Mucopolysaccharidosis III clinical trials
The registry lists 58 studies for Mucopolysaccharidosis III across more than 10 countries. 16 are recruiting or about to start. 47 of them are mainly about Mucopolysaccharidosis III (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-02.
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Status
Who runs them
Shire (6) · Allievex Corporation (5) · Alexion Pharmaceuticals, Inc. (5) · Ultragenyx Pharmaceutical Inc (4) · LYSOGENE (3) · Phoenix Nest (3) · Masonic Cancer Center, University of Minnesota (2) · Swedish Orphan Biovitrum (2)
What is being tested here
Drugs under test in Mucopolysaccharidosis III studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6, phase 3 first
- Apr 2027Study of Cannabidiol in Sanfilippo Syndrome · Phase 3 · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
- Aug 2027Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA · Phase 3 · Ultragenyx Pharmaceutical Inc
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Dec 2026MPS (RaDiCo Cohort) (RaDiCo-MPS) · Institut National de la Santé Et de la Recherche Médicale, France
- Jun 2027Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA) · GC Biopharma Corp
- Oct 2027Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE · Allievex Corporation
Who these studies take
Of the 8 Mucopolysaccharidosis III studies recruiting now, 5 (63%) set no upper age limit and 1 (13%) stop at 75 or younger. 7 (88%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 7 (88%) | 7 (88%) |
| 18 | 8 (100%) | 8 (100%) |
| 30 | 7 (88%) | 7 (88%) |
| 40 | 7 (88%) | 7 (88%) |
| 50 | 7 (88%) | 7 (88%) |
| 65 | 7 (88%) | 7 (88%) |
| 75 | 7 (88%) | 7 (88%) |
| 85 | 7 (88%) | 7 (88%) |
Across all 58 studies, recruiting or not: 52% stop at 75 or younger and 50% at 65; 98% take children; 0% are for women only and 0% for men only; 3% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Started recruiting1
Studies
Recruiting first, then most recently updated. Showing 50 of 58. Browse all 58 → or search them.
- Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)RecruitingNCT06181136Phase 1/21 country
- Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)AvailableNCT077338561 country
- MPS IIIA Disease Monitoring ProgramNot yet recruitingNCT07855562
- Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSHRecruitingNCT02716246Phase 2/33 countries
- Intracerebroventricular Tralesinidase Alfa in Participants With MPS IIIB (Sanfilippo Syndrome Type B)Not yet recruitingNCT07579910Phase 3
- Study of Cannabidiol in Sanfilippo SyndromeRecruitingNCT06333041Phase 2/31 country
- Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIBNot yet recruitingNCT07818759Phase 1/21 country
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT053680381 country
- Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIAEnrolling by invitationNCT04360265Phase 33 countries
- Natural History to Assess Disease in Patients With MPS IIICNot yet recruitingNCT077120031 country
- Natural History Study of Participants With Sanfilippo Syndrome Type IIICRecruitingNCT058251312 countries
- MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingNCT060366931 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS IIIRecruitingNCT06614894Phase 2/31 country
- The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)RecruitingNCT057056741 country
- Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLEEnrolling by invitationNCT05492799Phase 45 countries
- An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIBActive, not recruitingNCT06488924Phase 1/21 country
- Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)Active, not recruitingNCT06567769Phase 12 countries
- Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIAActive, not recruitingNCT06095388Phase 1/21 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- A Natural History Study of Sanfilippo Syndrome Type DCompletedNCT056488511 country
- Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIAActive, not recruitingNCT04201405Phase 1/21 country
- A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)CompletedNCT055232061 country
- Open-label Study of Anakinra in MPS IIICompletedNCT04018755Phase 1/21 country
- Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA DiseaseTerminatedNCT04088734Phase 1/23 countries
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- A Treatment Extension Study of Mucopolysaccharidosis Type IIIBStatus unknownNCT03784287Phase 27 countries
- A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)Active, not recruitingNCT032270429 countries
- A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101TerminatedNCT046559113 countries
- Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIBTerminatedNCT03315182Phase 1/24 countries
- A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA PatientsCompletedNCT03811028Phase 1/22 countries
- A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA PatientsCompletedNCT03423186Phase 1/23 countries
- Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)Status unknownNCT03612869Phase 2/35 countries
- Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA PatientsCompletedNCT01155778Phase 1/22 countries
- Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA)TerminatedNCT01299727Phase 1/22 countries
- A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)CompletedNCT010473061 country
- An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.TerminatedNCT02350816Phase 27 countries
- Natural History Observational Study of MPS IIIa in SMCStatus unknownNCT04918641
- Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA DiseaseCompletedNCT02060526Phase 28 countries
- Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)CompletedNCT015097683 countries
- Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease NetworkCompletedNCT015868711 country
- A Treatment Study of Mucopolysaccharidosis Type IIIBCompletedNCT02754076Phase 1/27 countries
- A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)CompletedNCT024939988 countries
- Intracerebral Gene Therapy in Children With Sanfilippo Type B SyndromeCompletedNCT03300453Phase 1/21 country
- Neurobehavioral Phenotypes in MPS IIICompletedNCT018739111 country
- Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool ChildrenCompletedNCT019380141 country
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 21 country
- Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIBTerminatedNCT022934086 countries
- Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)CompletedNCT02324049Phase 1/23 countries
- A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered IntravenouslyTerminatedNCT02618512Phase 1/21 country