Insulin-Like Growth Factor-1 DeficiencyPhase 2/3NCT00125190
Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With IGF-1 Deficiency
A Phase 2/3 study testing rhIGF-1 (mecasermin) for a period of 86 weeks in people with Insulin-Like Growth Factor-1 Deficiency and Growth Disorders, run by Ipsen at 1 site in United States.
Trial summary
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity. Read more about this study
- Who is it for?
- Ages 3 years and older
- Conditions the study is for
- Insulin-Like Growth Factor-1 Deficiency, Growth Disorders
- What is being tested?
- rhIGF-1 (mecasermin) for a period of 86 weeks
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2005to Jan 2009
- 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 3 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Chronological age ≥ 3
- Chronological age or bone age ≤ 12 for boys and ≤ 11 for girls
- Prepubertal at Visit 1
- Height SD score of < -2
- IGF-1 SD score of < -2
Who cannot join
- Prior treatment with GH, IGF-1, or other growth-influencing medications
- Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
- Chronic illness such as diabetes, cystic fibrosis, etc.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Controlled Study
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.
Read the detailed description
Growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).
The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.
- Being tested
- rhIGF-1 (mecasermin) for a period of 86 weeks
- Keywords
- Primary IGF-1 Deficiency, IGF-1
- Medical subject headings
- Growth Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhIGF-1 (mecasermin) for a period of 86 weeksDrug
Once a day rhIGF-1 injections
| Study group | Role | What they receive |
|---|---|---|
| rhIGF-1 QD | Experimental | Subjects received subcutaneous injection (SC) injections of rhIGF-1 once a day. Receives: Drug: rhIGF-1 (mecasermin) for a period of 86 weeks |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height Velocity From Pretreatment (Week 0) to Week 34
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward.
Measured: Pretreatment to Week 34
Height Velocity From Week 34 to 86
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward.
Measured: Week 34 to 86
Secondary outcomes (5)
Change in Height SD Score From Pretreatment to Week 34
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. The SD score is calculated as the subject value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child.
Measured: Pretreatment and Week 34
Change in Height SD Score From Pretreatment to Week 86
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. Subjects were repositioned between each measurement. The SD score is calculated as the patient value minus the mean divided by the standard deviation. The mean and the standard deviation vary depending on the age and sex of the child.
Measured: Pretreatment and Week 86
Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age
Plain X-rays of the left hand and wrist were exposed for bone age appraisal. The films were sent to a central facility for standardized evaluation.
Measured: Pretreatment to Week 86
Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86
Growth factor panels for measuring IGFBP-1, IGFBP-2 and IGFBP-3 were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile.
Measured: Pretreatment and Week 86
Percent Change in Serum Concentration of ALS From Pretreatment to Week 86
Growth factor panels for measuring ALS were evaluated from screening and at each study visit up to Week 86. Inter-quartile range (Q1-Q3) is 10th to 90th percentile.
Measured: Pretreatment and Week 86
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2005 (estimated)
- Study startJul 2005
- Primary completionJan 2009
- Study completionJan 2009
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 Insulin-Like Growth Factor-1 DeficiencyStudies 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
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Study Director, M.D., Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00125190
- Sponsor's protocol ID
- MS308
- Other IDs
- 2019-001095-11, 2019
- Status last verified
- Jul 2020
- First submitted
- 27 Jul 2005
- Last updated
- 27 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Short Stature Associated With Primary IGF-1 Deficiency: A Multi-Center, Open Label, Concentration-Controlled Study
- ClinicalTrials.gov ID
- NCT00125190
- Acronym / sponsor ID
- MS308
- Keywords
- Primary IGF-1 Deficiency, IGF-1
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- Jul 2005
- Primary completion
- Jan 2009 (actual)
- Study completion
- Jan 2009 (actual)
- First posted
- 29 Jul 2005 (estimated)
- Results
- Posted 15 Apr 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.
Detailed description
Growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in growth hormone (GH) action. In this protocol, primary IGFD is defined as short stature (<-2 standard deviations [SDs] below the mean for age and gender), and abnormal serum IGF-1 (<-2 SDs below the mean for age and gender).
The trial is an open-label, concentration-controlled trial conducted at up to 20 centers throughout the United States.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rhIGF-1 QD | Experimental | Drug: rhIGF-1 (mecasermin) for a period of 86 weeksSubjects received subcutaneous injection (SC) injections of rhIGF-1 once a day. |
Who may qualify
- Ages
- 3 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Chronological age ≥ 3
- Chronological age or bone age ≤ 12 for boys and ≤ 11 for girls
- Prepubertal at Visit 1
- Height SD score of < -2
- IGF-1 SD score of < -2
Key exclusion criteria (3 listed)
- Prior treatment with GH, IGF-1, or other growth-influencing medications
- Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
- Chronic illness such as diabetes, cystic fibrosis, etc.
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 |
|---|---|---|
| rhIGF-1 (mecasermin) for a period of 86 weeksOnce a day rhIGF-1 injections | Drug | rhIGF-1 QD |
Primary outcome
Height Velocity From Pretreatment (Week 0) to Week 34
Time frame: Pretreatment to Week 34
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 34 heights were imputed using the last height SD score carried forward.
Height Velocity From Week 34 to 86
Time frame: Week 34 to 86
Height was measured standing without shoes as the average of three measurements by the same observer using identical technique with a Harpenden or other wall mounted stadiometer. The subject was repositioned between each measurement. Height velocity during an interval of time is defined as the change in height during the time interval divided by the duration of the time interval. Missing Week 86 heights were imputed using the last height SD score carried forward.
5 secondary outcomes
Change in Height SD Score From Pretreatment to Week 34
Time frame: Pretreatment and Week 34
Change in Height SD Score From Pretreatment to Week 86
Time frame: Pretreatment and Week 86
Change in Bone Age From Pretreatment to Week 86 Minus Change in Chronological Age
Time frame: Pretreatment to Week 86
Percent Change in Serum Concentration of IGFBP-1, IGFBP-2 and IGFBP-3 From Pretreatment to Week 86
Time frame: Pretreatment and Week 86
Percent Change in Serum Concentration of ALS From Pretreatment to Week 86
Time frame: Pretreatment and Week 86
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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 Study Director, M.D. 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 rhIGF-1 (mecasermin) for a period of 86 weeks in people with Insulin-Like Growth Factor-1 Deficiency and Growth Disorders, 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 5 inclusion and 3 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 27 Jul 2020. 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 15 Apr 2010). 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?
Height Velocity From Pretreatment (Week 0) to Week 34 (time frame: Pretreatment to Week 34). 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?
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 27 Jul 2020.