Endocrine System DiseasesPhase 3NCT00174291

Prevention of Growth Retardation by Early Treatment With Growth Hormone (GH) in Children With CJA Treated by Corticosteroid Therapy

A Phase 3 study testing Somatropin in people with Endocrine System Diseases, run by Pfizer at 1 site in France.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial summary

To evaluate the effect of increasing the growth hormone dose on the statural response To assess the value of early treatment during the course of arthritic disease by comparing the height acquired in the medium term by children in the two groups: treated from the start, or 1 year to 15 months after the diagnosis of… Read more about this study

Who is it for?: Ages 4 to 14 years
Conditions the study is for: Endocrine System Diseases
What is being tested?: Somatropin
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 21 participants
Where: 1 siteFrance
Study dates: Mar 2002to Oct 2011
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 4 to 14 years
All sexes
No healthy volunteers
Child

Who may be able to join

This study will be proposed to all patients previously included in study CTN 97-8129-016

Who cannot join

Discontinuation of corticosteroid therapy for more than a year during study CTN 97-8129-016
Patients withdrawing from the study prematurely

Trial locations and contacts

1 site in 1 country.

France1 site

Pfizer Investigational Site
Paris, 75019

Other trials in France

About this study

Official title: Prevention of Retarded Growth by Early Treatment With Recombinant Human Growth Factor Genotonorm (Registered) in Children With Systemic Forms of Chronic Juvenile Arthritis Receiving Long-term Corticosteroid Therapy. Extension of the Study Beyond Three Years

To evaluate the effect of increasing the growth hormone dose on the statural response
To assess the value of early treatment during the course of arthritic disease by comparing the height acquired in the medium term by children in the two groups: treated from the start, or 1 year to 15 months after the diagnosis of CJA, or treated for 4 years after the diagnosis

Read the detailed description

This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.

Conditions: Endocrine System Diseases
Being tested: Somatropin
Medical subject headings: Endocrine System Diseases

What participants receive

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

SomatropinDrug
Liquid, daily to final height Maximum Dosage: 50 µg/kg/day

Study groups in NCT00174291
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 Annual Rate of Growth Standard Deviation Score (SDS) at Year 3
Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, Year 3
Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Final Height
Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, final height (assessed up to Year 9.5)
Change From Baseline in Height Standard Deviation Score (SDS) at Year 3
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, Year 3
Change From Baseline in Height Standard Deviation Score (SDS) at Final Height
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, final height (assessed up to Year 9.5)
Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Year 3
Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, Year 3
Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Final Height
Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, final height (assessed up to Year 9.5)

Secondary outcomes (6)

Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Measured: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP3) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Measured: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Lean Mass and Fat Mass at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
Lean mass and fat mass: measurements of body composition assessed using Dual Energy X-ray Absorptiometry (DEXA) scan.
Measured: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
Change From Baseline in Bone Mineralization at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
Bone mineralization, an estimate of the amount of mineral (such as calcium) in the bone, was assessed using DEXA scan.
Measured: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
Change From Baseline in Weight Standard Deviation Score (SDS) at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Body weight was measured using a balance scale. Weight SDS was obtained by measuring the weight, subtracting age- and gender- appropriate mean weight and dividing the result by standard deviation of that mean (as obtained from age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Measured: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Corticosteroid Dose at 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Measured: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9

Timeline

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

Mar 2002Oct 2011

First posted15 Sep 2005 (estimated)
Study startMar 2002
Primary completionOct 2011
Study completionOct 2011

Why it stopped: See termination reason in detailed description.

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 Endocrine System DiseasesStudies 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: Non-randomised
Intervention model: Single group
Masking: None (open label)
Enrolment: 21 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00174291
Sponsor's protocol ID: 307-MET-9002-052
Other IDs: A6281024
Status last verified: Oct 2012
First submitted: 9 Sep 2005
Last updated: 4 Dec 2012
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

David H, Aupiais C, Louveau B, Quartier P, Jacqz-Aigrain E, Carel JC, Simon D. Growth Outcomes After GH Therapy of Patients Given Long-Term Corticosteroids for Juvenile Idiopathic Arthritis. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4578-4587. doi: 10.1210/jc.2017-01455.
PubMed 29029101

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?

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Study at a glance

From the registry record, last updated by the sponsor on 4 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Prevention of Retarded Growth by Early Treatment With Recombinant Human Growth Factor Genotonorm (Registered) in Children With Systemic Forms of Chronic Juvenile Arthritis Receiving Long-term Corticosteroid Therapy. Extension of the Study Beyond Three Years
ClinicalTrials.gov ID: NCT00174291
Acronym / sponsor ID: 307-MET-9002-052
Conditions: Endocrine System Diseases
Phase: Phase 3
Study type: Interventional
Recruitment status: TerminatedReason stopped: See termination reason in detailed description.
Enrolment: 21 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Mar 2002
Primary completion: Oct 2011 (actual)
Study completion: Oct 2011 (actual)
First posted: 15 Sep 2005 (estimated)
Results: Posted 4 Dec 2012

Clinical summary

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

To evaluate the effect of increasing the growth hormone dose on the statural response
To assess the value of early treatment during the course of arthritic disease by comparing the height acquired in the medium term by children in the two groups: treated from the start, or 1 year to 15 months after the diagnosis of CJA, or treated for 4 years after the diagnosis

Detailed description

This trial terminated on 10-Jun-2011 due to prolonged issues with drug accountability and data collection discrepancies. The decision to terminate was not based on any safety concerns.

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Somatropin	Experimental	Drug: Somatropin

Who may qualify

Ages: 4 to 14 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (1 listed)

This study will be proposed to all patients previously included in study CTN 97-8129-016

Key exclusion criteria (2 listed)

Discontinuation of corticosteroid therapy for more than a year during study CTN 97-8129-016
Patients withdrawing from the study prematurely

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 to final height Maximum Dosage: 50 µg/kg/day	Drug	Somatropin

Primary outcome

Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Year 3
Time frame: Baseline, Year 3
Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Final Height
Time frame: Baseline, final height (assessed up to Year 9.5)
Annual rate of growth SDS was obtained by measuring the annual growth rate, subtracting chronological age- and gender-appropriate mean annual growth rate and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Height Standard Deviation Score (SDS) at Year 3
Time frame: Baseline, Year 3
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Height Standard Deviation Score (SDS) at Final Height
Time frame: Baseline, final height (assessed up to Year 9.5)
Height was measured using a wall mounted device (example, Harpenden stadiometer). Height SDS was obtained by measuring the height, subtracting chronological age- and gender-appropriate mean height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Year 3
Time frame: Baseline, Year 3
Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.
Change From Baseline in Predicted Height Standard Deviation Score (SDS) at Final Height
Time frame: Baseline, final height (assessed up to Year 9.5)
Predicted height was calculated according to Greulich and Pyle using Bayley Pinneau method. Predicted height SDS was obtained by calculating the predicted height, subtracting chronological age- and gender-appropriate mean predicted height and dividing the result by standard deviation of that mean (as obtained from chronological age- and gender-specific population reference data). SDS indicated how many standard deviations higher (in case of positive SDS) or lower (in case of negative SDS) participant's value was relative to the mean of the reference population.

6 secondary outcomes

Change From Baseline in Insulin-like Growth Factor-1 (IGF-1) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Time frame: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Insulin-like Growth Factor Binding Protein 3 (IGFBP3) Concentration at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Time frame: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Lean Mass and Fat Mass at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
Time frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
Change From Baseline in Bone Mineralization at Year 1, 2, 3, 4, 5, 6, 7, 8 and 9
Time frame: Baseline, Year 1, 2, 3, 4, 5, 6, 7, 8, 9
Change From Baseline in Weight Standard Deviation Score (SDS) at Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Time frame: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9
Change From Baseline in Corticosteroid Dose at 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5 and 9
Time frame: Baseline, Year 0.5, 1, 1.5, 2, 2.5, 3, 3.5, 4, 4.5, 5, 5.5, 6, 6.5, 7, 7.5, 8, 8.5, 9

Results and safety

Results posted to the registry on 4 Dec 2012, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Somatropin (With Previous Somatropin Exposure)	11	6
Somatropin (Without Previous Somatropin Exposure)	10	4

Primary outcome: Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Year 3

Time frame: Baseline, Year 3. Median (Full Range). Unit: SDS

Measure	Somatropin (With Previous Somatropin Exposure) n = 6	Somatropin (Without Previous Somatropin Exposure) n = 9
Baseline	-0.21 (-3.0 to 4.5)	-2.96 (-6.4 to 1.5)
Change at Year 3	1.34 (-3.6 to 6.2)	3.52 (-5.1 to 11.2)

Population analysed: Full Analysis Set (FAS) included all participants who received at least 1 dose of study treatment and had at least 1 post-baseline height measurement. Here, N (number of participants analyzed) signifies those participants who were evaluable for this measure.

Primary outcome: Change From Baseline in Annual Rate of Growth Standard Deviation Score (SDS) at Final Height

Time frame: Baseline, final height (assessed up to Year 9.5)

Population analysed: Data was not analyzed because of change in planned analysis after early termination of the study.

Primary outcome: Change From Baseline in Height Standard Deviation Score (SDS) at Year 3

Time frame: Baseline, Year 3. Median (Full Range). Unit: SDS

Measure	Somatropin (With Previous Somatropin Exposure) n = 6	Somatropin (Without Previous Somatropin Exposure) n = 9
Baseline	-0.61 (-3.7 to 1.4)	-2.34 (-4.8 to -1.4)
Change at Year 3	0.73 (-2.4 to 2.6)	1.21 (-0.7 to 2.4)

Population analysed: FAS included all participants who received at least 1 dose of study treatment and had at least 1 post-baseline height measurement. Here, N (number of participants analyzed) signifies those participants who were evaluable for this measure.

Primary outcome: Change From Baseline in Height Standard Deviation Score (SDS) at Final Height

Time frame: Baseline, final height (assessed up to Year 9.5). Median (Full Range). Unit: SDS

Measure	Somatropin (With Previous Somatropin Exposure) n = 3	Somatropin (Without Previous Somatropin Exposure) n = 2
Baseline	0.96 (-3.7 to 2.3)	-0.94 (-1.4 to -0.5)
Change at Final Height	-0.43 (-0.8 to 0.5)	0.08 (-0.7 to 0.9)

6 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
Somatropin (With Previous Somatropin Exposure)	6 of 11 (54.5%)	11 of 11 (100%)	Not reported
Somatropin (Without Previous Somatropin Exposure)	2 of 10 (20%)	9 of 10 (90%)	Not reported

Limitations and caveats (from the sponsor): Data for bone mineral density (BMD), bone mineral content (BMC) and IGFBP3 were not analyzed because of change in planned analysis after the study was prematurely terminated due to Good Clinical Practice (GCP) non-compliance issues.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2012.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 29029101David H, Aupiais C, Louveau B, Quartier P, Jacqz-Aigrain E, Carel JC, Simon D. Growth Outcomes After GH Therapy of Patients Given Long-Term Corticosteroids for Juvenile Idiopathic Arthritis. J Clin Endocrinol Metab. 2017 Dec 1;102(12):4578-4587. doi: 10.1210/jc.2017-01455.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

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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

1 site 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 Endocrine System Diseases, run by Pfizer at 1 site 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 1 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: stopped early, as of the sponsor's last update on 4 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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 4 Dec 2012). 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 Annual Rate of Growth Standard Deviation Score (SDS) at Year 3 (time frame: Baseline, Year 3). The study lists 6 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 NCT00174291, 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 4 Dec 2012.