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

December 17, 2013 updated by: Janbernd Kirschner, University Hospital Freiburg

Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?

The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Berlin, Germany, 13353
        • Charité, Department of Neurpaediatrics
    • Bavaria
      • Munich, Bavaria, Germany, 80337
        • von Haunersches Kinderspital
    • Niedersachsen
      • Göttingen, Niedersachsen, Germany, 37075
        • University Children's Hospital
    • Nordrhein-Westfalen
      • Essen, Nordrhein-Westfalen, Germany, 45122
        • University Children's Hospital
    • Rheinland Pfalz
      • Mainz, Rheinland Pfalz, Germany, 55110
        • University Children's Hospital
    • Schleswig-Holstein
      • Kiel, Schleswig-Holstein, Germany, 24105
        • University Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 35 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / 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 Names:
  • 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 Names:
  • Penfill®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Primary: sum of strength (hand held myometry)
Time Frame: 20 weeks
20 weeks

Secondary Outcome Measures

Outcome Measure
Time Frame
Functional (time) tests, lung function, quality of life,
Time Frame: 20 weeks
20 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital Freiburg

Collaborators

Novo Nordisk A/S

Investigators

  • Principal Investigator: Rudolf Korinthenberg, Professor, University medical centre Freiburg, children's hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2007

Primary Completion (Actual)

February 1, 2010

Study Completion (Actual)

August 1, 2011

Study Registration Dates

First Submitted

September 12, 2007

First Submitted That Met QC Criteria

September 20, 2007

First Posted (Estimate)

September 21, 2007

Study Record Updates

Last Update Posted (Estimate)

December 18, 2013

Last Update Submitted That Met QC Criteria

December 17, 2013

Last Verified

December 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SMA-GH
  • 2005-002822-78

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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