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

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ClinicalTrials.gov Identifier: NCT00533221
Recruitment Status : Completed
First Posted : September 21, 2007
Last Update Posted : December 18, 2013
Sponsor:
Collaborator:
Novo Nordisk A/S
Information provided by (Responsible Party):
Janbernd Kirschner, 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.

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 Type : Interventional  (Clinical Trial)
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 : October 2007
Actual Primary Completion Date : February 2010
Actual Study Completion Date : August 2011


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 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®



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

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


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Ages Eligible for Study:   6 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00533221


Locations
Germany
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
Investigators
Principal Investigator: Rudolf Korinthenberg, Professor University medical centre Freiburg, children's hospital

Additional Information:
Publications of Results:
Responsible Party: Janbernd Kirschner, MD, University Hospital Freiburg
ClinicalTrials.gov Identifier: NCT00533221     History of Changes
Other Study ID Numbers: SMA-GH
2005-002822-78
First Posted: September 21, 2007    Key Record Dates
Last Update Posted: December 18, 2013
Last Verified: December 2013

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

Additional relevant MeSH terms:
Spinal Muscular Atrophies of Childhood
Atrophy
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