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

• Register: EUCTR
• Last refreshed on: 12 February 2018
• Main ID: EUCTR2016-000951-29-IT
• Date of registration: 07/02/2018
• Prospective Registration: No
• Primary sponsor: SAREPTA THERAPEUTICS, INC.
• Public title: A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients
• Scientific title: An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping - N/A
• Date of first enrolment: 13/06/2017
• Target sample size: 12
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-000951-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: no; Single blind: no; Double blind: no; Parallel group: Cross over: Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, France, Germany, Italy, United Kingdom

Contacts

• Name: Clinical Trial Information
• Affiliation: Voisin Consulting

• Name: Clinical Trial Information
• Affiliation: Voisin Consulting

Key inclusion & exclusion criteria

Inclusion criteria:
1. Be a male between 6 months to 48 months of age, inclusive.
2. Have an established clinical diagnosis of DMD with a deletion mutation amenable to exon 51 skipping (e.g., deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, 52-63).
3. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study requirements and is 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: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Has received any pharmacologic treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing at Week 1 (e.g., growth hormone, anabolic steroids).
2. Has received previous or current treatment with any experimental treatment. Prior drisapersen therapy is permitted if a patient has not received drisapersen for 6 months prior to the Week 1 dose.
3. Has a clinically significant illness other than DMD, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioural disease, or malignancy likely to impair the patient's ability to participate in this study.
4. Has a clinically significant laboratory abnormality that is either not expected or is of a greater severity than what is expected in DMD patients.
5. Has any other condition that, in the Investigator's opinion, could interfere with the patient's participation in the study.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Intervention(s)

Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eteplirsen
CAS Number: 1173755-55-9
Current Sponsor code: AVI-4658
Other descriptive name: ETEPLIRSEN
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 50-

Primary Outcome(s)

Main Objective: To evaluate the safety and tolerability of eteplirsen administered once weekly by intravenous (IV) infusion in male Duchenne muscular dystrophy (DMD) patients ages 6 months to 48 months, inclusive

Timepoint(s) of evaluation of this end point: Incidence of AEs: continuous
Clinical safety laboratory sample: Screening, Weeks 2, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 and End-of-Study Follow-up Visit
Vital signs: Screening, baseline, on weekly infusion days, Week 96 and End-of-Study Follow-up Visit
Physical examination: Screening, baseline, Week 4, 8, 12, 16, 20-48, 60, 72, 84, 96 and End-of-Study Follow-up Visit
ECG: Screening, Weeks 8, 12, 24, 48, 60, 72, 84, 96
ECHO: Screening, Weeks 24, 48, 72, 96

Secondary Objective: To determine the pharmacokinetics (PK) of eteplirsen at the 2-, 10-, 20- and 30-mg/kg dose levels, administered once weekly by IV infusion in male DMD patients ages 6 months to 48 months, inclusive

Primary end point(s): Safety and tolerability of eteplirsen as measured by:
• Incidence of adverse events (AEs)
• Abnormal changes from baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis)
• Abnormal changes from baseline or worsening of vital signs
• Abnormal changes from baseline or worsening of physical examination findings
• Abnormal changes from baseline or clinically significant worsening of ECGs and ECHOs

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: PK plasma and urine sampling: Weeks 2, 6, 8, 10, 24

Secondary end point(s): PK of eteplirsen by population PK methods, including assessment of the following PK parameters (if evaluable):
• Maximum plasma concentration (Cmax)
• Time of Cmax (Tmax)
• AUC
• Apparent volume of distribution at steady state (Vss)
• Clearance (CL)
• Elimination half-life (t½)
• Amount of drug eliminated in urine (Ae%)

Secondary ID(s)

2016-000951-29-BE

4658-102

Source(s) of Monetary Support

Sarepta Therapeutics, Inc.