Mucopolysaccharidosis Type 3a — Clinical Trials

MONDO:00096555 active·2 recruitingLast updated: Mar 19, 2026, 12:43 AM

Trial Landscape

Mucopolysaccharidosis Type 3a has an emerging clinical trial program, driven by drug treatment and biological therapy.

Recruiting

Active Trials

By Approach

Drug Treatment5

Biological Therapy1

1 additional trials involve behavioral, dietary, diagnostic, or observational approaches.

By Phase

Phase 1

Phase 2

Phase 3

Sponsor Types

Industry4Academic/Other1

Top Sponsors

Denali Therapeutics Inc.

GC Biopharma Corp

Ultragenyx Pharmaceutical Inc

Trial Locations

United States4 trials

Australia2 trials

Spain2 trials

Japan1 trial

South Korea1 trial

United Kingdom1 trial

Trials often recruit across multiple countries, so totals exceed the trial count.

5 Trials

Phase 2RecruitingGene Therapy

NCT02716246

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Sponsor:Ultragenyx Pharmaceutical Inc

United StatesStarted: Apr 2016Est. completion: Jul 2027Updated: Dec 2025

Phase 1Recruiting

NCT06567769

Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

Sponsor:GC Biopharma Corp

United StatesStarted: Nov 2024Est. completion: Jun 2027Updated: Aug 2025

Phase 3Enrolling by InvitationGene Therapy

NCT04360265

Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA

Sponsor:Ultragenyx Pharmaceutical Inc

United StatesStarted: Sep 2020Est. completion: Aug 2027Updated: Jan 2026

Phase 1Active, Not Recruiting

NCT06181136

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

Sponsor:Denali Therapeutics Inc.

United StatesStarted: Dec 2023Est. completion: Aug 2028Updated: Dec 2025

Phase 1Active, Not RecruitingGene Therapy

NCT04201405

Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

Sponsor:University of Manchester

United KingdomStarted: Jan 2020Est. completion: Oct 2026Updated: Mar 2025

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 19, 2026 at 12:43 AM