Study with adult patients with Prader-Willi syndrome, which evaluate the effect of growth hormone therapy on muscle tone and its relation with muscle strength and body composition assessed with imaging procedures.

Phase 1

• Conditions: Patients with Prader-Willi Syndrome (SPW) with Growth hormone deficit.; MedDRA version: 20.0Level: PTClassification code 10036476Term: Prader-Willi syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2017-002164-41-ES
• Lead Sponsor: Fundació Parc Taulí

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-06-21
• Last Update Posted: 21 August 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Patients with PWS = 18 years
• Patients who have demonstrated a GH deficiency in at least one of the
two GH stimuli tests performed in routine clinical practice
• Patients (or their legal guardians if they are legally incapacitated) who
have signed informed consent to participate in the study. In the case of
patients legally incapacitated, they will give their assent in participating.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Contraindications for MRI
• Inability to stand still during MRI
• Impossibility to understand the paradigm to be realized during the MRI
• Severe obesity, uncontrolled diabetes, severe untreated obstructive
sleep apnea, active cancer and active psychosis
• Patients who present any other contraindication to treatment with GH
according to the data sheet.
• Any circumstance that at the discretion of the physician may pose a
risk or clinical harm to the patient's participation in the study or
interfere with the assessments of the same.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To assess the muscle tone with functional magnetic resonance imaging (fMRI) in adult patients undergoing SPW with hormone deficit, before and after 12 months of GH treatment.;Secondary Objective: 1. To assess the effect of GH treatment on the muscle strength before and after 12 months of treatment.<br>2. To assess the effect of GH treatment on the body composition before and after 12 months of treatment.<br>3. To study if there are changes on different cognitives aspects before and after 12 months of treatment.<br>4. To study if there are changes in some of the metabolism regulatory hormones (leptina and kisspeptina) and if they correlate with body composition changes.<br>5. To study the sleeping architecture, the <br>presence of respiratory sleep disorder and the sleeping quality, before and after 12 months of GH treatment.;Primary end point(s): Muscle tone with functional magnetic resonance imaging (fMRI);Timepoint(s) of evaluation of this end point: 12 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1. Study of muscle strength<br>2. Study of body composition<br>3. Study of bone mineral density<br>4. Mental and cognitive assessment<br>5. Polysomnography<br>6. Hormonal study<br>7. Safety;Timepoint(s) of evaluation of this end point: 12 months