Ignite Creation Date:
2024-05-06 @ 8:11 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06280209
Status:
RECRUITING
Last Update Posted:
2024-06-18
First Post:
2024-01-26
Brief Title:
A Phase 12 Study to Assess the Safety Tolerability Pharmacokinetics and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
A Phase 12 Open-Label Dose Escalation Study to Assess the Safety Tolerability Pharmacokinetics and Pharmacodynamics of Multiple Intravenous Doses of BMN 351 in Participants With Duchenne Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy DMD with a genetic mutation amenable to exon 51 skipping
Detailed Description:
This is Phase 12 open-label multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy DMD with genetic mutations amenable to exon 51 skipping
Participants will be assigned to one of three groups called cohorts Cohort 1 2 or 3 Cohort 1 participants are further divided into Cohort 1A and Cohort 1B In Cohort 1A 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351 In part 2 the participants in cohort 1A will transition to once weekly dosing The participants in Cohort 1B 2 and 3 will initiate low medium and high doses of BMN 351 and continue once weekly dosing at that same dose The study will enroll approximately 18 participants
Participants will be assigned to one of three groups called cohorts Cohort 1 2 or 3 Cohort 1 participants are further divided into Cohort 1A and Cohort 1B In Cohort 1A 3 participants will receive increasing doses once every 2 weeks with a visit to assess safety measures collected the week after dosing prior to escalating doses of BMN 351 In part 2 the participants in cohort 1A will transition to once weekly dosing The participants in Cohort 1B 2 and 3 will initiate low medium and high doses of BMN 351 and continue once weekly dosing at that same dose The study will enroll approximately 18 participants
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2023-506737-30-00 | OTHER | EU CT | None |