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

Primary Purpose

Muscular Atrophy, Spinal

Status
Completed
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
somatotropin
Placebo
Sponsored by
University Hospital Freiburg
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Atrophy, Spinal focused on measuring growth hormone, strength, Spinal muscular atrophy type II and III

Eligibility Criteria

6 Years - 35 Years (Child, Adult)All SexesDoes not accept 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

Sites / Locations

  • von Haunersches Kinderspital
  • University Children'S Hospital
  • University Children'S Hospital
  • University Children'S Hospital
  • University Children'S Hospital
  • Charité, Department of Neurpaediatrics

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Somatotropin

Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

Primary: sum of strength (hand held myometry)

Secondary Outcome Measures

Functional (time) tests, lung function, quality of life,

Full Information

First Posted
September 12, 2007
Last Updated
December 17, 2013
Sponsor
University Hospital Freiburg
Collaborators
Novo Nordisk A/S
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1. Study Identification

Unique Protocol Identification Number
NCT00533221
Brief Title
Pilot Study of Growth Hormon to Treat SMA Typ II and III
Official Title
Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?
Study Type
Interventional

2. Study Status

Record Verification Date
December 2013
Overall Recruitment Status
Completed
Study Start Date
October 2007 (undefined)
Primary Completion Date
February 2010 (Actual)
Study Completion Date
August 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University Hospital Freiburg
Collaborators
Novo Nordisk A/S

4. Oversight

Data Monitoring Committee
Yes

5. 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.
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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Atrophy, Spinal
Keywords
growth hormone, strength, Spinal muscular atrophy type II and III

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Somatotropin
Arm Type
Active Comparator
Arm Description
subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin
Intervention Type
Drug
Intervention Name(s)
somatotropin
Other Intervention Name(s)
Penfill®
Intervention Description
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
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Penfill®
Intervention Description
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
Primary Outcome Measure Information:
Title
Primary: sum of strength (hand held myometry)
Time Frame
20 weeks
Secondary Outcome Measure Information:
Title
Functional (time) tests, lung function, quality of life,
Time Frame
20 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
35 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rudolf Korinthenberg, Professor
Organizational Affiliation
University medical centre Freiburg, children's hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
von Haunersches Kinderspital
City
Munich
State/Province
Bavaria
ZIP/Postal Code
80337
Country
Germany
Facility Name
University Children'S Hospital
City
Göttingen
State/Province
Niedersachsen
ZIP/Postal Code
37075
Country
Germany
Facility Name
University Children'S Hospital
City
Essen
State/Province
Nordrhein-Westfalen
ZIP/Postal Code
45122
Country
Germany
Facility Name
University Children'S Hospital
City
Mainz
State/Province
Rheinland Pfalz
ZIP/Postal Code
55110
Country
Germany
Facility Name
University Children'S Hospital
City
Kiel
State/Province
Schleswig-Holstein
ZIP/Postal Code
24105
Country
Germany
Facility Name
Charité, Department of Neurpaediatrics
City
Berlin
ZIP/Postal Code
13353
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
24300782
Citation
Kirschner J, Schorling D, Hauschke D, Rensing-Zimmermann C, Wein U, Grieben U, Schottmann G, Schara U, Konrad K, Muller-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.
Results Reference
result
Links:
URL
http://www.uniklinik-freiburg.de/kinderklinik
Description
University Children's Hospital Freiburg, Germany

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

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