Trial record 9 of 14 for:    "spinal muscular atrophy with respiratory distress" OR "Infantile Spinal Muscular Atrophy" OR "Spinal Muscular Atrophies of Childhood"

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

This study has been completed.
Sponsor:
Collaborator:
Novo Nordisk A/S
Information provided by (Responsible Party):
Janbernd Kirschner, University Hospital Freiburg
ClinicalTrials.gov Identifier:
NCT00533221
First received: September 12, 2007
Last updated: December 17, 2013
Last verified: December 2013
  Purpose

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
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, 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 University Hospital Freiburg:

Primary Outcome Measures:
  • Primary: sum of strength (hand held myometry) [ Time Frame: 20 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Functional (time) tests, lung function, quality of life, [ Time Frame: 20 weeks ] [ Designated as safety issue: No ]

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

  Eligibility

Ages Eligible for Study:   6 Years to 35 Years
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
  More Information

Additional Information:
Publications:
Responsible Party: Janbernd Kirschner, MD, University Hospital Freiburg
ClinicalTrials.gov Identifier: NCT00533221     History of Changes
Other Study ID Numbers: SMA-GH, 2005-002822-78
Study First Received: September 12, 2007
Last Updated: December 17, 2013
Health Authority: Germany: Federal Institute for Drugs and Medical Devices
Germany: Ethics Commission

Keywords provided by 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
Central Nervous System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Motor Neuron Disease
Nervous System Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Neuromuscular Diseases
Neuromuscular Manifestations
Pathological Conditions, Anatomical
Signs and Symptoms
Spinal Cord Diseases

ClinicalTrials.gov processed this record on October 21, 2014