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4
results for
“rare disease orphan drug”
· Recruiting
NCT07243626
Recruiting
Efficacy and Safety of Envafolimab Plus Doxorubicin and Ifosfamide for Advanced Soft Tissue Sarcoma
Phase 2
Soft Tissue Sarcoma
Envafolimab combined with an AI regimen (doxorubicin and ifosfamide)
Second Affiliated Hospital, School of Medicine, Zhejiang University
15 participants
1 site · China
Completes Dec 2026
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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
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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
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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
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