants101 sites · Australia, Austria, BelgiumCompletes Dec 2031

Compare CT.gov Full analysis →

Recruiting

Purine Supplementation in Patients With AICA-Ribosiduria

N/AAICA-ribosiduria Due to ATIC Deficiency

purine-rich diet

Centre Hospitalier Universitaire de Saint Etienne10 participants1 site · FranceCompletes Mar 2027

Compare CT.gov Full analysis →

Recruiting

RAPA-501 Therapy for ALS

Phase 2Phase 3Amyotrophic Lateral Sclerosis

RAPA-501 Autologous T stem cells

Rapa Therapeutics LLC41 participants1 site · United StatesCompletes Jul 2027

Compare CT.gov Full analysis →

Recruiting

Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome

Phase 2Fragile X Syndrome (FXS)Fragile X Syndrome

CTH120CTH120 placebo hard capsules

Connecta Therapeutics, S.L.30 participants2 sites · SpainCompletes May 2027

Compare CT.gov Full analysis →

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

N/ARare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence

Sanford Health20,000 participants2 sites · United States, AustraliaCompletes Dec 2100

Compare CT.gov Full analysis →

Recruiting

Automatic Phenotyping of Patients on 2D Photography

N/ADysmorphiaOrphan DiseasesDysmorphies Craniofaciales

Clinical data reuse

Imagine Institute22,000 participants1 site · FranceCompletes Mar 2028

Compare CT.gov Full analysis →

Recruiting

National Registry of Rare Kidney Diseases

N/AAdenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis

UK Kidney Association35,000 participants1 site · United KingdomCompletes Dec 2039

Compare CT.gov Full analysis →

Recruiting

Rituximab Therapy in Anti-Myelin Associated Glycoprotein Patients With Characteristics of Good Responders

Phase 3Anti-MAG Neuropathy

Rituximab infusionPlacebo infusionPremedications

Centre Hospitalier Universitaire de Saint Etienne90 participants15 sites · FranceCompletes Dec 2027

Compare CT.gov Full analysis →

Recruiting

A Precision Medicine Approach to Assess Protein Function Loss and the Potential for CFTR Function Restoration in Cystic Fibrosis and Related Disorders

N/ACystic Fibrosis (CF)

Istituto Giannina Gaslini165 participants3 sites · ItalyCompletes Dec 2026

Compare CT.gov Full analysis →

1–12 of 12