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

Phase 2

Completed

• Conditions: Muscular Atrophy, Spinal
• Interventions: Drug: somatotropin; Drug: Placebo

• Registration Number: NCT00533221
• Lead Sponsor: University Hospital Freiburg

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

Study Timeline

• Study First Submitted: 2007-09-12
• Study First Submitted QC: 2007-09-20
• Study First Posted: 2007-09-21
• Study Start: 2007-10
• Primary Completion: 2010-02
• Study Completion: 2011-08
• Status Verified: 2013-12
• Last Update Submitted: 2013-12-17
• Last Update Posted: 2013-12-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 20

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Somatotropin	somatotropin	subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application
Placebo	Placebo	12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin

Primary Outcome Measures

Name	Time	Method
Primary: sum of strength (hand held myometry)	20 weeks

Secondary Outcome Measures

Name	Time	Method
Functional (time) tests, lung function, quality of life,	20 weeks

Trial Locations

Locations (3)

Charité, Department of Neurpaediatrics; 🇩🇪 Berlin, Germany

von Haunersches Kinderspital; 🇩🇪 Munich, Bavaria, Germany

University Children'S Hospital; 🇩🇪 Kiel, Schleswig-Holstein, Germany