About
Therapies for Sanfilippo (SF) Syndrome Type A and Type B.
Areas of Focus
Categories
Biologics & BiopharmaceuticalGene Therapy, RNA & Peptide TherapyMidwest
Team (18)
Clinical Trials (11)
View allTotal Trials
11
As Lead Sponsor
9
As Collaborator
2
Total Enrollment
1,076
By Phase
PHASE3: 4PHASE1/PHASE2: 3PHASE2/PHASE3: 1PHASE2: 1
By Status
COMPLETED: 5TERMINATED: 3ACTIVE_NOT_RECRUITING: 1ENROLLING_BY_INVITATION: 1RECRUITING: 1
Clinical Trials Pipeline11 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
NCT05725018Lead Sponsor
A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients
PHASE3Active, not recruiting
12 participants
Started: Apr 2, 2023 · Completed: Sep 30, 2025
NCT05708677Lead Sponsor
A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB
N/AEnrolling by invitation
22 participants
Started: Feb 9, 2021 · Completed: Aug 31, 2036
NCT04655911Lead Sponsor
A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101
N/ATerminated
1 participants
Started: Oct 28, 2020 · Completed: Apr 7, 2022
NCT04227106Lead Sponsor
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
PHASE3Completed
11 participants
Started: Jan 10, 2020 · Completed: Oct 18, 2022
NCT04088734Collaborator
Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease
PHASE1/PHASE2Terminated
5 participants
Started: Sep 18, 2019 · Completed: Mar 10, 2022
Locations (1)
Headquarters
Cleveland, OH, United States
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