E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Prader Willi Syndrome
Chromosome defect on chromosome 15 |
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E.1.1.2 | Therapeutic area | Diseases [C] - Hormonal diseases [C19] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10041331 |
E.1.2 | Term | Somatotrophin |
E.1.2 | System Organ Class | 10022891 - Investigations |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
- A manualized treatment for an optimal dosage GH in young adults with Prader Willi Syndrome
- To assess the long term effects and safety of GH-treatment in a dose of 0.33mg/m2/day on:
- body composition
- carbohydrate metabolism
- serum lipids
- blood pressure
- behavioral problems
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E.2.2 | Secondary objectives of the trial |
- To study the effects of long term GH-treatment on thyroid hormone levels, IGF-I and IGF binding proteins, adiponectin, leptin and ghrelin.
- To study the compliance to the diet.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Young adults with PWS who have been treated with GH for several years in the original Cohort study or Transition study will be included. They will be treated with GH according to this new follow-up protocol from 3 months after the end of the Transition study or when the inclusion in the Transition study has been completed. |
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E.4 | Principal exclusion criteria |
The study participation will be closed to patients who do not meet the above mentioned inclusion criteria and/or have:
- non cooperative behavior
- extremely low dietary intake of less than minimal required intake according to WHO
- medication to reduce weight (fat)
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E.5 End points |
E.5.1 | Primary end point(s) |
- Weight, Waist-hip ratio
- Blood pressure
• DXA for body composition and bone density, radiation exposure 200 mSv per scan
Laboratory assessments
- ASAT, ALAT, Creatinin,
- Fasting glucose and insulin, OGTT
- Fasting lipid levels: trygliceride (TG), total cholesterol, LDL, HDL and Lipoprotein [a]
- IGF-I, IGFBP-1, IGFBP-3
- T4, free T4, TSH, T3, Reverse T3
- Leptin, Adiponectin, Ghrelin
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At six months, 12 months and 24 months |
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E.5.2 | Secondary end point(s) |
• Nutritional intake during 7 days (via daily intake book)
Fasting glucose and insulin, OGTT
- Fasting lipid levels: trygliceride (TG), total cholesterol, LDL, HDL and Lipoprotein [a]
- IGF-I, IGFBP-1, IGFBP-3
- T4, free T4, TSH, T3, Reverse T3
- Leptin, Adiponectin, Ghrelin
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At six months, 12 months and 24 months |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
Registration of growth hormone therapy in adults with Prader Willi Syndroom in The Netherlands |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |