Growth Hormone Insensitivity SyndromePhase 2/3NCT00571727
Long-Term Treatment With rhIGF-1 in GHIS
A Phase 2/3 study testing mecasermin in people with Growth Hormone Insensitivity Syndrome, run by Ipsen at 1 site in United States.
Trial summary
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD). Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Growth Hormone Insensitivity Syndrome
- What is being tested?
- mecasermin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- 1 siteUnited States
- Study dates
- 20 May 1991to 15 Dec 2011
- Phase
- Phase 2/3
- 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 2 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Height <-2SD for age and gender
- IGF-1 <-2SD for age and gender
- Evidence of GH resistance
Who cannot join
- closed epiphyses
- prior active malignancy
- major organ disfunction
- treatment with medications that would diminish growth
- clinically significant cardiac abnormalities
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Study of Long-Term Human Recombinant Insulin-Like Growth Factor-1 (rhIGF-1) in Children With Short Stature Due to Growth Hormone Insensitivity Syndrome (GHIS)
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
- Conditions
- Growth Hormone Insensitivity Syndrome
- Being tested
- mecasermin
- Keywords
- growth, insulin like growth factor, Laron syndrome
- Medical subject headings
- Laron Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mecaserminDrug
Also called Increlex
injections BID of rhIGF-1, mecasermin
| Study group | Role | What they receive |
|---|---|---|
| mecasermin, injections BID of rhIGF-1 | Experimental | Not described Receives: Drug: mecasermin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Height Velocity Up to 12 Years
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Measured: Baseline (Pre-dose) and up to 12 years
Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Measured: Baseline (Pre-dose) and 1 year
Secondary outcomes (3)
Height Velocity Standard Deviation Score Up to 12 Years
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome.
Measured: Baseline (Pre-dose) and up to 12 years
Height Standard Deviation Score Up to 12 Years
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome.
Measured: Baseline (Pre-dose) and up to 12 years
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Measured: Baseline (Pre-dose) and up to 19 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2007 (estimated)
- Study start20 May 1991
- Primary completion15 Dec 2011
- Study completion15 Dec 2011
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 Insensitivity SyndromeStudies of the same condition, open and under way.
- More trials run by IpsenThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 92 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University of Oklahoma
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00571727
- Sponsor's protocol ID
- Study 1419
- Status last verified
- Jul 2023
- First submitted
- 10 Dec 2007
- Last updated
- 6 Jul 2023
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
Publications
Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE; GH Insensitivity Syndrome Collaborative Group. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007 Mar;92(3):902-10. doi: 10.1210/jc.2006-1610. Epub 2006 Dec 27.
PubMed 17192294
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Long-Term Human Recombinant Insulin-Like Growth Factor-1 (rhIGF-1) in Children With Short Stature Due to Growth Hormone Insensitivity Syndrome (GHIS)
- ClinicalTrials.gov ID
- NCT00571727
- Acronym / sponsor ID
- Study 1419
- Conditions
- Growth Hormone Insensitivity Syndrome
- Keywords
- growth, insulin like growth factor, Laron syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 92 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Collaborators
- University of Oklahoma
- Study start
- 20 May 1991 (actual)
- Primary completion
- 15 Dec 2011 (actual)
- Study completion
- 15 Dec 2011 (actual)
- First posted
- 12 Dec 2007 (estimated)
- Results
- Posted 6 Jul 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| mecasermin, injections BID of rhIGF-1 | Experimental | Drug: mecasermin |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Height <-2SD for age and gender
- IGF-1 <-2SD for age and gender
- Evidence of GH resistance
Key exclusion criteria (5 listed)
- closed epiphyses
- prior active malignancy
- major organ disfunction
- treatment with medications that would diminish growth
- clinically significant cardiac abnormalities
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 |
|---|---|---|
| mecaserminAlso: Increlexinjections BID of rhIGF-1, mecasermin | Drug | mecasermin, injections BID of rhIGF-1 |
Primary outcome
Annualized Height Velocity Up to 12 Years
Time frame: Baseline (Pre-dose) and up to 12 years
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment
Time frame: Baseline (Pre-dose) and 1 year
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
3 secondary outcomes
Height Velocity Standard Deviation Score Up to 12 Years
Time frame: Baseline (Pre-dose) and up to 12 years
Height Standard Deviation Score Up to 12 Years
Time frame: Baseline (Pre-dose) and up to 12 years
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height
Time frame: Baseline (Pre-dose) and up to 19 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 17192294Chernausek SD, Backeljauw PF, Frane J, Kuntze J, Underwood LE; GH Insensitivity Syndrome Collaborative Group. Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity. J Clin Endocrinol Metab. 2007 Mar;92(3):902-10. doi: 10.1210/jc.2006-1610. Epub 2006 Dec 27.
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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.
Ipsen Medical Director study director
Ipsen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing mecasermin in people with Growth Hormone Insensitivity Syndrome, run by Ipsen at 1 site 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 3 inclusion and 5 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 6 Jul 2023. 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 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 6 Jul 2023). 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?
Annualized Height Velocity Up to 12 Years (time frame: Baseline (Pre-dose) and up to 12 years). 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 NCT00571727, 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 6 Jul 2023; AzTrials last refreshed its copy on 6 Nov 2026.