Clinical trial NCT00533221: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Muscular Atrophy, Spinal
Keywords: growth hormone
strength
Spinal muscular atrophy type II and III
Lead sponsor: University Hospital Freiburg
Sponsor class: OTHER
Collaborators: name: Novo Nordisk A/S
First posted: 2007-09-21
Enrollment: 20
Sex: ALL
Minimum age (years): 6
Maximum age (years): 35
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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

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

Primary outcomes:
measure: Primary: sum of strength (hand held myometry)
timeFrame: 20 weeks
Secondary outcomes:
measure: Functional (time) tests, lung function, quality of life,
timeFrame: 20 weeks
Other outcomes:
Locations:
city: Munich
country: Germany
facility: von Haunersches Kinderspital
state: Bavaria
city: Göttingen
country: Germany
facility: University Children'S Hospital
state: Niedersachsen
city: Essen
country: Germany
facility: University Children'S Hospital
state: Nordrhein-Westfalen
city: Mainz
country: Germany
facility: University Children'S Hospital
state: Rheinland Pfalz
city: Kiel
country: Germany
facility: University Children'S Hospital
state: Schleswig-Holstein
city: Berlin
country: Germany
facility: Charité, Department of Neurpaediatrics
Study officials:
affiliation: University medical centre Freiburg, children's hospital
name: Rudolf Korinthenberg, Professor
role: PRINCIPAL_INVESTIGATOR
References:
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.