Status | Study |
RECRUITING |
Study Name: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Condition: Mucopolysaccharidosis Type IIIA Date: 2023-12-04 Interventions: intravenous repeating dose |
Recruiting |
Study Name: Natural History Study of Patients With MPS IIIA Condition: Mucopolysaccharidosis IIIA Date: 2016-04-15 |
Recruiting |
Study Name: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Condition: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Date: 2016-03-17 Interventions: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS |
Recruiting |
Study Name: A Treatment Study of Mucopolysaccharidosis Type IIIB Condition: MPS III B Mucopolysaccharidosis Type IIIB Date: 2016-02-27 Interventions: Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Recruiting |
Study Name: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) Condition: Mucopolysaccharidosis Type IIIB Mucopolysaccharidosis Type 3 B Date: 2015-06-22 |
Enrolling by invitation |
Study Name: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. Condition: Sanfilippo Syndrome Mucopolysaccharidosis (MPS) Date: 2015-01-21 Interventions: Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |
Recruiting |
Study Name: Biomarker for Sanfilippo Disease Condition: Mucopolysaccharidosis Type 3 A Mucopolysaccharidosis Type 3 B Date: 2014-10-23 |
Completed |
Study Name: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease Condition: Sanfilippo Syndrome Date: 2014-02-10 Interventions: Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS] |
Active, not recruiting |
Study Name: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy Condition: Mucopolysaccharidosis Type III A Sanfilippo Disease Type A Date: 2014-01-16 Interventions: Genetic: SAF-301 |
Completed |
Study Name: Natural History Studies of Mucopolysaccharidosis III Condition: Mucopolysaccharidosis Type IIIA Mucopolysaccharidosis Type IIIB Date: 2013-11-26 Interventions: Procedure: Lumbar puncture Device: Magneti |