Pilot Study of Growth Hormon to Treat SMA Typ II and III

Study Overview
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Brief Summary
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The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.

Detailed Description
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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

Official Title
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Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?

Conditions
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Muscular Atrophy, Spinal

Intervention / Treatment
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* Drug: somatotropin
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years
Maximum Age: 35 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Somatotropin<br>subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application | Drug: somatotropin<br>* 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<br>* Other names: Penfill®;|
| Placebo Comparator: Placebo<br>12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin | Drug: Placebo<br>* 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<br>* Other names: Penfill®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Primary: sum of strength (hand held myometry) |  | 20 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Functional (time) tests, lung function, quality of life, |  | 20 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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growth hormone, strength, Spinal muscular atrophy type II and III