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

Muscular Atrophy, Spinal Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00533221
• Other study ID #: SMA-GH
• Secondary ID: 2005-002822-78
• Status: Completed
• Phase: Phase 2
• First received: September 12, 2007
• Last updated: December 17, 2013
• Start date: October 2007
• Est. completion date: August 2011

Study information

• Verified date: December 2013
• Source: University Hospital Freiburg
• Contact: n/a
• Is FDA regulated: No
• Health authority: Germany: Federal Institute for Drugs and Medical DevicesGermany: Ethics Commission
• Study type: Interventional

Clinical Trial Summary

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

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: August 2011
• Est. primary completion date: February 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 35 Years

Eligibility

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

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal

Intervention

Drug:
• somatotropin
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
• Placebo
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

Locations

Country	Name	City	State
Germany	Charité, Department of Neurpaediatrics	Berlin
Germany	University Children'S Hospital	Essen	Nordrhein-Westfalen
Germany	University Children'S Hospital	Göttingen	Niedersachsen
Germany	University Children'S Hospital	Kiel	Schleswig-Holstein
Germany	University Children'S Hospital	Mainz	Rheinland Pfalz
Germany	von Haunersches Kinderspital	Munich	Bavaria

Sponsors (2)

Lead Sponsor	Collaborator
University Hospital Freiburg	Novo Nordisk A/S

Country where clinical trial is conducted

Germany, 

References & Publications (1)

Kirschner J, Schorling D, Hauschke D, Rensing-Zimmermann C, Wein U, Grieben U, Schottmann G, Schara U, Konrad K, Müller-Felber W, Thiele S, Wilichowski E, Hobbiebrunken E, Stettner GM, Korinthenberg R. Somatropin treatment of spinal muscular atrophy: a pl — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Primary: sum of strength (hand held myometry)		20 weeks	No
Secondary	Functional (time) tests, lung function, quality of life,		20 weeks	No

External Links

•   University Children's Hospital Freiburg, Germany