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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

Tracking Information
First Submitted Date  ICMJE September 12, 2007
First Posted Date  ICMJE September 21, 2007
Last Update Posted Date December 18, 2013
Study Start Date  ICMJE October 2007
Actual Primary Completion Date February 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 20, 2007)
Primary: sum of strength (hand held myometry) [ Time Frame: 20 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 20, 2007)
Functional (time) tests, lung function, quality of life, [ Time Frame: 20 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pilot Study of Growth Hormon to Treat SMA Typ II and III
Official Title  ICMJE Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?
Brief Summary The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.
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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Muscular Atrophy, Spinal
Intervention  ICMJE
  • 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®
  • 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®
Study Arms  ICMJE
  • Active Comparator: Somatotropin
    subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application
    Intervention: Drug: somatotropin
  • Placebo Comparator: Placebo
    12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin
    Intervention: Drug: Placebo
Publications * 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 placebo-controlled, double-blind crossover pilot study. Neuromuscul Disord. 2014 Feb;24(2):134-42. doi: 10.1016/j.nmd.2013.10.011. Epub 2013 Nov 13.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: September 20, 2007)
20
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE August 2011
Actual Primary Completion Date February 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 35 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00533221
Other Study ID Numbers  ICMJE SMA-GH
2005-002822-78
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Janbernd Kirschner, University Hospital Freiburg
Study Sponsor  ICMJE University Hospital Freiburg
Collaborators  ICMJE Novo Nordisk A/S
Investigators  ICMJE
Principal Investigator: Rudolf Korinthenberg, Professor University medical centre Freiburg, children's hospital
PRS Account University Hospital Freiburg
Verification Date December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP