Growth DisordersPhase 3NCT00125164
Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
A Phase 3 study testing rhIGF-1 (mecasermin, Tercica, Inc.) in people with Growth Disorders and Insulin-Like Growth Factor-1 Deficiency, run by Ipsen at 1 site in United States.
Trial summary
This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Read more about this study
- Who is it for?
- Ages 3 to 12 years
- Conditions the study is for
- Growth Disorders, Insulin-Like Growth Factor-1 Deficiency
- What is being tested?
- rhIGF-1 (mecasermin, Tercica, Inc.)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 137 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2004to Jul 2008
- 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 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
- Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
- Prepubertal
- Height SD score of < -2
- IGF-1 SD score of < -2
Who cannot join
- Prior treatment with rhGH, rhIGF-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.
United States1 site
Ipsen (formerly Tercica)
Brisbane, California, 94005
About this study
Official title: Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Prepubertal Children With Growth Failure Associated With Primary IGF-1 Deficiency: A Phase 3, Randomized, Open Label, Observation-Controlled, Multicenter, Parallel-Dose Comparison Trial
This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.
Read the detailed description
Prepubertal 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 GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.
This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.
- Being tested
- rhIGF-1 (mecasermin, Tercica, Inc.)
- Keywords
- Primary IGF-1 Deficiency
- 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, Tercica, Inc.)Drug
Twice Daily Injection
| Study group | Role | What they receive |
|---|---|---|
| Untreated | No intervention | Observational Group |
| 40 μg/kg BID (twice daily dosing) | Experimental | Injection of rhIGF-1 40 μg/kg BID. Per protocol amendment these subjects were reassigned to receive 120 μg/kg BID. Due to the dose change, the efficacy results for these subjects were analysed in a separate subanalysis. For all outcome measures, mean and standard deviations were not calculated for this arm. Receives: Drug: rhIGF-1 (mecasermin, Tercica, Inc.) |
| 80 μg/kg BID (twice daily dosing) | Experimental | Injection of rhIGF-1 80 μg/kg BID Receives: Drug: rhIGF-1 (mecasermin, Tercica, Inc.) |
| 120 μg/kg BID (twice daily dosing) | Experimental | Injection of rhIGF-1 120 μg/kg BID Receives: Drug: rhIGF-1 (mecasermin, Tercica, Inc.) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height Velocity During the First Year - Intent to Treat (ITT)Population
Height to be 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. Reposition subject between each measurement.
Measured: Measured at baseline and at one year
Secondary outcomes (8)
Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population
Height to be 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. Reposition subject between each measurement. Please note that Standard Deviation (SD) Score is a term used in growth studies. 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: Measured at baseline and at one year
Changes in Bone Age From Baseline to One Year
Plain X-rays of the left hand and wrist exposed for bone age appraisal. The films are sent to a central facility for standardized evaluation.
Measured: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year
Blood sample was collected while subject is in a fasting state for measuring the level of IGF-1 in the growth factor panel.
Measured: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year
Blood sample was collected for measuring the level of IGF-2 in the growth factor panel.
Measured: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year
Blood sample was collected for measuring the level of insulin-like growth factor binding protein-2 (IGFBP-2) in the growth factor panel.
Measured: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year
Blood sample was collected while subject is in a fasting state for measuring the level of IGFBP-3 in the growth factor panel.
Measured: Measured at baseline and at one year
IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Blood drawn at Study Day 1, followed by 7 days of rhGH daily dosing at 0.05 mg/kg of body weight. Additional blood draw at Study Day 7.
Measured: Study Day 1 and Day 7
IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Blood drawn at Study Day 1, followed by 7 days of rhGH daily dosing at 0.05 mg/kg of body weight. Additional blood draw at Study Day 7.
Measured: Study Day 1 and Day 7
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 startMar 2004
- Primary completionJul 2008
- Study completionJul 2008
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 Growth DisordersStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 137 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00125164
- Sponsor's protocol ID
- MS301
- Other IDs
- 2019-001020-36, 2019
- Status last verified
- Sep 2019
- First submitted
- 27 Jul 2005
- Last updated
- 18 Sep 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2019. 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 Prepubertal Children With Growth Failure Associated With Primary IGF-1 Deficiency: A Phase 3, Randomized, Open Label, Observation-Controlled, Multicenter, Parallel-Dose Comparison Trial
- ClinicalTrials.gov ID
- NCT00125164
- Acronym / sponsor ID
- MS301
- Keywords
- Primary IGF-1 Deficiency
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 137 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- Mar 2004
- Primary completion
- Jul 2008 (actual)
- Study completion
- Jul 2008 (actual)
- First posted
- 29 Jul 2005 (estimated)
- Results
- Posted 30 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 determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.
Detailed description
Prepubertal 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 GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score[SDS]<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS <-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.
This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Untreated | No intervention | –Observational Group |
| 40 μg/kg BID (twice daily dosing) | Experimental | Drug: rhIGF-1 (mecasermin, Tercica, Inc.)Injection of rhIGF-1 40 μg/kg BID. Per protocol amendment these subjects were reassigned to receive 120 μg/kg BID. Due to the dose change, the efficacy results for these subjects were analysed in a separate subanalysis. For all outcome measures, mean and standard deviations were not calculated for this arm. |
| 80 μg/kg BID (twice daily dosing) | Experimental | Drug: rhIGF-1 (mecasermin, Tercica, Inc.)Injection of rhIGF-1 80 μg/kg BID |
| 120 μg/kg BID (twice daily dosing) | Experimental | Drug: rhIGF-1 (mecasermin, Tercica, Inc.)Injection of rhIGF-1 120 μg/kg BID |
Who may qualify
- Ages
- 3 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
- Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
- Prepubertal
- Height SD score of < -2
- IGF-1 SD score of < -2
Key exclusion criteria (3 listed)
- Prior treatment with rhGH, rhIGF-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, Tercica, Inc.)Twice Daily Injection | Drug | 120 μg/kg BID (twice daily dosing), 40 μg/kg BID (twice daily dosing), 80 μg/kg BID (twice daily dosing) |
Primary outcome
Height Velocity During the First Year - Intent to Treat (ITT)Population
Time frame: Measured at baseline and at one year
Height to be 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. Reposition subject between each measurement.
8 secondary outcomes
Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population
Time frame: Measured at baseline and at one year
Changes in Bone Age From Baseline to One Year
Time frame: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year
Time frame: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year
Time frame: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year
Time frame: Measured at baseline and at one year
Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year
Time frame: Measured at baseline and at one year
IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Time frame: Study Day 1 and Day 7
IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
Time frame: Study Day 1 and Day 7
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Growth Disorders
Recruiting
Short-Term Reduced Dietary Protein in ER+/HER2- Breast Cancer Patients During Neoadjuvant Chemotherapy
Breast Cancer, Hormone Receptor Positive / HER2-negative Breast Cancer
RecruitingPhase 2
Study of Patritumab Deruxtecan Plus Pembrolizumab With Other Anticancer Agents in Participants With High-Risk Early-Stage Triple-Negative or Hormone Receptor-Low Positive/HER-2 Negative Breast Cancer (MK-1022-010, HERTHENA-Breast-03)
Breast Neoplasms, Breast Cancer
RecruitingPhase 1/2
A Study of VRN110755 in Patients With EGFR-Mutant Non-Small Cell Lung Cancer
EGFR-Mutant Non-Small Cell Lung Cancer
Active, not recruitingPhase 2
Safety, Preliminary Effectiveness of BNT327, an Investigational Therapy for Patients With Small-cell Lung Cancer in Combination With Chemotherapy
Extensive-stage Small-cell Lung Cancer, Small-cell Lung Cancer
Not yet recruitingPhase 3
Phase 3 Study to Evaluate the Efficacy and Safety of Oral Minoxidil (N1087) in Men With Androgenetic Alopecia.
Androgenetic Alopecia, Androgenetic Alopecia (AGA)
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 3 study testing rhIGF-1 (mecasermin, Tercica, Inc.) in people with Growth Disorders and Insulin-Like Growth Factor-1 Deficiency, 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 18 Sep 2019. 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 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 30 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 During the First Year - Intent to Treat (ITT)Population (time frame: Measured at baseline and at one year). 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 18 Sep 2019.