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

This study has been completed.
Novo Nordisk A/S
Information provided by (Responsible Party):
Janbernd Kirschner, University Hospital Freiburg Identifier:
First received: September 12, 2007
Last updated: December 17, 2013
Last verified: December 2013
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.

Condition Intervention Phase
Muscular Atrophy, Spinal Drug: somatotropin Drug: Placebo Phase 2

Study Type: Interventional
Study Design: 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?

Resource links provided by NLM:

Further study details as provided by Janbernd Kirschner, University Hospital Freiburg:

Primary Outcome Measures:
  • Primary: sum of strength (hand held myometry) [ Time Frame: 20 weeks ]

Secondary Outcome Measures:
  • Functional (time) tests, lung function, quality of life, [ Time Frame: 20 weeks ]

Enrollment: 20
Study Start Date: October 2007
Study Completion Date: August 2011
Primary Completion Date: February 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: 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 Name: Penfill®

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

Ages Eligible for Study:   6 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts 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
  Contacts and Locations
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Please refer to this study by its identifier: NCT00533221

von Haunersches Kinderspital
Munich, Bavaria, Germany, 80337
University Children'S Hospital
Göttingen, Niedersachsen, Germany, 37075
University Children'S Hospital
Essen, Nordrhein-Westfalen, Germany, 45122
University Children'S Hospital
Mainz, Rheinland Pfalz, Germany, 55110
University Children'S Hospital
Kiel, Schleswig-Holstein, Germany, 24105
Charité, Department of Neurpaediatrics
Berlin, Germany, 13353
Sponsors and Collaborators
University Hospital Freiburg
Novo Nordisk A/S
Principal Investigator: Rudolf Korinthenberg, Professor University medical centre Freiburg, children's hospital
  More Information

Additional Information:
Responsible Party: Janbernd Kirschner, MD, University Hospital Freiburg Identifier: NCT00533221     History of Changes
Other Study ID Numbers: SMA-GH
Study First Received: September 12, 2007
Last Updated: December 17, 2013

Keywords provided by Janbernd Kirschner, University Hospital Freiburg:
growth hormone
Spinal muscular atrophy type II and III

Additional relevant MeSH terms:
Spinal Muscular Atrophies of Childhood
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn processed this record on September 20, 2017