Idiopathic Short StaturePhase 4NCT01438801
Predictive Value of the Insulin-like Growth Factor-1 (IGF-1) Generation Test for the Growth Response to Growth Hormone Treatment (PRED-IGF)
A Phase 4 study testing Nutropin [Somatropin (rDNA origin) for injection] in people with Idiopathic Short Stature, run by Ipsen.
Trial summary
The evaluation of a standardized diagnostic test to predict the growth response in a 1 year trial with Growth Hormone (GH) treatment (carried out in the context of regular patient care) in non GH deficient short children with low serum insulin-like growth factor-1 (IGF-1). Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Idiopathic Short Stature
- What is being tested?
- Nutropin [Somatropin (rDNA origin) for injection]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- May 2013to 31 May 2013
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Age 2.0 - 9.0 years for females, 2.0 - 10.0 yrs for males.
- Prepubertal stage (Tanner 1 for breast (B1) in females, or Tanner 1 for genitals (G1) for males).
- Bone age < 10 'years' (males) or < 9 'years' (females) according to Greulich and Pyle. The bone age will be read by the pediatric endocrinologist responsible for the pre-study screening visit.
- Height SDS < -2.5 for ethnically adequate references. For children of Dutch or Western European origin the 1997 nation-wide references for Dutch children will be used. For children of Moroccan or Turkish origin, the respective reference charts will be used. For children of other ethnicities, the 1977 North American (NCHS/WHO) reference will be used, as these charts have been accepted by WHO as world-wide standard from the age of 5 years.
Who cannot join
- Has a history of hypersensitivity to growth hormone or phenol (conservative added to GH in NutropinAq), or drugs with a similar chemical structure.
- Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardise the subject's safety or decrease the chance of obtaining satisfactory data needed to achieve the objective(s) of the study.
- Has a birth weight and/or length below -2 SDS for Swedish reference charts. Patients will not be excluded due to an unknown birth weight or length.
- Has a known cause of short stature, or any significant concomitant disease that is likely to interfere with growth or with the study schedule/objectives, or is a known contraindication to GH treatment.
About this study
Official title: Predictive Value of Baseline and Stimulated Serum IGF-1 and IGFBP-3 During a Dose-escalation IGF-1 Generation Test for the 1 Year Growth Response to Growth Hormone (GH) Therapy in Short Children With Low IGF-1 and a Normal GH Peak in a Provocation Test
The evaluation of a standardized diagnostic test to predict the growth response in a 1 year trial with Growth Hormone (GH) treatment (carried out in the context of regular patient care) in non GH deficient short children with low serum insulin-like growth factor-1 (IGF-1).
- Conditions
- Idiopathic Short Stature
- Being tested
- Nutropin [Somatropin (rDNA origin) for injection]
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nutropin [Somatropin (rDNA origin) for injection]Drug
Doses in the diagnostic protocol are 0.7 and 1.4 mg/m2/day, injected by subcutaneous injection at bedtime for 2 weeks, divided by washout periods of preferably 4 weeks (accepted range 4-6 weeks). An additional period of 2 weeks on 2.8 mg/m2/day (after a washout period of 4-6 weeks) will be added if the IGF-I response on either dosage is insufficient (serum IGF-I SDS <0).
| Study group | Role | What they receive |
|---|---|---|
| NutropinAq | Experimental | Not described Receives: Drug: Nutropin [Somatropin (rDNA origin) for injection] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Peak serum IGF-1 level with GH (1.4 mg/m2/day) change from baseline.
Measured: Week 2
Secondary outcomes (7)
Delta IGF-1 SDS on 0.7 & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Delta insulin-like growth factor binding protein-3 (IGFBP-3) standard deviation score (SDS) on 0.7 & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Delta of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Delta of the ratio IGF-1:IGFBP-2 (insulin-like growth factor binding protein-2) SDS on 0.7 mg & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Peak IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Peak of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Peak of the ratio IGF-1:IGFBP-2 SDS on 0.7 mg & 1.4 mg GH/m2/day
Measured: Baseline, week 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2011 (estimated)
- Study startMay 2013 (estimated)
- Primary completion31 May 2013 (estimated)
- Study completion31 May 2013 (estimated)
Why it stopped: Participant enrollment was not feasible
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies 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 4
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, MD, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01438801
- Sponsor's protocol ID
- A-95-58035-017
- Other IDs
- 2010-019980-13
- Status last verified
- Aug 2019
- First submitted
- 24 Aug 2011
- Last updated
- 5 Aug 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Predictive Value of Baseline and Stimulated Serum IGF-1 and IGFBP-3 During a Dose-escalation IGF-1 Generation Test for the 1 Year Growth Response to Growth Hormone (GH) Therapy in Short Children With Low IGF-1 and a Normal GH Peak in a Provocation Test
- ClinicalTrials.gov ID
- NCT01438801
- Acronym / sponsor ID
- A-95-58035-017
- Conditions
- Idiopathic Short Stature
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Participant enrollment was not feasible
- Enrolment
- 0 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- May 2013 (estimated)
- Primary completion
- 31 May 2013 (estimated)
- Study completion
- 31 May 2013 (estimated)
- First posted
- 22 Sep 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The evaluation of a standardized diagnostic test to predict the growth response in a 1 year trial with Growth Hormone (GH) treatment (carried out in the context of regular patient care) in non GH deficient short children with low serum insulin-like growth factor-1 (IGF-1).
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NutropinAq | Experimental | Drug: Nutropin [Somatropin (rDNA origin) for injection] |
Who may qualify
- Ages
- 2 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Age 2.0 - 9.0 years for females, 2.0 - 10.0 yrs for males.
- Prepubertal stage (Tanner 1 for breast (B1) in females, or Tanner 1 for genitals (G1) for males).
- Bone age < 10 'years' (males) or < 9 'years' (females) according to Greulich and Pyle. The bone age will be read by the pediatric endocrinologist responsible for the pre-study screening visit.
- Height SDS < -2.5 for ethnically adequate references. For children of Dutch or Western European origin the 1997 nation-wide references for Dutch children will be used. For children of Moroccan or Turkish origin, the respective reference charts will be used. For children of other ethnicities, the 1977 North American (NCHS/WHO) reference will be used, as these charts have been accepted by WHO as world-wide standard from the age of 5 years.
Key exclusion criteria (4 listed)
- Has a history of hypersensitivity to growth hormone or phenol (conservative added to GH in NutropinAq), or drugs with a similar chemical structure.
- Has abnormal baseline findings, any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardise the subject's safety or decrease the chance of obtaining satisfactory data needed to achieve the objective(s) of the study.
- Has a birth weight and/or length below -2 SDS for Swedish reference charts. Patients will not be excluded due to an unknown birth weight or length.
- Has a known cause of short stature, or any significant concomitant disease that is likely to interfere with growth or with the study schedule/objectives, or is a known contraindication to GH treatment.
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 |
|---|---|---|
| Nutropin [Somatropin (rDNA origin) for injection]Doses in the diagnostic protocol are 0.7 and 1.4 mg/m2/day, injected by subcutaneous injection at bedtime for 2 weeks, divided by washout periods of preferably 4 weeks (accepted range 4-6 weeks). An additional period of 2 weeks on 2.8 mg/m2/day (after a washout period of 4-6 weeks) will be added if the IGF-I response on either dosage is insufficient (serum IGF-I SDS <0). | Drug | NutropinAq |
Primary outcome
Peak serum IGF-1 level with GH (1.4 mg/m2/day) change from baseline.
Time frame: Week 2
7 secondary outcomes
Delta IGF-1 SDS on 0.7 & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Delta insulin-like growth factor binding protein-3 (IGFBP-3) standard deviation score (SDS) on 0.7 & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Delta of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Delta of the ratio IGF-1:IGFBP-2 (insulin-like growth factor binding protein-2) SDS on 0.7 mg & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Peak IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Peak of the ratio IGF-1:IGFBP-3 SDS on 0.7 mg & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Peak of the ratio IGF-1:IGFBP-2 SDS on 0.7 mg & 1.4 mg GH/m2/day
Time frame: Baseline, week 2
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2019.
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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, MD study director
Ipsen
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Nutropin [Somatropin (rDNA origin) for injection] in people with Idiopathic Short Stature, run by Ipsen.
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 4 inclusion and 4 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: withdrawn, as of the sponsor's last update on 5 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Peak serum IGF-1 level with GH (1.4 mg/m2/day) change from baseline. (time frame: Week 2). 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 5 Aug 2019.