Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?

NCT01298180 | Completed Phase 4

• 2 other identifiers: 0811601National PHRC 2008
• Study Type: interventional
• Target: 111
• Locations: 1 country
• Sites: 26

Brief Summary

The purpose of this study is to estimate the sensibility at the growth hormone in vivo at the children presenting a Prader-Willi syndrome (SPW) in comparison with children presenting a deficit in growth hormone (GHD).

Conditions

Prader-Willi Syndrome; Growth Hormone Deficiency

Keywords

Prader-Willi Syndrome; Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (5)

• Measure of the circulating rates of IGF-I under treatment.

  Before starting treatment: baseline (J0)

• Measure of the circulating rates of IGF-I under treatment.

  1 month (M1)

• Measure of the circulating rates of IGF-I under treatment.

  3 month (M3)

• Measure of the circulating rates of IGF-I under treatment.

  6 month (M6)

• Measure of the circulating rates of IGF-I under treatment.

  1 year (M12)

Secondary Outcomes (16)

• Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.

  Before starting treatment (J0)

• Measure of physical composition's variation.

  Before starting treatment (J0)

• Measure of blood sugar level, H.G.P.O., and hyperglycaemia.

  Before starting treatment (J0)

• Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.

  Before starting treatment (J0)

• Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.

  3 months (M3)

Study Arms (5)

SPW

EXPERIMENTAL

Children presenting a Prader-Willi Syndrome

Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.

GHD

EXPERIMENTAL

Patient deficient in Growth Hormone

Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.

SPW-B

EXPERIMENTAL

Patient with Prader-Willi Syndrome who has Biopsy

Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.; Procedure: biopsy

T

EXPERIMENTAL

Patient Control

Procedure: biopsy

SPW-GH-B

EXPERIMENTAL

Patient with Prader-Willi Syndrome taking growth Hormone and who has biopsy

Procedure: biopsy

Interventions

Growth hormone (Genotonorm® or Omnitrope®) DRUG

drug : the treatment will be begun in progressive dose by beginning by ¼ of the dose the first week, then ½ of the dose the second week, then 3/4 of the dose the third week and total dose the fourth week.

GHD; SPW; SPW-B

DEXA, blood tests, H.G.P.O, osseous age. PROCEDURE

SPW, GHD, SPW-B : blood tests : centralized dosage H.G.P.O : adjusted to children's age.

GHD; SPW; SPW-B

biopsy PROCEDURE

Biopsy : Cutaneous and fat tissue biopsy.

SPW-B; SPW-GH-B; T

Eligibility Criteria

• Age: 1 Year - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• SPW and SPW-B :
• Female or male child of age \> or = 1 year
• Child naïve of treatment by GH and that must begin a treatment with GH
• Child covered by a national insurance scheme or an equivalent
• Signature of the informed consent by one of both holders of the parental authority
• GHD :
• Female or male child of age \> or = 1 year
• Child paired for the age (+/-on 1 year) and for the sex with regard to the group SWP
• Child presenting a GH\* deficiency defined by :
• Growth criteria of size (size) \< 2 DS) Criteria of speed of growth (speed of growth \< 1 DS over the last year) 2 tests of pharmacological stimulation of GH with peak GH max \< 20 mUI
• Child naïve of treatment by GH and that must begin a treatment with GH
• Child covered by a national insurance scheme or an equivalent
• Signature of the informed consent by one of both holders of the parental authority \* The deficit in GH can be isolated or associated with one or several other hormonal deficits: deficit in TSH, deficit in ACTH, deficit in LH-FSH, deficit in prolactin. The child GHD can thus receive other treatments associated with the growth hormone.
• T : controls
• Female or male child of age \> or = 1 year

You may not qualify if:

• SPW and GHD
• Child presenting a contraindication to the taking of growth hormone :
• Growth cartilage welded
• Tumoral pathology in process of evolution
• Corticosteroid therapy (not substitute)
• Allergy known about solvent
• Badly balanced diabetes
• Child presenting a hypersensitivity to the active principle or to one of the excipients of Genotonorm ® or Omnitrope ®
• Child presenting a severe obesity (defined by a report weight / size \> 200 %)
• Child presenting clinical signs ENT (snores associated with a hypertrophy of the adenoids vegetations and\\or the tonsils)
• Child presenting clinical signs evoking a respiratory illness of the sleep (night-respiratory snores, respiratory breaks during the sleep)
• SPW-B:
• Child presenting a hypersensitivity to the local anaesthetic with amide connecion
• Child presenting a hypersensitivity to the components of the bandage Emlapatch®
• Child presenting a hypersensitivity to one of the components of the lidocaïne aguettant without conservative®

Sponsors & Collaborators

• University Hospital, Toulouse: lead

Study Sites (26)

CHU Amiens Hôpital Nord Service Pédiatrie - Place Victor Pauchet

Amiens, 80054, France

Location

CHU Angers - 4 rue Larrey

Angers, 49000, France

Location

CHG Avignon - 305, rue Raoul Follereau

Avignon, 84902, France

Location

CHU Besançon Hôpital Saint Jacques - 2 Place Saint Jacques

Besançon, 25000, France

Location

CHU Bordeaux Hôpital Pellegrin Service endocrinologie de l'enfant - Place Amélie Raba Léon

Bordeaux, 33076, France

Location

CHU Brest Département de Pédiatrie - 5, ave Foch

Brest, 29609, France

Location

CHU Dijon Service de pédiatrie - 2, Bd Maréchal de lattre de Tassigny

Dijon, 21000, France

Location

CHU Grenoble Service de pédiatrie - BP 217

Grenoble, 38043, France

Location

CHU La Rochelle Service de Pédiatrie - Rue du Dr Schweitzer

La Rochelle, 17000, France

Location

CHRU Lille Hôpital Jeanne de Flandre service de Pédiatrie

Lille, 59037, France

Location

CHU Limoges Hôpital Mère Enfant Service Pédiatrie - 8, ave du Larrey

Limoges, 87042, France

Location

CHU Lorient Hôpital du Scorff Pôle Femme Mère Enfant - Rue Guiguen

Lorient, 56100, France

Location

CHU Lyon Hôpital Debrousse service Pédiatrie

Lyon, 69322, France

Location

AP-HM Hôptal La Timone Service de Pédiatrie Mutidisciplinaire

Marseille, 13385, France

Location

CHU Montpellier Hôpital Arnaud de Villeneuve - 371 ave du doyen Gaston Giraud

Montpellier, 34000, France

Location

CHU Nantes Hôpital Mère Enfant Service de Pédiatrie

Nantes, 44093, France

Location

CHU Nice Hôpital Archet 2 - 151 route Saint Antoine de Ginestière

Nice, 06202, France

Location

AP-HP Hôpital Necker Enfants Malades Service d'endocrinologie pédiatrique - 149 route de Sèvres

Paris, 75015, France

Location

CHU Poitiers Service de Pédiatrie - Rue de la Miléterie

Poitiers, 86021, France

Location

CHU Reims Service de Pédiatrie - 47, rue Cognacq-Jay

Reims, 51092, France

Location

CHU Rouen Hôpital Nicolle - 1, rue de Germont

Rouen, 76000, France

Location

CHU Saint-Etienne Hôpital Nord Service de Pédiatrie

Saint-Etienne, 42055, France

Location

CHU Strasbourg Hôpital Haute-Pierre - Avenue Molière

Strasbourg, 67200, France

Location

CHU Toulouse Hôpital des Enfants Service d'endocrinologie - 330 ave de Grande Bretagne

Toulouse, 31059, France

Location

CHRU Tours Centre de Pédiatrie Gatien de Clocheville

Tours, 37044, France

Location

Hôpital d'Enfants - Rue Morvan

Vandœuvre-lès-Nancy, 54511, France

Location

Related Publications (3)

• Cadoudal T, Buleon M, Sengenes C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumie A, Salles JP, Tauber M, Valet P. Impairment of adipose tissue in Prader-Willi syndrome rescued by growth hormone treatment. Int J Obes (Lond). 2014 Sep;38(9):1234-40. doi: 10.1038/ijo.2014.3. Epub 2014 Jan 10.

  PMID: 24406482 RESULT

• Eddiry S, Diene G, Molinas C, Salles J, Auriol FC, Gennero I, Bieth E, Skryabin BV, Rozhdestvensky TS, Burnett LC, Leibel RL, Tauber M, Salles JP. SNORD116 and growth hormone therapy impact IGFBP7 in Prader-Willi syndrome. Genet Med. 2021 Sep;23(9):1664-1672. doi: 10.1038/s41436-021-01185-y. Epub 2021 May 26.

  PMID: 34040195 RESULT

• Bieth E, Eddiry S, Gaston V, Lorenzini F, Buffet A, Conte Auriol F, Molinas C, Cailley D, Rooryck C, Arveiler B, Cavaille J, Salles JP, Tauber M. Highly restricted deletion of the SNORD116 region is implicated in Prader-Willi Syndrome. Eur J Hum Genet. 2015 Feb;23(2):252-5. doi: 10.1038/ejhg.2014.103. Epub 2014 Jun 11.

  PMID: 24916642 RESULT

MeSH Terms

Conditions

Prader-Willi Syndrome; Dwarfism, Pituitary

Interventions

Growth Hormone; Human Growth Hormone; Absorptiometry, Photon; Hematologic Tests; Biopsy

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases; Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins; Radiography; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis; Densitometry; Photometry; Chemistry Techniques, Analytical; Investigative Techniques; Clinical Laboratory Techniques; Cytodiagnosis; Cytological Techniques; Specimen Handling; Diagnostic Techniques, Surgical; Surgical Procedures, Operative

Study Officials

• Maithé TAUBER, MD

  University Hospital, Toulouse

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 6, 2009
• First Posted: February 17, 2011
• Study Start: January 1, 2009
• Primary Completion: May 1, 2013
• Study Completion: May 1, 2013
• Last Updated: September 24, 2021

Record last verified: 2021-09

Locations

FR (26)