A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants with Duchenne Muscular Dystrophy

BioMarin Pharmaceutical

Status and phase

Enrolling

Phase 2

Phase 1

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: BMN 351

Study type

Interventional

Funder types

Industry

Identifiers

NCT06280209

2023-506737-30-00 (Other Identifier)

351-201

Details and patient eligibility

About

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.

Full description

This is Phase 1/2, 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.

Enrollment

18 estimated patients

Sex

Male

Ages

4 to 10 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Age 4 to 10
Diagnosis of Duchenne muscular dystrophy with a specific genetic change amenable to exon 51 skipping
Able to walk
Not requiring assistance from a ventilator to breathe
Currently on consistent doses of steroid treatment for the last 12 weeks

Exclusion criteria

The participant will have some initial clinical labs and studies to assess baseline level of heart and lung function.
Treatment with an exon skipping therapy within 12 weeks prior to the first visit.
Any history of treatment with gene therapy

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

18 participants in 4 patient groups

Cohort 1A

Experimental group

Description:

Cohort 1A will consist of both a single ascending dose (SAD) part and a multiple ascending dose (MAD). BMN 351 will be administered once every 2 weeks during the SAD portion of the study for up to 8 weeks and once weekly during the MAD portion for up to 56 weeks.

Treatment:

Drug: BMN 351

Cohort 1B

Experimental group

Description:

BMN 351 low dose will be administered once weekly for up to 48 weeks

Treatment:

Drug: BMN 351

Cohort 2

Experimental group

Description:

BMN 351 medium dose will be administered once weekly for up to 48 weeks