About
Orphan Disease Treatments
Areas of Focus
Categories
Small Molecule PharmaceuticalVirginia, DC, DE & Maryland
Team (5)
Clinical Trials (17)
View allTotal Trials
17
As Lead Sponsor
6
As Collaborator
11
Total Enrollment
963
By Phase
PHASE3: 3PHASE2: 5PHASE1: 3EARLY_PHASE1: 1NA: 2PHASE1/PHASE2: 1
By Status
COMPLETED: 12TERMINATED: 3WITHDRAWN: 1ACTIVE_NOT_RECRUITING: 1
Clinical Trials Pipeline17 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
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Today
Hover over trial bars to see details
Recent Trials
NCT06260748Lead Sponsor
A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
PHASE3Withdrawn
Started: May 27, 2024 · Completed: Dec 31, 2025
NCT04231266Lead Sponsor
Multi-Center Study of ManNAc for GNE Myopathy
PHASE2Active, not recruiting
54 participants
Started: Apr 5, 2022 · Completed: Oct 30, 2025
NCT05499026Lead Sponsor
Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
N/ACompleted
28 participants
Started: Dec 9, 2014 · Completed: Jun 3, 2015
NCT02043587Collaborator
Chemotherapy With Liposomal Cytarabine CNS Prophylaxis for Adult Acute Lymphoblastic Leukemia & Lymphoblastic Lymphoma
PHASE2Terminated
31 participants
Started: Jan 31, 2014 · Completed: Apr 14, 2022
NCT01954017Lead Sponsor
STP206 for the Prevention of Necrotizing Enterocolitis (NEC)
PHASE1Completed
103 participants
Started: Jan 30, 2014 · Completed: Oct 22, 2018
Locations (1)
Headquarters
Rockville, MD, United States
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