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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT03992430
Other study ID # 4658-402
Secondary ID
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date July 13, 2020
Est. completion date November 30, 2024

Study information

Verified date April 2024
Source Sarepta Therapeutics, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Eteplirsen
Solution for intravenous (IV) infusion.

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Sponsors (1)

Lead Sponsor Collaborator
Sarepta Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Colombia,  Czechia,  Denmark,  France,  Germany,  Greece,  Hungary,  India,  Ireland,  Italy,  Jordan,  Korea, Republic of,  Mexico,  Netherlands,  New Zealand,  Norway,  Poland,  Romania,  Serbia,  Slovenia,  Spain,  Switzerland,  Taiwan,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Part 1: Incidence of Adverse Events (AEs) Up to Week 148
Primary Part 2: Change From Baseline in the NSAA Total Score at Week 144 Baseline, Week 144
Secondary Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test Baseline, Week 144
Secondary Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) Baseline, Week 144
Secondary Part 2: Change from Baseline in Forced Vital Capacity Percent Predicted (FVC%p) at Week 144 Baseline, Week 144
Secondary Part 2: Time to Loss of Ambulation (LOA) Baseline up to Week 144
Secondary Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression Baseline, Postdose (at Week 24, Week 48, or Week 144)
Secondary Part 2: Incidence of Adverse Events (AEs) Baseline up to Week 148
Secondary Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen 0 (predose) to 2 hours postdose up to Week 144
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