Clinical trial NCT01298180: Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?
Official title: Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Prader-Willi Syndrome
Growth Hormone Deficiency
Keywords: Prader-Willi Syndrome
Growth Hormone Deficiency
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
First posted: 2011-02-17
Enrollment: 111
Sex: ALL
Minimum age (years): 1
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).

Detailed description: 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) by the measure of the circulating rates of IGF-I under treatment.

Eligibility criteria: Inclusion Criteria:

1. 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
2. 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.
3. T : controls

   * 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 hospitalized at the hospital of the children of the University Hospital of Toulouse for a programmed surgical operation
   * Child covered by a national insurance scheme or an equivalent
   * Signature of the informed consent by one of both holders of the parental authority
4. SPW-GH-B :

   * Female or male child of age \> or = 1 year
   * Child hospitalized for a programmed surgical operation
   * Child covered by a national insurance scheme or an equivalent
   * Child treated with GH for at least 3 month
   * Signature of the informed consent by one of both holders of the parental authority

Exclusion Criteria:

1. 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)
2. 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®
   * Child presenting a porphyria
   * Child presenting a congenital methemoglobinemia
   * Child presenting a contraindication to Meopa : patients requiring a ventilation in pure oxygen, intracranial High blood pressure, Any change of the state of consciousness, preventing the cooperation of the patient, Pneumothorax, Bubbles of emphysema, Gaseous embolism, Accident of dive, abdominal gaseous Distension, Patient having received recently an ophthalmic gas (SF6, C3F8, C2F6) used in the eye surgery as long as persists a bubble of gas inside the eye and at least during a period of 3 months. Grave postoperative complications can arise in touch with the increase of the pressure intraocular, facial Traumatism interesting the region of application of the mask
3. T : controls

   * Chronicle pathology in which an abnormality of growth would be involved
   * Other hormonal abnormalities
   * Children receiving a treatment on the long range, corticosteroid therapy in particular, being able to interfere with the sensibility to GH or to the insulin
   * Holder of the parental authority under supervision, guardianship or under protection of justice
   * Participation in another study simultaneously at this one

Primary outcomes:
measure: Measure of the circulating rates of IGF-I under treatment.
timeFrame: Before starting treatment: baseline (J0)
measure: Measure of the circulating rates of IGF-I under treatment.
timeFrame: 1 month (M1)
measure: Measure of the circulating rates of IGF-I under treatment.
timeFrame: 3 month (M3)
measure: Measure of the circulating rates of IGF-I under treatment.
timeFrame: 6 month (M6)
measure: Measure of the circulating rates of IGF-I under treatment.
timeFrame: 1 year (M12)
Secondary outcomes:
measure: Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.
timeFrame: Before starting treatment (J0)
measure: Measure of physical composition's variation.
timeFrame: Before starting treatment (J0)
measure: Measure of blood sugar level, H.G.P.O., and hyperglycaemia.
timeFrame: Before starting treatment (J0)
measure: Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.
timeFrame: Before starting treatment (J0)
measure: Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.
timeFrame: 3 months (M3)
measure: Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.
timeFrame: 6 months (M6)
measure: Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.
timeFrame: 1 year (M12)
measure: Measure of physical composition's variation.
timeFrame: 3 months (M3)
measure: Measure of physical composition's variation.
timeFrame: 6 months (M6)
measure: Measure of physical composition's variation.
timeFrame: 1 year (M12)
measure: Measure of blood sugar level, H.G.P.O., and hyperglycaemia.
timeFrame: 3 months (M3)
measure: Measure of blood sugar level, H.G.P.O., and hyperglycaemia.
timeFrame: 6 months (M6)
measure: Measure of blood sugar level, H.G.P.O., and hyperglycaemia.
timeFrame: 1 year (M12)
measure: Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.
timeFrame: 3 months (M3)
measure: Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.
timeFrame: 6 months (M6)
measure: Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.
timeFrame: 1 year (M12)
Other outcomes:
Locations:
city: Amiens
country: France
facility: CHU Amiens Hôpital Nord Service Pédiatrie - Place Victor Pauchet
city: Angers
country: France
facility: CHU Angers - 4 rue Larrey
city: Avignon
country: France
facility: CHG Avignon - 305, rue Raoul Follereau
city: Besancon
country: France
facility: CHU Besançon Hôpital Saint Jacques - 2 Place Saint Jacques
city: Bordeaux
country: France
facility: CHU Bordeaux Hôpital Pellegrin Service endocrinologie de l'enfant - Place Amélie Raba Léon
city: Brest
country: France
facility: CHU Brest Département de Pédiatrie - 5, ave Foch
city: Dijon
country: France
facility: CHU Dijon Service de pédiatrie - 2, Bd Maréchal de lattre de Tassigny
city: Grenoble
country: France
facility: CHU Grenoble Service de pédiatrie - BP 217
city: La Rochelle
country: France
facility: CHU La Rochelle Service de Pédiatrie - Rue du Dr Schweitzer
city: Lille
country: France
facility: CHRU Lille Hôpital Jeanne de Flandre service de Pédiatrie
city: Limoges
country: France
facility: CHU Limoges Hôpital Mère Enfant Service Pédiatrie - 8, ave du Larrey
city: Lorient
country: France
facility: CHU Lorient Hôpital du Scorff Pôle Femme Mère Enfant - Rue Guiguen
city: Lyon
country: France
facility: CHU Lyon Hôpital Debrousse service Pédiatrie
city: Marseille
country: France
facility: AP-HM Hôptal La Timone Service de Pédiatrie Mutidisciplinaire
city: Montpellier
country: France
facility: CHU Montpellier Hôpital Arnaud de Villeneuve - 371 ave du doyen Gaston Giraud
city: Nantes
country: France
facility: CHU Nantes Hôpital Mère Enfant Service de Pédiatrie
city: Nice
country: France
facility: CHU Nice Hôpital Archet 2 - 151 route Saint Antoine de Ginestière
city: Paris
country: France
facility: AP-HP Hôpital Necker Enfants Malades Service d'endocrinologie pédiatrique - 149 route de Sèvres
city: Poitiers
country: France
facility: CHU Poitiers Service de Pédiatrie - Rue de la Miléterie
city: Reims
country: France
facility: CHU Reims Service de Pédiatrie - 47, rue Cognacq-Jay
city: Rouen
country: France
facility: CHU Rouen Hôpital Nicolle - 1, rue de Germont
city: Saint-etienne
country: France
facility: CHU Saint-Etienne Hôpital Nord Service de Pédiatrie
city: Strasbourg
country: France
facility: CHU Strasbourg Hôpital Haute-Pierre - Avenue Molière
city: Toulouse
country: France
facility: CHU Toulouse Hôpital des Enfants Service d'endocrinologie - 330 ave de Grande Bretagne
city: Tours
country: France
facility: CHRU Tours Centre de Pédiatrie Gatien de Clocheville
city: Vandoeuvre Les Nancy
country: France
facility: Hôpital d'Enfants - Rue Morvan
Study officials:
affiliation: University Hospital, Toulouse
name: Maithé TAUBER, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.