Clinical trials: Mucopolysaccharidoses
Mucopolysaccharidosis I
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT02371226
- Testing
- AGT-181 (HIRMAb-IDUA)
- Where
- 6 sites in United States
- Volunteers
- 3 people
- Sponsor
- ArmaGen, Inc
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
The clinical trial P1-SAF-301 is an open-label, single arm, monocentric, phase I/II clinical study evaluating the tolerance and the safety of intracerebral administration of adeno-associated viral vector serotype 10 carrying the…
- Trial name
- NCT01474343
- Testing
- SAF-301
- Where
- 2 sites in France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
Sanfilippo Syndrome
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase).
- Trial name
- NCT02060526
- Testing
- Recombinant human heparan N-sulfatase [rhHNS]
- Where
- 10 sites in United States, Argentina and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire