Muscular Atrophy, Spinal
Conditions
Keywords
growth hormone, strength, Spinal muscular atrophy type II and III
Brief summary
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.
Detailed description
This pilot study is planned as a placebo-controlled cross-over trial in a limited number of patients. It is the aim to investigate the effect of GH on the short-term changes of strength and to investigate the tolerability of the treatment. If the results of the study are positive, further studies of longer duration addressing the development of motor function and quality of life could follow
Interventions
DRUGsomatotropin
0,015 mg/kg/d as one dose s.c. in the evening over one week followed by a 11 week period with 0,03 mg/kg/d s.c. as one dose in the evening
DRUGPlacebo
0,015 mg/kg/d as one dose s.c. in the evening over one week followed by a 11 week period with 0,03 mg/kg/d s.c. as one dose in the evening
Sponsors
Novo Nordisk A/S
University Hospital Freiburg
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
6 Years to 35 Years
Healthy volunteers
No
Inclusion criteria
* genetically confirmed diagnosis of Spinal Muscular Disease * Spinal muscular atrophy type II or III * age between 6 years and 35 years * ability to perform the tests for measurement of muscle strength (handheld myometry) * informed consent of the patient and/or parents
Exclusion criteria
* pregnancy or lactation * woman with child bearing potential without contraception * overweight or BMI over 30 kg/m² * Treatment with other drugs, that can influence strength 8 weeks before participation in the study or during participation * medical history or evidence of a malignant or cerebral tumor * cardiovascular, intestinal, endocrinologically or airway disease * Hypertension * growth hormone deficiency * hypersensitivity to one component part of the study medication * participation on a clinical trial during the study or 3 month before * abuse to drugs or alcohol * patient incapable of contracting or not able to understand the character, meaning and consequences of the clinical trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary: sum of strength (hand held myometry) | 20 weeks |
Secondary
| Measure | Time frame |
|---|---|
| Functional (time) tests, lung function, quality of life, | 20 weeks |
Countries
Germany
Outcome results
None listed