Clinical trials: Sanfilippo Syndrome Type A
- Condition Sanfilippo Syndrome Type A
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03811028
- Testing
- SOBI003
- Where
- 3 sites in United States, Turkey (Türkiye)
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03423186
- Testing
- SOBI003
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Sanfilippo Syndrome Type A
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including…
- Trial name
- NCT01047306
- Testing
- assessment
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Shire