NCT00533221

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2007

Typical duration for phase_2

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 12, 2007

Completed
9 days until next milestone

First Posted

Study publicly available on registry

September 21, 2007

Completed
10 days until next milestone

Study Start

First participant enrolled

October 1, 2007

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2010

Completed
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2011

Completed
Last Updated

December 18, 2013

Status Verified

December 1, 2013

Enrollment Period

2.3 years

First QC Date

September 12, 2007

Last Update Submit

December 17, 2013

Conditions

Muscular Atrophy, Spinal

Keywords

growth hormonestrengthSpinal muscular atrophy type II and III

Outcome Measures

Primary Outcomes (1)

  • Primary: sum of strength (hand held myometry)

    20 weeks

Secondary Outcomes (1)

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

    20 weeks

Study Arms (2)

Somatotropin

ACTIVE COMPARATOR

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

Drug: somatotropin

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

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

Also known as: Penfill®
Somatotropin
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

Also known as: Penfill®
Placebo

Eligibility Criteria

Age6 Years - 35 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

von Haunersches Kinderspital

Munich, Bavaria, 80337, Germany

Location

University Children'S Hospital

Göttingen, Lower Saxony, 37075, Germany

Location

University Children'S Hospital

Essen, North Rhine-Westphalia, 45122, Germany

Location

University Children'S Hospital

Mainz, Rhineland-Palatinate, 55110, Germany

Location

University Children'S Hospital

Kiel, Schleswig-Holstein, 24105, Germany

Location

Charité, Department of Neurpaediatrics

Berlin, State of Berlin, 13353, Germany

Location

Related Publications (1)

  • 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.

    PMID: 24300782RESULT

Related Links

MeSH Terms

Conditions

Muscular Atrophy, SpinalSpinal Muscular Atrophies of Childhood

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular DiseasesHeredodegenerative Disorders, Nervous SystemGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Pituitary Hormones, AnteriorPituitary HormonesPeptide HormonesHormonesHormones, Hormone Substitutes, and Hormone AntagonistsPeptidesAmino Acids, Peptides, and Proteins

Study Officials

  • Rudolf Korinthenberg, Professor

    University medical centre Freiburg, children's hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

September 12, 2007

First Posted

September 21, 2007

Study Start

October 1, 2007

Primary Completion

February 1, 2010

Study Completion

August 1, 2011

Last Updated

December 18, 2013

Record last verified: 2013-12

Locations

DE(6)