Phase 2
N=18
Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT01037309 ↗Enrolled (actual)
18
Serious AEs
7.4%
Results posted
Apr 2015
Primary outcome: Primary: Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts — 1; 1; 0; 1 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PRO044 SC (Drug); PRO044 IV (Drug)
- Age
- Pediatric · 5+ yrs
- Sex
- Male
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- May 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Increase in Dystrophin Expression in the Muscle Biopsies by Immunofluorescence Analyses of Cross-sections and by Western Blot Analyses of Total Protein Extracts |
1; 1; 0; 1; 1; 2 | — |
| PRIMARY Safety and Tolerability of PRO044 |
3; 3; 3; 3; 3; 3 | — |
| SECONDARY PRO044 Pharmacokinetic Cmax (μg/mL) Following Subcutaneous Administration |
0.4; 1.6; 4.2; 6.2; 4.9; 5.2 | — |
Summary
The purpose of this study is to see whether PRO044 is safe and effective to use as medication for DMD patients with a mutation around location 44 in the DNA for the dystrophin protein.
Eligibility Criteria
Inclusion Criteria
- Boys aged between 5 and 16 years inclusive.
- Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044.
- Life expectancy of at least 6 months.
- No previous treatment with investigational medicinal treatment within 6 months prior to the start of the (pre)-screening for the study.
- No previous treatment with idebenone within 6 months prior to the start of the (pre)-screening for the study.
- Willing and able to adhere to the study visit schedule and other protocol requirements.
- Written informed consent signed (by parent(s)/legal guardian and/or the patient, according to the local regulations).
- Glucocorticosteroids use which is stable for at least 2 months prior first drug administration.
Exclusion Criteria
- Aberrant RNA splicing and/or aberrant response to PRO044, detected by in vitro PRO044 assay during pre-screening.
- Known presence of dystrophin in ≥ 5% of fibers in a pre-study diagnostic muscle biopsy.
- Severe muscle abnormalities defined as increased signal intensity in >50% of the tibialis anterior muscle at MRI.
- FEV1 and/or FVC < 60% of predicted.
- Current or history of liver or renal disease.
- Acute illness within 4 weeks prior to treatment (Day 1) which may interfere with the measurements.
- Severe mental retardation which in the opinion of the investigator prohibits participation in this study.
- Severe cardiac myopathy which in the opinion of the investigator prohibits participation in this study.
- Need for mechanical ventilation.
- Creatinine concentration above 1.5 times the upper limit of normal (age corrected).
- Serum ASAT and/or ALAT concentration(s) which suggest hepatic impairment.
- Use of anticoagulants, antithrombotics or antiplatelet agents.
- Use of idebenone.
- Use of any investigational product within 6 months prior to the start of the (pre)-screening for the study.
- Subject has donated blood less than 90 days before the start of the (pre)-screening for the study.
- Current or history of drug and/or alcohol abuse.
- Participation in another trial with an investigational product.
Data sourced from ClinicalTrials.gov (NCT01037309). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.