A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy

• Register: EUCTR
• Last refreshed on: 5 August 2013
• Main ID: EUCTR2009-013762-63-NL
• Date of registration: 25/08/2009
• Prospective Registration: Yes
• Primary sponsor: Prosensa Therapeutics B.V
• Public title: A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy
• Scientific title: A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy
• Date of first enrolment: 16/04/2010
• Target sample size: 18
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-013762-63
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:

Countries of recruitment

Belgium, Italy, Netherlands, Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
1.Age at least 5 and not older than 16 years on the day of first drug administration
2.Duchenne muscular dystrophy resulting from a mutation correctable by treatment with PRO044
3.Life expectancy at least 6 months after inclusion in the trial


Are the trial subjects under 18? yes
Number of subjects for this age range:
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. Ventilator dependency
2. Previous treatment with investigational medicinal treatment within 6 months prior to the start of the pre-screening for the study
3. Previous treatment with idebenone within 6 months prior to the start of the pre-screening for the study.



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

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Product Name: PRO044
Product Code: PRO044
Pharmaceutical Form: Solution for injection
INN or Proposed INN: h44AON188
Current Sponsor code: PRO044
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 92-

Primary Outcome(s)

Primary end point(s): This is an exploratory study. The primary endpoints are safety and tolerability measures:
- Adverse events
- Local tolerability
- Safety biochemistry parameters
- Safety hematology parameters
- Coagulation (aPTT)
- Cystatin C
- Urinalysis (dipstick, quantitative protein and creatinine, and creatinine, and a-1-microglobulin)
- Complement split products (C3a, SC5b-9, Bb)
- Cytokines (IL-6, TNF-a) and chemokine (MCP-1)
- Antibodies to dystrophin
- ECG parameters

Secondary endpoints (also exploratory) relating to efficacy are:
- Presence of (BMD like) dystrophin expression after treatment (in muscle biopsy)
- Production of exon skip 44 mRNA (in muscle biopsy and mononuclear blood cells)
- Muscle function (timed tests and 6-minutes walk test)
- Muscle strength (handheld myometry and spirometry)

Main Objective: To assess the safety and tolerability of PRO044 at different dose levels in patients with Duchenne muscular dystrophy

Secondary Objective: To assess the effect of PRO044 at different dose levels in patients with Duchenne muscular dystrophy
To determine the pharmacokinetics of PRO044 at different dose levels after subcutaneous administration in patients with Duchenne muscular dystrophy.

Secondary ID(s)

PRO044-CLIN-01