Idiopathic Short StaturePhase 3NCT00396097

Individualized, Target-Driven Treatment Of Children With Idiopathic Short Stature

A Phase 3 study testing Genotropin in people with Idiopathic Short Stature, run by Pfizer at 43 sites in United States.

Completed

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

Official record

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Trial summary

Who is it for?: Ages 3 to 10 years
Conditions the study is for: Idiopathic Short Stature
What is being tested?: Genotropin
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 316 participants
Where: 43 sitesUnited States
Study dates: Dec 2006to Aug 2012
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 3 to 10 years
All sexes
No healthy volunteers
Child

Who may be able to join

Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females
Naive to Growth Hormone treatment

Who cannot join

Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

Trial locations and contacts

43 sites in 1 country.

City, postcode or hospital

43 sites in 1 country

United States43 sites

Childrens Hospital Colorado
Aurora, Colorado, 80045
University of Maryland Baltimore
Baltimore, Maryland, 21201
Johns Hopkins Pediatric Endocrinology
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Children's Hospital
Boston, Massachusetts, 02115
Maimonides Medical Center
Brooklyn, New York, 11219
Women & Children's Hospital of Buffalo
Buffalo, New York, 14222
Medical University of South Carolina
Charleston, South Carolina, 29425
Memorial Pediatric Specialty Clinic
Colorado Springs, Colorado, 80909
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205
Central Ohio Pediatric Endocrinology/Diabetes Services (COPEDS)
Columbus, Ohio, 43235
Ellen Sher, MD PA
Dallas, Texas, 75230
Children's Medical Center
Dallas, Texas, 75235
Cooks Children Medical Center/Dept. of Pediatric Endocrinology
Fort Worth, Texas, 76104
Pediatric Endocrine Associates
Greenwood Village, Colorado, 80111
Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
Children's Mercy Hospital
Kansas City, Missouri, 64108
The Steven and Alexandra Cohen Children's Medical Center of New York (CCMC)
Lake Success, New York, 11042
Arkansas Children's Hospital
Little Rock, Alaska, 72202
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
Saint Barnabas Ambulatory Care Center
Livingston, New Jersey, 07039
Pediatric Endocrine Associates at The Longmont Clinic
Longmont, Colorado, 80501
Children's Hospital of Los Angeles
Los Angeles, California, 90027
The Endocrine Clinic P.C.
Memphis, Tennessee, 38119
Miller School Of Medicine, University of Miami/Jackson Memorial Medical Center
Miami, Florida, 33136
Pediatric Endocrinology Associates
Miami, Florida, 33155
Winthrop University Hospital
Mineola, New York, 11501
Morristown Memorial Hospital
Morristown, New Jersey, 07962
Vanderbilt University
Nashville, Tennessee, 37232
Mount Sinai School of Medicine
New York, New York, 10029-6574
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Nemours Children's Clinic
Orlando, Florida, 32806
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
Pediatric Alliance, PC
Pittsburgh, Pennsylvania, 15218
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Rady Children's Hospital - San Diego
San Diego, California, 32123-4282
Pediatric Endocrinology of San Diego Medical Group Incorporated
San Diego, California, 92123
Diabetes & Endocrine Center for Children & Young Adults at Phelps Memorial Hospital
Sleepy Hollow, New York, 10591
Baystate Medical Center Children's Hospital
Springfield, Massachusetts, 01199
Children's Endocrine Care of St. Louis, LLC
St Louis, Missouri, 63141
Nancy Wright, MD
Tallahassee, Florida, 32308
Children's Hospital at Montefiore
The Bronx, New York, 10467-2490

Other trials in United States

About this study

Official title: A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regimen To Standard Dosing Of Genotropin

To demonstrate that an individualized, formula-based Genotropin regimen for children with Idiopathic Short Stature will lead to a targeted height gain (to reach the target of 10th percentile (%), or -1.3 SDS) during 24 months of treatment. The endpoint at 4 years is to explore treatment efficiency over four years of two formula-based dose regimens (sub-arms) compared to standard treatment

Conditions: Idiopathic Short Stature
Being tested: Genotropin
Keywords: ISS, human growth hormone

What participants receive

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

GenotropinDrug
Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.
GenotropinDrug
Compare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.

Study groups in NCT00396097
Study group	Role	What they receive
Standard	Active comparator	Standard daily HGH treatment Receives: Drug: Genotropin
Formula-based	Active comparator	Formula-based dose regimen Receives: Drug: Genotropin

What is being measured

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

Absolute On-target Difference (AOTD) at 24 Months
This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).
Measured: 2 years

Secondary outcomes (5)

Variability of Height SDS at 24 Months
The continuous endpoint of variability of height SDS at 24 months was defined as the SD of the 24 month height SDS.
Measured: 2 years
Time Cost (Months Until >= -2 SDS)
Time cost was defined as the number of months needed until height SDS was within the normal limit (ie, >= -2SDS).
Measured: 2 years
Computed Cost of Height Gain at 48 Months
The computed cost of height gain was defined as the amount of drug used relative to the observed height-gain, in terms of mg/cm, this was calculated at Month 48.
Measured: 4 years
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months
The estimated cost of long-term height gain until FAH was calculated.
Measured: 4 years
Change From Baseline in Height SDS at 48 Months.
Change in height SDS was measured at 48 months.
Measured: 4 years

Timeline

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

Dec 2006Aug 2012

First posted6 Nov 2006 (estimated)
Study startDec 2006
Primary completionAug 2012
Study completionAug 2012

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.
Other trials for Idiopathic Short StatureStudies 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: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 316 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00396097
Sponsor's protocol ID: A6281280
Status last verified: Mar 2016
First submitted: 2 Nov 2006
Last updated: 5 Apr 2016
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

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
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Recruiting
A Clinical Study of Long-Term Administration of Inpegsomatropin Injection in Participants With Idiopathic Short Stature Who Have Completed Phase Ⅲ Clinical Trials
Idiopathic Short Stature
Not yet recruitingPhase 3
Phase 3 Study to Evaluate the Efficacy and Safety of Human Growth Hormone Injection (Sinotropin AQ) for the Treatment of Children With Idiopathic Short Stature
Idiopathic Short Stature (ISS)
Active, not recruitingPhase 3
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
SGA, Turner Syndrome

All Idiopathic Short Stature trials

Study at a glance

From the registry record, last updated by the sponsor on 5 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Four Year Open Label Multi Center Randomized Two Arm Study Of Genotropin In Idiopathic Short Stature Patients: Comparing An Individualized, Target Driven Treatment Regimen To Standard Dosing Of Genotropin
ClinicalTrials.gov ID: NCT00396097
Acronym / sponsor ID: A6281280
Conditions: Idiopathic Short Stature
Keywords: ISS, human growth hormone
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 316 participants (actual)
Lead sponsor: PfizerIndustry (pharma, biotech, devices)
Study start: Dec 2006
Primary completion: Aug 2012 (actual)
Study completion: Aug 2012 (actual)
First posted: 6 Nov 2006 (estimated)
Results: Posted 13 Nov 2013

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: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Standard	Active comparator	Drug: GenotropinStandard daily HGH treatment
Formula-based	Active comparator	Drug: GenotropinFormula-based dose regimen

Who may qualify

Ages: 3 to 10 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

Prepubertal children with bone ages between 3 and 10 years of age for males and 3 and 9 years of age for females
Naive to Growth Hormone treatment

Key exclusion criteria (1 listed)

Abnormal karyotype. Small Gestational Age and Skeletal dysplasia.

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
GenotropinCompare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.	Drug	Standard
GenotropinCompare daily injections of formula-based HGH treatment to daily injections of standard HGH treatment in subjects with Idiopathic Short Stature over 24 months period followed by an exploratory 24 months period.	Drug	Formula-based

Primary outcome

Absolute On-target Difference (AOTD) at 24 Months
Time frame: 2 years
This was defined as an absolute difference between the 24-month height standard deviation score (SDS) and targeted 24-month height SDS (10th percentile (%), or -1.3 SDS). SDS indicates how similar the participant was to the reference population. These were calculated using 2000 Center for the Disease Control (CDC) growth reference tables (by age and gender).

5 secondary outcomes

Variability of Height SDS at 24 Months
Time frame: 2 years
Time Cost (Months Until >= -2 SDS)
Time frame: 2 years
Computed Cost of Height Gain at 48 Months
Time frame: 4 years
Estimated Cost of Height Gain Estimated Until Full Adult Height (FAH) at 48 Months
Time frame: 4 years
Change From Baseline in Height SDS at 48 Months.
Time frame: 4 years

Results and safety

Results posted to the registry on 13 Nov 2013, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Standard Dose Arm	114	88
Individualized Dose Arm	202	156

Primary outcome: Absolute On-target Difference (AOTD) at 24 Months

Time frame: 2 years. Mean (Standard Deviation). Unit: Standard Deviation Score (SDS)

Measure	Standard Dose Arm n = 114	Individualized Dose Arm n = 202
Value	0.603 (0.2948)	0.625 (0.3003)

Standard Dose Arm vs Individualized Dose Arm: Mean Difference (Final Values): -0.006, p = 0.5762, ANCOVA

Population analysed: The Full Analysis Set (FAS) included all randomized subjects who received at least 1 dose of study treatment and had at least 1 post-baseline height SDS value available. Last observation carried forward (LOCF) rule was applied to impute Month 24 missing height SDS data.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: 4 years. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Standard Dose Arm	13 of 118 (11%)	103 of 118 (87.3%)	Not reported
Individualized Dose Arm	7 of 198 (3.5%)	165 of 198 (83.3%)	Not reported

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2016.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
To obtain contact information for a study center near you, click here.Link provided by the sponsor.

Related trials for Idiopathic Short Stature

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
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Recruiting
A Clinical Study of Long-Term Administration of Inpegsomatropin Injection in Participants With Idiopathic Short Stature Who Have Completed Phase Ⅲ Clinical Trials
Idiopathic Short Stature
Not yet recruitingPhase 3
Phase 3 Study to Evaluate the Efficacy and Safety of Human Growth Hormone Injection (Sinotropin AQ) for the Treatment of Children With Idiopathic Short Stature
Idiopathic Short Stature (ISS)
Active, not recruitingPhase 3
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
SGA, Turner Syndrome

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

43 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 Genotropin in people with Idiopathic Short Stature, run by Pfizer at 43 sites in United States.

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 2 inclusion and 1 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 5 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 43 sites in United States. 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 13 Nov 2013). 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?

Absolute On-target Difference (AOTD) at 24 Months (time frame: 2 years). 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 NCT00396097.

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 5 Apr 2016.