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Prader-Willi SyndromePhase 4NCT01298180

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

A Phase 4 study testing Growth hormone (Genotonorm® or Omnitrope®), DEXA, blood tests, H.G.P.O, osseous age. and biopsy in people with Prader-Willi Syndrome and Growth Hormone Deficiency, run by University Hospital, Toulouse at 26 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

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). Read more about this study

Who is it for?
Ages 1 year to 5 years
What is being tested?
Growth hormone (Genotonorm® or Omnitrope®), DEXA, blood tests, H.G.P.O, osseous age. and biopsy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
111 participants
Where
26 sitesFrance
Study dates
Jan 2009to May 2013
Phase
Phase 4
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 5 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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 :
Show all 25Show fewer
  • 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
  • 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
  • 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

Who cannot join

  • 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
Show all 24Show fewer
  • 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®
  • 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
  • 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

Trial locations and contacts

26 sites in 1 country.

26 sites in 1 country

France26 sites

  • CHU Amiens Hôpital Nord Service Pédiatrie - Place Victor Pauchet

    Amiens, 80054

  • CHU Angers - 4 rue Larrey

    Angers, 49000

  • CHG Avignon - 305, rue Raoul Follereau

    Avignon, 84902

  • CHU Besançon Hôpital Saint Jacques - 2 Place Saint Jacques

    Besançon, 25000

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

    Bordeaux, 33076

  • CHU Brest Département de Pédiatrie - 5, ave Foch

    Brest, 29609

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

    Dijon, 21000

  • CHU Grenoble Service de pédiatrie - BP 217

    Grenoble, 38043

  • CHU La Rochelle Service de Pédiatrie - Rue du Dr Schweitzer

    La Rochelle, 17000

  • CHRU Lille Hôpital Jeanne de Flandre service de Pédiatrie

    Lille, 59037

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

    Limoges, 87042

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

    Lorient, 56100

  • CHU Lyon Hôpital Debrousse service Pédiatrie

    Lyon, 69322

  • AP-HM Hôptal La Timone Service de Pédiatrie Mutidisciplinaire

    Marseille, 13385

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

    Montpellier, 34000

  • CHU Nantes Hôpital Mère Enfant Service de Pédiatrie

    Nantes, 44093

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

    Nice, 06202

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

    Paris, 75015

  • CHU Poitiers Service de Pédiatrie - Rue de la Miléterie

    Poitiers, 86021

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

    Reims, 51092

  • CHU Rouen Hôpital Nicolle - 1, rue de Germont

    Rouen, 76000

  • CHU Saint-Etienne Hôpital Nord Service de Pédiatrie

    Saint-Etienne, 42055

  • CHU Strasbourg Hôpital Haute-Pierre - Avenue Molière

    Strasbourg, 67200

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

    Toulouse, 31059

  • CHRU Tours Centre de Pédiatrie Gatien de Clocheville

    Tours, 37044

  • Hôpital d'Enfants - Rue Morvan

    Vandœuvre-lès-Nancy, 54511

Other trials in France

About this study

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).

Read the 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.

Being tested
Growth hormone (Genotonorm® or Omnitrope®), DEXA, blood tests, H.G.P.O, osseous age., biopsy
Keywords
Prader-Willi Syndrome, Growth Hormone Deficiency
Medical subject headings
Prader-Willi Syndrome, Dwarfism, Pituitary

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • 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.

  • DEXA, blood tests, H.G.P.O, osseous age.Procedure or surgery

    SPW, GHD, SPW-B :

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

  • biopsyProcedure or surgery

    Biopsy : Cutaneous and fat tissue biopsy.

Study groups in NCT01298180
Study groupRoleWhat they receive
SPWExperimentalChildren presenting a Prader-Willi Syndrome
Receives: Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.
GHDExperimentalPatient deficient in Growth Hormone
Receives: Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.
SPW-BExperimentalPatient with Prader-Willi Syndrome who has Biopsy
Receives: Drug: Growth hormone (Genotonorm® or Omnitrope®); Procedure: DEXA, blood tests, H.G.P.O, osseous age.; Procedure: biopsy
TExperimentalPatient Control
Receives: Procedure: biopsy
SPW-GH-BExperimentalPatient with Prader-Willi Syndrome taking growth Hormone and who has biopsy
Receives: Procedure: biopsy

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured: Before starting treatment: baseline (J0)

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

    Measured: 1 month (M1)

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

    Measured: 3 month (M3)

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

    Measured: 6 month (M6)

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

    Measured: 1 year (M12)

Secondary outcomes (16)

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

    Measured: Before starting treatment (J0)

  2. Measure of physical composition's variation.

    Measured: Before starting treatment (J0)

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

    Measured: Before starting treatment (J0)

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

    Measured: Before starting treatment (J0)

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

    Measured: 3 months (M3)

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

    Measured: 6 months (M6)

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

    Measured: 1 year (M12)

  8. Measure of physical composition's variation.

    Measured: 3 months (M3)

  9. Measure of physical composition's variation.

    Measured: 6 months (M6)

  10. Measure of physical composition's variation.

    Measured: 1 year (M12)

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

    Measured: 3 months (M3)

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

    Measured: 6 months (M6)

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

    Measured: 1 year (M12)

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

    Measured: 3 months (M3)

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

    Measured: 6 months (M6)

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

    Measured: 1 year (M12)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Feb 2011 (estimated)
  2. Study startJan 2009
  3. Primary completionMay 2013
  4. Study completionMay 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
111 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01298180
Sponsor's protocol ID
0811601
Other IDs
National PHRC 2008
Status last verified
Sep 2021
First submitted
6 Nov 2009
Last updated
24 Sep 2021
Results posted
No
Data monitoring committee
No

Publications

  • 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.

    PubMed 24406482

  • 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.

    PubMed 34040195

  • 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.

    PubMed 24916642

  • Salles J, Eddiry S, Amri S, Galindo M, Lacassagne E, George S, Mialhe X, Lhuillier E, Franchitto N, Jeanneteau F, Gennero I, Salles JP, Tauber M. Differential DNA methylation in iPSC-derived dopaminergic neurons: a step forward on the role of SNORD116 microdeletion in the pathophysiology of addictive behavior in Prader-Willi syndrome. Mol Psychiatry. 2024 Sep;29(9):2742-2752. doi: 10.1038/s41380-024-02542-4. Epub 2024 Apr 2.

    PubMed 38561465

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Growth hormone (Genotonorm® or Omnitrope®), DEXA, blood tests, H.G.P.O, osseous age. and biopsy in people with Prader-Willi Syndrome and Growth Hormone Deficiency, run by University Hospital, Toulouse at 26 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 24 Sep 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 26 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Measure of the circulating rates of IGF-I under treatment. (time frame: Before starting treatment: baseline (J0)). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01298180, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2021.

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