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

University Hospital Freiburg20 enrolled

Overview

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

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

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Muscular Atrophy, Spinal

Interventions

somatotropinDRUG

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

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 6 YearsMax age: 35 Years

Medical Language ↔ Plain English

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

Locations (6)

von Haunersches Kinderspital

Munich, Bavaria, Germany

University Children'S Hospital

Göttingen, Lower Saxony, Germany

University Children'S Hospital

Essen, North Rhine-Westphalia, Germany

University Children'S Hospital

Mainz, Rhineland-Palatinate, Germany

Outcomes

Primary Outcomes

Primary: sum of strength (hand held myometry)

Time frame: 20 weeks

Secondary Outcomes

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

Time frame: 20 weeks

Data from ClinicalTrials.gov

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