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

Sponsor

University Hospital Freiburg (Other)

Overall Status

Completed

CT.gov ID

NCT00533221

Collaborator

Novo Nordisk A/S (Industry)

Enrollment

Locations

Arms

Duration (Months)

3.3

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Muscular Atrophy, Spinal	Drug: somatotropin Drug: Placebo	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

20 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Oct 1, 2007

Actual Primary Completion Date :

Feb 1, 2010

Actual Study Completion Date :

Aug 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Somatotropin subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application	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 Other Names: Penfill®
Placebo Comparator: Placebo 12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin	Drug: 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 Other Names: Penfill®

Arm

Intervention/Treatment

Active Comparator: Somatotropin

subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application

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

Other Names:

Penfill®

Placebo Comparator: Placebo

12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin

Drug: 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

Other Names:

Penfill®

Outcome Measures

Primary Outcome Measures

Primary: sum of strength (hand held myometry) [20 weeks]

Secondary Outcome Measures

Functional (time) tests, lung function, quality of life, [20 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 35 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	von Haunersches Kinderspital	Munich	Bavaria	Germany	80337
2	University Children'S Hospital	Göttingen	Niedersachsen	Germany	37075
3	University Children'S Hospital	Essen	Nordrhein-Westfalen	Germany	45122
4	University Children'S Hospital	Mainz	Rheinland Pfalz	Germany	55110
5	University Children'S Hospital	Kiel	Schleswig-Holstein	Germany	24105
6	Charité, Department of Neurpaediatrics	Berlin		Germany	13353