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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
University Hospital, Toulouse
ClinicalTrials.gov Identifier:
NCT01298180
First received: November 6, 2009
Last updated: October 14, 2014
Last verified: October 2014
  Purpose

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).


Condition Intervention Phase
Prader-Willi Syndrome
Growth Hormone Deficiency
Drug: Growth hormone (Genotonorm® or Omnitrope®)
Procedure: DEXA, blood tests, H.G.P.O, osseous age.
Procedure: biopsy
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?

Resource links provided by NLM:


Further study details as provided by University Hospital, Toulouse:

Primary Outcome Measures:
  • Measure of the circulating rates of IGF-I under treatment. [ Time Frame: Before starting treatment: baseline (J0) ] [ Designated as safety issue: No ]
  • Measure of the circulating rates of IGF-I under treatment. [ Time Frame: 1 month (M1) ] [ Designated as safety issue: No ]
  • Measure of the circulating rates of IGF-I under treatment. [ Time Frame: 3 month (M3) ] [ Designated as safety issue: No ]
  • Measure of the circulating rates of IGF-I under treatment. [ Time Frame: 6 month (M6) ] [ Designated as safety issue: No ]
  • Measure of the circulating rates of IGF-I under treatment. [ Time Frame: 1 year (M12) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin. [ Time Frame: Before starting treatment (J0) ] [ Designated as safety issue: No ]
  • Measure of physical composition's variation. [ Time Frame: Before starting treatment (J0) ] [ Designated as safety issue: No ]
  • Measure of blood sugar level, H.G.P.O., and hyperglycaemia. [ Time Frame: Before starting treatment (J0) ] [ Designated as safety issue: No ]
  • Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy. [ Time Frame: Before starting treatment (J0) ] [ Designated as safety issue: No ]
  • Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin. [ Time Frame: 3 months (M3) ] [ Designated as safety issue: No ]
  • Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin. [ Time Frame: 6 months (M6) ] [ Designated as safety issue: No ]
  • Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin. [ Time Frame: 1 year (M12) ] [ Designated as safety issue: No ]
  • Measure of physical composition's variation. [ Time Frame: 3 months (M3) ] [ Designated as safety issue: No ]
  • Measure of physical composition's variation. [ Time Frame: 6 months (M6) ] [ Designated as safety issue: No ]
  • Measure of physical composition's variation. [ Time Frame: 1 year (M12) ] [ Designated as safety issue: No ]
  • Measure of blood sugar level, H.G.P.O., and hyperglycaemia. [ Time Frame: 3 months (M3) ] [ Designated as safety issue: No ]
  • Measure of blood sugar level, H.G.P.O., and hyperglycaemia. [ Time Frame: 6 months (M6) ] [ Designated as safety issue: No ]
  • Measure of blood sugar level, H.G.P.O., and hyperglycaemia. [ Time Frame: 1 year (M12) ] [ Designated as safety issue: No ]
  • Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy. [ Time Frame: 3 months (M3) ] [ Designated as safety issue: No ]
  • Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy. [ Time Frame: 6 months (M6) ] [ Designated as safety issue: No ]
  • Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy. [ Time Frame: 1 year (M12) ] [ Designated as safety issue: No ]

Enrollment: 111
Study Start Date: January 2009
Study Completion Date: May 2013
Primary Completion Date: May 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: SPW
Children presenting a Prader-Willi Syndrome
Drug: Growth hormone (Genotonorm® or Omnitrope®)
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.
Procedure: DEXA, blood tests, H.G.P.O, osseous age.

SPW, GHD, SPW-B :

blood tests : centralized dosage H.G.P.O : adjusted to children's age.

Experimental: GHD
Patient deficient in Growth Hormone
Drug: Growth hormone (Genotonorm® or Omnitrope®)
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.
Procedure: DEXA, blood tests, H.G.P.O, osseous age.

SPW, GHD, SPW-B :

blood tests : centralized dosage H.G.P.O : adjusted to children's age.

Experimental: SPW-B
Patient with Prader-Willi Syndrome who has Biopsy
Drug: Growth hormone (Genotonorm® or Omnitrope®)
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.
Procedure: DEXA, blood tests, H.G.P.O, osseous age.

SPW, GHD, SPW-B :

blood tests : centralized dosage H.G.P.O : adjusted to children's age.

Procedure: biopsy
Biopsy : Cutaneous and fat tissue biopsy.
Experimental: T
Patient Control
Procedure: biopsy
Biopsy : Cutaneous and fat tissue biopsy.
Experimental: SPW-GH-B
Patient with Prader-Willi Syndrome taking growth Hormone and who has biopsy
Procedure: biopsy
Biopsy : Cutaneous and fat tissue biopsy.

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

Ages Eligible for Study:   1 Year to 5 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01298180

Locations
France
CHU Amiens Hôpital Nord Service Pédiatrie - Place Victor Pauchet
Amiens, France, 80054
CHU Angers - 4 rue Larrey
Angers, France, 49000
CHG Avignon - 305, rue Raoul Follereau
Avignon, France, 84902
CHU Besançon Hôpital Saint Jacques - 2 Place Saint Jacques
Besancon, France, 25000
CHU Bordeaux Hôpital Pellegrin Service endocrinologie de l'enfant - Place Amélie Raba Léon
Bordeaux, France, 33076
CHU Brest Département de Pédiatrie - 5, ave Foch
Brest, France, 29609
CHU Dijon Service de pédiatrie - 2, Bd Maréchal de lattre de Tassigny
Dijon, France, 21000
CHU Grenoble Service de pédiatrie - BP 217
Grenoble, France, 38043
CHU La Rochelle Service de Pédiatrie - Rue du Dr Schweitzer
La Rochelle, France, 17000
CHRU Lille Hôpital Jeanne de Flandre service de Pédiatrie
Lille, France, 59037
CHU Limoges Hôpital Mère Enfant Service Pédiatrie - 8, ave du Larrey
Limoges, France, 87042
CHU Lorient Hôpital du Scorff Pôle Femme Mère Enfant - Rue Guiguen
Lorient, France, 56100
CHU Lyon Hôpital Debrousse service Pédiatrie
Lyon, France, 69322
AP-HM Hôptal La Timone Service de Pédiatrie Mutidisciplinaire
Marseille, France, 13385
CHU Montpellier Hôpital Arnaud de Villeneuve - 371 ave du doyen Gaston Giraud
Montpellier, France, 34000
CHU Nantes Hôpital Mère Enfant Service de Pédiatrie
Nantes, France, 44093
CHU Nice Hôpital Archet 2 - 151 route Saint Antoine de Ginestière
Nice, France, 06202
AP-HP Hôpital Necker Enfants Malades Service d'endocrinologie pédiatrique - 149 route de Sèvres
Paris, France, 75015
CHU Poitiers Service de Pédiatrie - Rue de la Miléterie
Poitiers, France, 86021
CHU Reims Service de Pédiatrie - 47, rue Cognacq-Jay
Reims, France, 51092
CHU Rouen Hôpital Nicolle - 1, rue de Germont
Rouen, France, 76000
CHU Saint-Etienne Hôpital Nord Service de Pédiatrie
Saint-etienne, France, 42055
CHU Strasbourg Hôpital Haute-Pierre - Avenue Molière
Strasbourg, France, 67200
CHU Toulouse Hôpital des Enfants Service d'endocrinologie - 330 ave de Grande Bretagne
Toulouse, France, 31059
CHRU Tours Centre de Pédiatrie Gatien de Clocheville
Tours, France, 37044
Hôpital d'Enfants - Rue Morvan
Vandoeuvre Les Nancy, France, 54511
Sponsors and Collaborators
University Hospital, Toulouse
Investigators
Principal Investigator: Maithé TAUBER, MD University Hospital, Toulouse
  More Information

No publications provided

Responsible Party: University Hospital, Toulouse
ClinicalTrials.gov Identifier: NCT01298180     History of Changes
Other Study ID Numbers: 0811601, National PHRC 2008
Study First Received: November 6, 2009
Last Updated: October 14, 2014
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by University Hospital, Toulouse:
Prader-Willi Syndrome
Growth Hormone Deficiency

Additional relevant MeSH terms:
Dwarfism, Pituitary
Endocrine System Diseases
Prader-Willi Syndrome
Syndrome
Abnormalities, Multiple
Bone Diseases
Bone Diseases, Developmental
Bone Diseases, Endocrine
Brain Diseases
Central Nervous System Diseases
Chromosome Disorders
Congenital Abnormalities
Disease
Dwarfism
Genetic Diseases, Inborn
Hypopituitarism
Hypothalamic Diseases
Intellectual Disability
Musculoskeletal Diseases
Nervous System Diseases
Neurobehavioral Manifestations
Neurologic Manifestations
Nutrition Disorders
Obesity
Overnutrition
Pathologic Processes
Pituitary Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 23, 2014