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

Growth Hormone Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01298180
• Other study ID #: 0811601
• Secondary ID: National PHRC 20
• Status: Completed
• Phase: Phase 4
• Start date: January 2009
• Est. completion date: May 2013

Study information

• Verified date: September 2021
• Source: University Hospital, Toulouse
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 111
• Est. completion date: May 2013
• Est. primary completion date: May 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 5 Years

Eligibility

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

Related Conditions & MeSH terms

• Growth Hormone Deficiency
• Prader-Willi Syndrome
• Syndrome

Intervention

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.
• biopsy
Biopsy : Cutaneous and fat tissue biopsy.

Locations

Country	Name	City	State
France	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	Besancon
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	Vandoeuvre Les Nancy

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Toulouse

Country where clinical trial is conducted

France, 

References & Publications (3)

Bieth E, Eddiry S, Gaston V, Lorenzini F, Buffet A, Conte Auriol F, Molinas C, Cailley D, Rooryck C, Arveiler B, Cavaillé J, Salles JP, Tauber M. Highly restricted deletion of the SNORD116 region is implicated in Prader-Willi Syndrome. Eur J Hum Genet. 20 — View Citation

Cadoudal T, Buléon M, Sengenès C, Diene G, Desneulin F, Molinas C, Eddiry S, Conte-Auriol F, Daviaud D, Martin PG, Bouloumié A, Salles JP, Tauber M, Valet P. Impairment of adipose tissue in Prader-Willi syndrome rescued by growth hormone treatment. Int J — View 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 May 26. doi: 10. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Measure of the circulating rates of IGF-I under treatment.		Before starting treatment: baseline (J0)
Primary	Measure of the circulating rates of IGF-I under treatment.		1 month (M1)
Primary	Measure of the circulating rates of IGF-I under treatment.		3 month (M3)
Primary	Measure of the circulating rates of IGF-I under treatment.		6 month (M6)
Primary	Measure of the circulating rates of IGF-I under treatment.		1 year (M12)
Secondary	Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.		Before starting treatment (J0)
Secondary	Measure of physical composition's variation.		Before starting treatment (J0)
Secondary	Measure of blood sugar level, H.G.P.O., and hyperglycaemia.		Before starting treatment (J0)
Secondary	Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.		Before starting treatment (J0)
Secondary	Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.		3 months (M3)
Secondary	Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.		6 months (M6)
Secondary	Measure of the circulating rate of IGFBP-3, GHBP, ghrelin and apelin.		1 year (M12)
Secondary	Measure of physical composition's variation.		3 months (M3)
Secondary	Measure of physical composition's variation.		6 months (M6)
Secondary	Measure of physical composition's variation.		1 year (M12)
Secondary	Measure of blood sugar level, H.G.P.O., and hyperglycaemia.		3 months (M3)
Secondary	Measure of blood sugar level, H.G.P.O., and hyperglycaemia.		6 months (M6)
Secondary	Measure of blood sugar level, H.G.P.O., and hyperglycaemia.		1 year (M12)
Secondary	Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.		3 months (M3)
Secondary	Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.		6 months (M6)
Secondary	Measure of the sensibility at the growth hormone in vitro, on fibroblasts and adipocytes obtained by biopsy.		1 year (M12)