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

NCT ID: NCT01298180

Last Updated: 2021-09-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 111 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-01-31
• Study Completion Date: 2013-05-31

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.

Conditions

Prader-Willi Syndrome; Growth Hormone Deficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

SPW

Children presenting a Prader-Willi Syndrome

Group Type: EXPERIMENTAL

Growth hormone (Genotonorm® or Omnitrope®)

Intervention Type: 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.

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

Intervention Type: PROCEDURE

SPW, GHD, SPW-B :

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

GHD

Patient deficient in Growth Hormone

Group Type: EXPERIMENTAL

Growth hormone (Genotonorm® or Omnitrope®)

Intervention Type: 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.

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

Intervention Type: PROCEDURE

SPW, GHD, SPW-B :

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

SPW-B

Patient with Prader-Willi Syndrome who has Biopsy

Group Type: EXPERIMENTAL

Growth hormone (Genotonorm® or Omnitrope®)

Intervention Type: 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.

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

Intervention Type: PROCEDURE

SPW, GHD, SPW-B :

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

biopsy

Intervention Type: PROCEDURE

Biopsy : Cutaneous and fat tissue biopsy.

T

Patient Control

Group Type: EXPERIMENTAL

biopsy

Intervention Type: PROCEDURE

Biopsy : Cutaneous and fat tissue biopsy.

SPW-GH-B

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

Group Type: EXPERIMENTAL

biopsy

Intervention Type: PROCEDURE

Biopsy : Cutaneous and fat tissue biopsy.

Interventions

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.

Intervention Type: DRUG

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.

Intervention Type: PROCEDURE

biopsy

Biopsy : Cutaneous and fat tissue biopsy.

Intervention Type: PROCEDURE

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

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Toulouse

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Maithé TAUBER, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Toulouse

Locations

CHU Amiens Hôpital Nord Service Pédiatrie - Place Victor Pauchet

Amiens, , France

CHU Angers - 4 rue Larrey

Angers, , France

CHG Avignon - 305, rue Raoul Follereau

Avignon, , France

CHU Besançon Hôpital Saint Jacques - 2 Place Saint Jacques

Besançon, , France

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

Bordeaux, , France

CHU Brest Département de Pédiatrie - 5, ave Foch

Brest, , France

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

Dijon, , France

CHU Grenoble Service de pédiatrie - BP 217

Grenoble, , France

CHU La Rochelle Service de Pédiatrie - Rue du Dr Schweitzer

La Rochelle, , France

CHRU Lille Hôpital Jeanne de Flandre service de Pédiatrie

Lille, , France

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

Limoges, , France

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

Lorient, , France

CHU Lyon Hôpital Debrousse service Pédiatrie

Lyon, , France

AP-HM Hôptal La Timone Service de Pédiatrie Mutidisciplinaire

Marseille, , France

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

Montpellier, , France

CHU Nantes Hôpital Mère Enfant Service de Pédiatrie

Nantes, , France

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

Nice, , France

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

Paris, , France

CHU Poitiers Service de Pédiatrie - Rue de la Miléterie

Poitiers, , France

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

Reims, , France

CHU Rouen Hôpital Nicolle - 1, rue de Germont

Rouen, , France

CHU Saint-Etienne Hôpital Nord Service de Pédiatrie

Saint-Etienne, , France

CHU Strasbourg Hôpital Haute-Pierre - Avenue Molière

Strasbourg, , France

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

Toulouse, , France

CHRU Tours Centre de Pédiatrie Gatien de Clocheville

Tours, , France

Hôpital d'Enfants - Rue Morvan

Vandœuvre-lès-Nancy, , France

Countries

France

References

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.

Reference Type: RESULT

PMID: 24406482 (View on PubMed)

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.

Reference Type: RESULT

PMID: 34040195 (View on PubMed)

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.

Reference Type: RESULT

PMID: 24916642 (View on PubMed)

Other Identifiers

National PHRC 2008

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

0811601

Identifier Type: -

Identifier Source: org_study_id