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Completed

NCT Number: NCT00533221

Pilot Study of Growth Hormon to Treat SMA Typ 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.

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Key information

Conditions

Muscular Atrophy, Spinal Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases Spinal Muscular Atrophies of Childhood

Age range

6 year–35 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

von Haunersches Kinderspital, Munich, Bavaria, Germany

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Somatotropin

Drug

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

Drug

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®

Primary outcomes

  1. Primary: sum of strength (hand held myometry)

    Time frame: 20 weeks

Secondary outcomes

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

    Time frame: 20 weeks

Sponsors and collaborators

Lead sponsor

University Hospital Freiburg

Other

Collaborators

  • Novo Nordisk A/S

Registry information

Official study title

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

Important dates

Study start
2007
Primary completion
2010
Study completion
2011
First posted
Sep 21, 2007
Registry last updated
Dec 18, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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