Clinical trials: Sanfilippo B
Mucopolysaccharidosis III, Type B (MPS IIIB), Sanfilippo B
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 Intravenously
This study evaluated the safety and tolerability of intravenous (IV) administration of SBC-103 in previously studied, SBC-103 treatment naïve patients with mucopolysaccharidosis III, type B (MPS IIIB, Sanfilippo B) who…
- Trial name
- CL01-T (NCT02618512)
- Testing
- SBC-103
- Where
- United Kingdom
- Volunteers
- 3 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Mucopolysaccharidosis Type III A, Sanfilippo Disease Type A
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
P2-SAF-301 is an open-label interventional study without administration of investigational product, evaluating the long-term safety and tolerability of intracerebral SAF-301 previously administered to 4 patients with Sanfilippo…
- Trial name
- NCT02053064
- Testing
- SAF-301
- Where
- France
- Volunteers
- 4 people
- Sponsor
- LYSOGENE
MPS IIIB (Sanfilippo Syndrome)
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
- Trial name
- NCT02293382
- Where
- 5 sites in United States, Brazil and 3 more
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
MPS IIIB (Sanfilippo B Syndrome)
Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
The purpose of the study is to characterize structural abnormalities in the brain and the integrity of the blood brain barrier in patients with mucopolysaccharidosis type IIIB (MPS IIIB).
- Trial name
- NCT02090179
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- Alexion Pharmaceuticals, 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