Pilot study of growth hormone to treat spinal muscular atrophy type II and III

Start Date
01/10/2007
Expected end Date
09/05/2021
Location
Germany
Status
Ongoing but has finished recruiting patients

What is the aim of the trial?

Personal observations and case reports suggest that patients with neuromuscular disorders who take growth hormone experience increased muscle force.
This Phase 2 trial therefore aims to determine whether growth hormone can increase strength in people with spinal muscular atrophy type 2 and 3, and to investigate the safety of the treatment.

Who can be involved in the trial?

This trial is for people with a genetically confirmed diagnosis of spinal muscular atrophy type II or III. Participants must be aged between 6 and 35 years, and be able to perform tests for measurements of muscle strength. For more details of who can participate in the trial, see ‘Further trial details’ below.

What happens during the trial?

Patients entered into the trial will be randomly assigned into one of two groups.

The first group will take a dose of the growth hormone ‘somatotropin’ every evening for 12 weeks, followed by 8 weeks during which they will take nothing, followed by another 12-week period in which they will take a dose of placebo every evening.

The second group will take a placebo every evening for 12 weeks, followed by 8 weeks during which they will take nothing, followed by another 12-week period in which they will take growth hormone every evening.

During the course of the study, participants will be regularly tested to determine their grip strength, muscle function and lung function. Their quality of life will also be assessed during the study by completing a questionnaire.

Where is the study taking place?

This trial is being carried out in seven different locations in Germany, including Freiburg, Berlin, Munich, Göttingen, Essen, Mainz and Kiel.

In most circumstances, for somebody to participate in a clinical trial, they need to live near the team of people who are conducting the research, because they need to be closely monitored.

How could the results of the trial benefit patients?

If this new drug is successful, it could help reduce the severity of the symptoms of spinal muscular atrophy.
If the results from the study are positive, further longer studies looking at the development of muscle function and quality of life will follow.

Contact Details:

Prof. Rudolf Korinthenberg

Tel: +49 (0)761 270 4314

Email: rudolf.korinthenberg@uniklinik-freiburg.de

Who is funding this study?

  • University Hospital Freiburg
  • Novo Nordisk

Official name of the Trial:

Pilot Study of Growth Hormone to Treat SMA Type II and III

Trial study number:

NCT00533221

Further trial details:

For further information on the trial and detailed inclusion and exclusion please click on the link below. Sometimes these details can be quite technical. If you have any questions please discuss this with your clinician or contact the clinical trial organisers.

http://clinicaltrials.gov/show/NCT00533221

Background information and related links:

Personal observations and case reports suggest that patients with neuromuscular disorders who take growth hormone experience increased muscle force.
This Phase 2 trial therefore aims to determine whether growth hormone can increase strength in people with spinal muscular atrophy type 2 and 3, and to investigate the safety of the treatment.

Keep in touch