Status:
TERMINATED
Proteasomal Inhibition for Patients With Mis-sense Mutated Dysferlin
Lead Sponsor:
University Hospital, Basel, Switzerland
Conditions:
Dysferlinopathy
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief Summary
Dysferlin is a protein with an important role in the repair of muscle surface membranes. Mutations in dysferlin cause different forms of muscular dystrophies. Dysferlinopathies are inherited in an aut...
Eligibility Criteria
Inclusion
- must carry at least one allele of a salvageable mis-sense mutation of dysferlin
- Age ≥ 18 years
- Written informed consent
Exclusion
- Bleeding disorder
- Acute or chronic kidney failure (CCL \<50 ml/min)
- Advanced liver disease or active hepatitis
- Congestive heart failure NYHA III and IV
- Pregnancy or nursing
- Immunosuppression (prednisolone doses below 20 mg/d are allowed)
- Therapy with strong inhibitors of cytochrome P450 3A4
- HCV or HIV infection
- Regular alcohol consumption (\>14 drinks a week)
- Drug addiction
Key Trial Info
Start Date :
December 1 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 15 2017
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT01863004
Start Date
December 1 2012
End Date
September 15 2017
Last Update
September 21 2017
Active Locations (1)
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1
Neuromuskuläres Zentrum, Universitätsspital Basel
Basel, Switzerland, 4031