Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2005-002822-78 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Novo Nordisk A/S | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Somatotropin | Active Comparator | subcutaneous application of somatotropin over 12 weeks followed by 8 weeks wash out period followed by 12 weeks subcutaneous placebo application |
|
| Placebo | Placebo Comparator | 12 weeks placebo subcutaneous application followed by 8 weeks wash out and 12 weeks subcutaneous application of somatotropin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| somatotropin | 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 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Primary: sum of strength (hand held myometry) | 20 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Functional (time) tests, lung function, quality of life, | 20 weeks |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Rudolf Korinthenberg, Professor | University medical centre Freiburg, children's hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| von Haunersches Kinderspital | Munich | Bavaria | 80337 | Germany | ||
| University Children'S Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 24300782 | Result | 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. |
| Label | URL |
|---|---|
| University Children's Hospital Freiburg, Germany | View source |
Not provided
Not provided
| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| D014897 | Spinal Muscular Atrophies of Childhood |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
Not provided
Not provided
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| ID | Term |
|---|---|
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Placebo | 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 |
|
|
| Göttingen |
| Lower Saxony |
| 37075 |
| Germany |
| University Children'S Hospital | Essen | North Rhine-Westphalia | 45122 | Germany |
| University Children'S Hospital | Mainz | Rhineland-Palatinate | 55110 | Germany |
| University Children'S Hospital | Kiel | Schleswig-Holstein | 24105 | Germany |
| Charité, Department of Neurpaediatrics | Berlin | State of Berlin | 13353 | Germany |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D006730 |
| Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |