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

NCT ID: NCT00533221

Last Updated: 2013-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-10-31

Study Completion Date

2011-08-31

Brief Summary

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

Detailed Description

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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

Conditions

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Muscular Atrophy, Spinal

Keywords

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growth hormone strength Spinal muscular atrophy type II and III

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Somatotropin

subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application

Group Type ACTIVE_COMPARATOR

somatotropin

Intervention Type 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

Placebo

12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type 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

Interventions

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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

Intervention Type 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

Intervention Type DRUG

Other Intervention Names

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Penfill® Penfill®

Eligibility Criteria

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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
Minimum Eligible Age

6 Years

Maximum Eligible Age

35 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novo Nordisk A/S

INDUSTRY

Sponsor Role collaborator

University Hospital Freiburg

OTHER

Sponsor Role lead

Responsible Party

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Janbernd Kirschner

MD

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Rudolf Korinthenberg, Professor

Role: PRINCIPAL_INVESTIGATOR

University medical centre Freiburg, children's hospital

Locations

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von Haunersches Kinderspital

Munich, Bavaria, Germany

Site Status

University Children'S Hospital

Göttingen, Lower Saxony, Germany

Site Status

University Children'S Hospital

Essen, North Rhine-Westphalia, Germany

Site Status

University Children'S Hospital

Mainz, Rhineland-Palatinate, Germany

Site Status

University Children'S Hospital

Kiel, Schleswig-Holstein, Germany

Site Status

Charité, Department of Neurpaediatrics

Berlin, State of Berlin, Germany

Site Status

Countries

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Germany

References

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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.

Reference Type RESULT
PMID: 24300782 (View on PubMed)

Related Links

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http://www.uniklinik-freiburg.de/kinderklinik

University Children's Hospital Freiburg, Germany

Other Identifiers

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2005-002822-78

Identifier Type: -

Identifier Source: secondary_id

SMA-GH

Identifier Type: -

Identifier Source: org_study_id