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Exploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.

Exploratory study on the local dystrophin production restoring effect and safety and tolerability of a single intramuscular dose of antisense oligoribonucleotide PRO051 in Duchenne muscular dystrophy patients.

Status
Active, not recruiting
Phases
Unknown
Study type
Interventional
Source
NL-OMON
Registry ID
NL-OMON21618
Enrollment
6
Registered
2006-06-22
Start date
2006-06-22
Completion date
Unknown
Last updated
2024-02-28

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy.

Interventions

Treatment with PRO-051.

Sponsors

ProSensa B.V. Wassenaarseweg 72 Leiden The Netherlands
Lead Sponsor

Eligibility

Inclusion criteria

Inclusion criteria: 1. Duchenne muscular dystrophy patients; 2. Age between 8 and 16 years; 3. Positive in vitro dystrophin RNA skip test in prior skin or muscle biopsy or in screening skin biopsy.

Exclusion criteria

Exclusion criteria: N/A

Design outcomes

Primary

MeasureTime frame
Pharmacodynamics (dystrophin restoring effect).—

Secondary

MeasureTime frame
Safety and tolerability.—

Contacts

Public ContactG. Platenburg

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)