Growth Hormone DeficiencyPhase 3NCT00163189

Evolution Of Growth Rate In Children With Growth Retardation Due to Glucocorticosteroid Therapy And Treated By Genotonorm

A Phase 3 study testing Somatropin in people with Growth Hormone Deficiency, run by Pfizer at 19 sites in France.

Completed

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

Trial summary

To estimate the evolution of height and growth rate over 5 years of growth hormone (GH) treatment To estimate the prognostic factors of growth rate with GH treatment To confirm the good clinical and biological safety of GH treatment in such children Read more about this study

Who is it for?: Any age
Conditions the study is for: Growth Hormone Deficiency
What is being tested?: Somatropin
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 98 participants
Where: 19 sitesFrance
Study dates: Jan 2005to Dec 2014
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Glucocorticosteroid treatment for 12 months at least
Bone age < 15 years for a boy and < 13 years for a girl
Child measured height < - 2 SD, Child currently treated by GH

Who cannot join

Known diabetes (type 1 or type 2)
A previous history of intolerance or hypersensitivity to the study drug or to drugs with similar chemical structures

Trial locations and contacts

19 sites in 1 country.

City, postcode or hospital

19 sites in 1 country

France19 sites

Hôpital Nord
Amiens, 80030
Centre Hospitalier Intercommunal d'Annemasse-Bonneville, Service de Pédiatrie et Néonatologie
Annemasse, 74107
Hôpital Saint Jacques
Besançon, 25030
Groupe hospitalier Est- Hôpital Femme, Mère, Enfant
Bron, 69677
Groupe hospitalier Est-Hôpital Femme, Mère, Enfant
Bron, 69677
CHU d'Estaing
Clermont-Ferrand, 63003
CHU de Grenoble, Hôpital Couple enfant.
Grenoble, 38043
CHU Timone Enfants
Marseille, 13385
Hôpital Arnaud De Villeneuve
Montpellier, 34059
CHU de Nantes, Hôpital Mère Enfant
Nantes, 44093
Hôpital Lenval
Nice, 06200
Hôpital Robert Debré
Paris, 75019
Hôpital Armand Trousseau
Paris, 75571
Groupe Hospitalier Necker - Enfants Malades
Paris, 75743
Centre de Perharidy
Roscoff, 29684
CHU Charles Nicolle
Rouen, 76031
Service de Pédiatrie- Centre Hospitalier Intercommunal
Saint-Germain-en-Laye, 78105
Hôpital des Enfants
Toulouse, 31059
CHU Tours - Centre Pediatrique Gatien de Cocheville
Tours, 37044

Other trials in France

About this study

Official title: Evolution Of Growth Rate In Children With Growth Retardation Related To Long-term Corticotherapy And Treated By Genotonorm

Conditions: Growth Hormone Deficiency
Being tested: Somatropin
Medical subject headings: Dwarfism, Pituitary

What participants receive

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

SomatropinDrug
Liquid, daily, during 3 years and extended to 5 years Dosage: 0,46 mg/kg/week - the maximum dose should not exceed 50 µg/kg/day

Study groups in NCT00163189
Study group	Role	What they receive
Somatropin	Experimental	Not described Receives: Drug: Somatropin

What is being measured

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

Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.
Measured: Baseline, Month 36
Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.
Measured: Baseline, Month 36

Secondary outcomes (11)

Mean Height
The standing height measurements were performed using a wall mounted device (example Harpenden Stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.
Measured: Baseline, Month 12, 24, 36, 48, 60
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
The standing height measurements were performed using a wall mounted device (example Harpenden Stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand.
Measured: Baseline, Month 12, 24, 36, 48, 60
Annual Growth Rate (AGR)
AGR at Yx was derived by subtracting AGR at baseline from Yx value. AGR was calculated each year and re scaled to 1 year if the interval between Yx and Y[x-1] was not 365 days, as long as a participant remained in the study. AGR at Yx was calculated using the previous height measurements (Y[x-1]) and height recorded at Yx (AGR Yx = [height Yx-height Y{x-1}] / ([date of Yx - date of Y{x-1}] /365.25). Yx refers to the value at particular timepoint x.
Measured: Baseline, Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Bone Age (BA)
GR SDS BA Yx = (GR Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. BA was estimated locally using an X-ray from the left wrist and hand.
Measured: Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Chronological Age (CA)
GR SDS CA Yx = (GR Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. GR in SDS was calculated using Sempe reference means and SD for GR. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.
Measured: Baseline, Month 12, 24, 36, 48, 60
Body Mass Index (BMI)
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m^2).
Measured: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height at Month 12, 24, 36, 48 and 60
The standing height measurements were performed using a wall mounted device (example Harpenden Stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded.
Measured: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, 24, 48 and 60
The standing height measurements were performed using a wall mounted device (example Harpenden Stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.
Measured: Baseline, Month 12, 24, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, 24, 36, 48 and 60
The standing height measurements were performed using a wall mounted device (example Harpenden Stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS BA Yx = (height Yx - reference mean for BA Yx) / reference SD for BA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. BA was estimated locally using an X-ray from the left wrist and hand.
Measured: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Bone Age (BA) at Month 12, 24, 36, 48 and 60
BA was estimated locally using an X-ray from the left wrist and hand.
Measured: Baseline, Month 12, 24, 36, 48, 60
Ratio of Bone Age (BA) to Chronological Age (CA)
BA was estimated locally using an X-ray from the left wrist and hand. CA at the date of corresponding X-ray (Date of X-ray - Date of birth)/365.25. Ratio of BA/CA at each annual study visit was calculated.
Measured: Baseline, Month 12, 24, 36, 48, 60

Timeline

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

Jan 2005Dec 2014

First posted13 Sep 2005 (estimated)
Study startJan 2005
Primary completionDec 2014
Study completionDec 2014

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
More trials run by PfizerThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 98 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00163189
Sponsor's protocol ID: A6281271
Other IDs: 2004-002992-17, 2004
Status last verified: Jun 2015
First submitted: 9 Sep 2005
Last updated: 21 Feb 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Basmaison O, Ranchin B, Zouater H, Robertson A, Gomez R, Koppiker N. Efficacy and safety of recombinant growth hormone treatment in children with growth retardation related to long-term glucocorticosteroid therapy. Ann Endocrinol (Paris). 2019 Sep;80(4):202-210. doi: 10.1016/j.ando.2019.02.001. Epub 2019 Mar 4.
PubMed 30910221

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age

All Growth Hormone Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 21 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Evolution Of Growth Rate In Children With Growth Retardation Related To Long-term Corticotherapy And Treated By Genotonorm
ClinicalTrials.gov ID: NCT00163189
Acronym / sponsor ID: A6281271
Conditions: Growth Hormone Deficiency
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 98 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Jan 2005
Primary completion: Dec 2014 (actual)
Study completion: Dec 2014 (actual)
First posted: 13 Sep 2005 (estimated)
Results: Posted 25 Jun 2015

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Somatropin	Experimental	Drug: Somatropin

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (3 listed)

Glucocorticosteroid treatment for 12 months at least
Bone age < 15 years for a boy and < 13 years for a girl
Child measured height < - 2 SD, Child currently treated by GH

Key exclusion criteria (2 listed)

Known diabetes (type 1 or type 2)
A previous history of intolerance or hypersensitivity to the study drug or to drugs with similar chemical structures

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SomatropinLiquid, daily, during 3 years and extended to 5 years Dosage: 0,46 mg/kg/week - the maximum dose should not exceed 50 µg/kg/day	Drug	Somatropin

Primary outcome

Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population
Time frame: Baseline, Month 36
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.
Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population
Time frame: Baseline, Month 36
Height was measured using a wall mounted device (example, Harpenden stadiometer). The standing height of the participant was measured two times and the mean of these measurements was recorded. Height SDS CA Yx = (height Yx - reference mean for CA Yx) / reference SD for CA Yx; Yx refers to the value at particular timepoint x. Height in SDS was calculated using Sempe reference means and SD for height. CA calculated as integer (Date of height measurement-Date of birth)/365.25*12.

11 secondary outcomes

Mean Height
Time frame: Baseline, Month 12, 24, 36, 48, 60
Mean Height Standard Deviation Score (SDS) for Bone Age (BA)
Time frame: Baseline, Month 12, 24, 36, 48, 60
Annual Growth Rate (AGR)
Time frame: Baseline, Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Bone Age (BA)
Time frame: Month 12, 24, 36, 48, 60
Growth Rate (GR) Standard Deviation Score (SDS) for Chronological Age (CA)
Time frame: Baseline, Month 12, 24, 36, 48, 60
Body Mass Index (BMI)
Time frame: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height at Month 12, 24, 36, 48 and 60
Time frame: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 12, 24, 48 and 60
Time frame: Baseline, Month 12, 24, 48, 60
Change From Baseline in Height Standard Deviation Score (SDS) for Bone Age (BA) at Month 12, 24, 36, 48 and 60
Time frame: Baseline, Month 12, 24, 36, 48, 60
Change From Baseline in Bone Age (BA) at Month 12, 24, 36, 48 and 60
Time frame: Baseline, Month 12, 24, 36, 48, 60
Ratio of Bone Age (BA) to Chronological Age (CA)
Time frame: Baseline, Month 12, 24, 36, 48, 60

Results and safety

Results posted to the registry on 25 Jun 2015, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Genotonorm	98	35

Primary outcome: Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population

Time frame: Baseline, Month 36. Mean (Standard Deviation). Unit: Standard Deviation Score (SDS)

Measure	Genotonorm n = 58
Baseline (n=58)	-2.91 (1.19)
Change at Month 36 (n=30)	0.80 (1.03)

Genotonorm: p <0.001, Student's paired t-test

Population analysed: Full analysis set (FAS) included all participants (excluding a site with GCP issues) who had received at least 1 injection of GH and had at least 1 post-Baseline measurement of height. Here "n" signifies those participants who were evaluable for the specified time point.

Primary outcome: Change From Baseline in Height Standard Deviation Score (SD) for Chronological Age (CA) at Month 36: Per Protocol (PP) Population

Time frame: Baseline, Month 36. Mean (Standard Deviation). Unit: SDS

Measure	Genotonorm n = 21
Baseline (n=21)	-2.86 (0.89)
Change at Month 36 (n=19)	0.81 (1.18)

Genotonorm: p = 0.008, Student's paired t-test

Population analysed: PP analysis set included all participants (excluding a site with GCP issues) who received at least 1 dose of GH, had at least 1 subsequent rating of height, no major protocol violation till first 3 years post initiation of treatment and total GH treatment duration of 36 months or more. "n"=participants evaluable at the specified time point.

11 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Genotonorm	45 of 98 (45.9%)	81 of 98 (82.7%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 30910221Basmaison O, Ranchin B, Zouater H, Robertson A, Gomez R, Koppiker N. Efficacy and safety of recombinant growth hormone treatment in children with growth retardation related to long-term glucocorticosteroid therapy. Ann Endocrinol (Paris). 2019 Sep;80(4):202-210. doi: 10.1016/j.ando.2019.02.001. Epub 2019 Mar 4.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

Related trials for Growth Hormone Deficiency

RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Pfizer CT.gov Call Center study director

Pfizer

19 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Somatropin in people with Growth Hormone Deficiency, run by Pfizer at 19 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 3 inclusion and 2 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 21 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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

Are results available for this study?

Yes. Results are posted in the registry (first posted 25 Jun 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Change From Baseline in Height Standard Deviation Score (SDS) for Chronological Age (CA) at Month 36: Full Analysis Population (time frame: Baseline, Month 36). The study lists 2 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 NCT00163189, which also lists 1 linked publication.

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 21 Feb 2024.