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Completed

NCT Number: NCT03616509

GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition

Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). The investigators has experience in the study of brain connectivity in these patients in relation to satiety. To date, there is no evidence about the effect of GH on central hypotonia (brain areas related with muscle tone maintenance). So, the main objective is to examine these anatomical areas before and one year after GH treatment.

Methodology: Structural and functional magnetic resonance imaging to 30 PWS patients before and after GH treatment and we will compare them to a control group.

Expected results: PWS group will show abnormal functional and structural connectivity in circuitry of muscle tone maintenance that will improve after GH treatment. These favorable changes and the absence of secondary effects will help to justify the use of this treatment and its inclusion in practical clinical guidelines of HNS for the management of this syndrome in the adulthood.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Parc Taulí Hospital Universitari

Sabadell, Barcelona, 08208, Spain

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • PWS >=18 years with growth hormone deficit
  • Signed informed consent by the patients or their legal guardian

Exclusion criteria

  • Severe obesity
  • No controled diabetes mellitus
  • No treated obstructive sleep apnea or severe obstructive sleep apnea
  • Active cancer
  • Active psychosis

Treatment and study plan

growth hormone

Drug

12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level

Other names: Genotonorm Miniquick

Placebo

Drug

2 months on placebo, sodium chloride 0,9% injections

Other names: Physiological serum (sodium chloride 0,9%)

Primary outcomes

  1. Examine brain connectivity areas related with muscle tone maintenance

    Time frame: 24 months

    Examine brain connectivity areas related with muscle tone maintenance making a functional MRI while the subject is making motor maneuvers, before anf after 12 months on GH treatment.

Secondary outcomes

  1. Evaluation of strength

    Time frame: 24 months

    Evaluation of strength before and after 12 months on GH treatment

  2. Evaluation of body composition

    Time frame: 24 months

    Evaluation of body composition by Dual energy x-ray absorptiometry (DEXA) before and after 12 months on GH treatment

Sponsors and collaborators

Lead sponsor

Corporacion Parc Tauli

Other

Collaborators

  • Parc de Salut Mar

Registry information

Official study title

Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition

Important dates

Study start
2017
Primary completion
2019
Study completion
2019
First posted
Aug 6, 2018
Registry last updated
Jan 27, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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