A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 24

Inclusion Criteria

A patient must meet all of the following criteria to be eligible to participate in this study:
1. Is male.
2. Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
3. Is 7 to 21 years of age, inclusive.
4. Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.
5. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a highly effective form of contraceptive during this timeframe.
6. Is willing to provide informed consent or informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 4
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

A patient who meets any of the following criteria will be excluded from this study:
1. Has a LVEF < 40.0% based on an ECHO performed within 12 weeks prior to Screening or at the Screening visit.
2. Has a QT interval corrected with Fridericia’s formula = 450 msec on the Screening ECG.
3. Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, ß-blockers, or potassium.
4. Requires anti-arrhythmic and/or diuretic therapy for heart failure.
5. Has a FVC < 40.0% of predicted value (according to the American Thoracic Society/European Respiratory Society criteria) within 12 weeks prior to Screening or at Screening.
6. Has a current infection, or history of an infection within 12 weeks prior to Screening requiring treatment with an antibiotic.
7. Has a known kidney disease or had an acute kidney injury within 24 weeks prior to Screening.
8. Use of any herbal medication/supplement containing aristolochic acid.
9. Treatment with any exon 51-skipping therapy within 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.
10. Participation in an interventional clinical trial within 12 weeks prior to Screening.
11. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Screening.
12. Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
13. Major surgery within 12 weeks prior to Screening, or planned surgery or procedures that would interfere with the conduct of the study.
14. Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy.
15. Any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the trial.
16. Inability to comply with the study protocol.
17. Is an employee of the Investigator or study center, with direct involvement in the proposed study or other studies under the direction of that Investigator or study center, as well as family members of the employees or the Investigator.
18. Any patient who is taking medications that increase the risk of bleeding, in the Investigator's opinion (eg, anticoagulants, antiplatelet agents, novel oral anticoagulants, selective norepinephrine reuptake inhibitors, etc.)
19. Platelet count < 150 × 103/µL.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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