Muscular Atrophy, SpinalPhase 2NCT00533221
Pilot Study of Growth Hormon to Treat SMA Typ II and III
A Phase 2 study testing somatotropin in people with Muscular Atrophy, Spinal, run by University Hospital Freiburg at 6 sites in Germany.
Trial summary
The purpose of this study is to determine whether Growth hormon can increase strength in spinal muscular atrophy type II and III. Read more about this study
- Who is it for?
- Ages 6 to 35 years
- Conditions the study is for
- Muscular Atrophy, Spinal
- What is being tested?
- somatotropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 6 sitesGermany
- Study dates
- Oct 2007to Aug 2011
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
Germany6 sites
Charité, Department of Neurpaediatrics
Berlin, State of Berlin, 13353
University Children'S Hospital
Essen, North Rhine-Westphalia, 45122
University Children'S Hospital
Göttingen, Lower Saxony, 37075
University Children'S Hospital
Kiel, Schleswig-Holstein, 24105
University Children'S Hospital
Mainz, Rhineland-Palatinate, 55110
von Haunersches Kinderspital
Munich, Bavaria, 80337
About this study
Official title: Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type 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.
Read the 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
- Conditions
- Muscular Atrophy, Spinal
- Being tested
- somatotropin, Placebo
- Keywords
- growth hormone, strength, Spinal muscular atrophy type II and III
- Medical subject headings
- Muscular Atrophy, Spinal, Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
somatotropinDrug
Also called Penfill®
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
Also called Penfill®
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
| Study group | Role | What they receive |
|---|---|---|
| Somatotropin | Active comparator | subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application Receives: Drug: somatotropin |
| Placebo | Placebo comparator | 12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary: sum of strength (hand held myometry)
Measured: 20 weeks
Secondary outcomes (1)
Functional (time) tests, lung function, quality of life,
Measured: 20 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2007 (estimated)
- Study startOct 2007
- Primary completionFeb 2010
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Freiburg
- Sponsor type
- University, hospital or other
- Collaborators
- Novo Nordisk A/S
- Responsible party
- Principal investigator, Janbernd Kirschner, University Hospital Freiburg
- Investigators
- Rudolf Korinthenberg, Professor, Principal investigator, University medical centre Freiburg, children's hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00533221
- Sponsor's protocol ID
- SMA-GH
- Other IDs
- 2005-002822-78, 2005
- Status last verified
- Dec 2013
- First submitted
- 12 Sep 2007
- Last updated
- 18 Dec 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 24300782
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Can Treatment With Human Growth Hormone Increase Strength in Spinal Muscular Atrophy Type II and III?
- ClinicalTrials.gov ID
- NCT00533221
- Acronym / sponsor ID
- SMA-GH
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- growth hormone, strength, Spinal muscular atrophy type II and III
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University Hospital FreiburgUniversity, hospital or other
- Collaborators
- Novo Nordisk A/S
- Study start
- Oct 2007
- Primary completion
- Feb 2010 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 21 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Somatotropin | Active comparator | Drug: somatotropinsubcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application |
| Placebo | Placebo comparator | Drug: Placebo12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin |
Who may qualify
- Ages
- 6 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| somatotropinAlso: Penfill®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 | Drug | Somatotropin |
| PlaceboAlso: Penfill®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 | Drug | Placebo |
Primary outcome
Primary: sum of strength (hand held myometry)
Time frame: 20 weeks
1 secondary outcome
Functional (time) tests, lung function, quality of life,
Time frame: 20 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2013.
- Results publication: PubMed 24300782Kirschner 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.
- University Children's Hospital Freiburg, GermanyLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rudolf Korinthenberg, Professor principal investigator
University medical centre Freiburg, children's hospital
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing somatotropin in people with Muscular Atrophy, Spinal, run by University Hospital Freiburg at 6 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Dec 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary: sum of strength (hand held myometry) (time frame: 20 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00533221, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2013.