Mucopolysaccharidosis I Clinical Trial
Official title:
Phase I/II Study of JR-171 ㏌ Patients With Mucopolysaccharidosis Type I
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Terminated |
NCT01675674 -
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
|
N/A | |
| Completed |
NCT00741338 -
Immune Tolerance Study With Aldurazyme® (Laronidase)
|
Phase 1/Phase 2 | |
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
| Active, not recruiting |
NCT04453085 -
An Extension Study of JR-171-101 Study in Patients With MPS I
|
Phase 1/Phase 2 | |
| Completed |
NCT05134571 -
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
|
Phase 4 | |
| Terminated |
NCT00418821 -
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
|
Phase 4 | |
| Completed |
NCT03071341 -
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
|
Phase 1/Phase 2 | |
| Completed |
NCT03053089 -
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I
|
Phase 1/Phase 2 | |
| Completed |
NCT00146757 -
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
|
Phase 2 | |
| Active, not recruiting |
NCT04628871 -
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
|
||
| Completed |
NCT02597114 -
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
|
Phase 1 | |
| Terminated |
NCT00748969 -
Clinical Trial of Growth Hormone in MPS I, II, and VI
|
Phase 2/Phase 3 | |
| Terminated |
NCT00215527 -
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
|
Phase 1 | |
| Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
||
| Completed |
NCT00146770 -
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
|
Phase 3 | |
| Completed |
NCT00176917 -
Stem Cell Transplantation for Hurler
|
Phase 2 | |
| Completed |
NCT00176891 -
Stem Cell Transplant w/Laronidase for Hurler
|
Phase 2 | |
| Active, not recruiting |
NCT03153319 -
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
|
Phase 1/Phase 2 | |
| Completed |
NCT00852358 -
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
|
N/A | |
| Enrolling by invitation |
NCT06103487 -
Long Term Follow-Up for RGX-111
|