drugset / Trial / NCT02232477
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Terminated
6 enrolled
agnes chen
National Institute of Neurological Disorders and Stroke (NINDS) · collabRare Diseases Clinical Research Network · collabThe Ryan Foundation · collabUCSF Benioff Children's Hospital Oakland · collabUniversity of California, Los Angeles · collabUniversity of Minnesota · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study.
Timeline
- Start
- 2014-08
- Primary completion
- 2020-02
- Completion
- 2020-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | laronidase | Protein / enzyme biologic | 1.74 mg | Intrathecal |