Noonan SyndromePhase 2NCT00351221
Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome
A Phase 2 study testing rhIGF-1/rhIGFBP-3 in people with Noonan Syndrome, run by Insmed Incorporated at 2 sites in United States.
Trial summary
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested as a possible cause of short stature seen in Noonan syndrome children. Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Noonan Syndrome
- What is being tested?
- rhIGF-1/rhIGFBP-3
- How many people
- 24 participants
- Where
- 2 sitesUnited States
- Study dates
- Start not givento not given
- Phase
- Phase 2
- 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 16 years
- All sexes
- Child
Who may be able to join
- A diagnosis of Noonan syndrome
- Height less than the 3rd percentile for age and sex (height SDS < -1.88)
- Basal IGF-I less than the mean for age and sex (IGF-I SDS < 0)
- Chronological age greater than 2 years
- Bone age ≤ 11 years for boys, and ≤ 10 years for girls
- Pre-pubertal
- Documented pre-treatment height velocity less than the mean for age and sex
Who cannot join
- Clinically significant diseases
- Chronic illnesses
- Prior treatment with rhIGF-1
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Columbus Children's Hospital
Columbus, Ohio, 43205
Schneider Children's Hospital
New Hyde Park, New York
About this study
Official title: A Phase 2, Open-Label, Multicenter, Clinical Trial to Evaluate the Pharmacokinetics, Safety and Efficacy of Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 in Children With Growth Failure Due to Noonan Syndrome
The trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested as a possible cause of short stature seen in Noonan syndrome children. Administration of our investigational product is intended to bypass the abnormalities in the GH-IGF axis, and hopefully improve body growth.
- Conditions
- Noonan Syndrome
- Being tested
- rhIGF-1/rhIGFBP-3
- Medical subject headings
- Noonan Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhIGF-1/rhIGFBP-3Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2006 (estimated)
Why it stopped: Terminated due to a patent legal settlement
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Noonan SyndromeStudies of the same condition, open and under way.
- 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 24 participants
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Kenneth Attie, MD, Study director, Insmed, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00351221
- Sponsor's protocol ID
- INMS-110-801
- Status last verified
- Feb 2007
- First submitted
- 11 Jul 2006
- Last updated
- 30 Mar 2007
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Multicenter, Clinical Trial to Evaluate the Pharmacokinetics, Safety and Efficacy of Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 in Children With Growth Failure Due to Noonan Syndrome
- ClinicalTrials.gov ID
- NCT00351221
- Acronym / sponsor ID
- INMS-110-801
- Conditions
- Noonan Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated due to a patent legal settlement
- Enrolment
- 24 participants
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- First posted
- 12 Jul 2006 (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 trial will investigate the treatment of growth failure in children with Noonan syndrome. Abnormalities in the growth hormone (GH) - insulin-like growth factor-I (IGF-I) axis resulting in low IGF-I levels have been suggested as a possible cause of short stature seen in Noonan syndrome children. Administration of our investigational product is intended to bypass the abnormalities in the GH-IGF axis, and hopefully improve body growth.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Age groups
- Child
Key inclusion criteria (7 listed)
- A diagnosis of Noonan syndrome
- Height less than the 3rd percentile for age and sex (height SDS < -1.88)
- Basal IGF-I less than the mean for age and sex (IGF-I SDS < 0)
- Chronological age greater than 2 years
- Bone age ≤ 11 years for boys, and ≤ 10 years for girls
Key exclusion criteria (3 listed)
- Clinically significant diseases
- Chronic illnesses
- Prior treatment with rhIGF-1
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/rhIGFBP-3 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early (reason given: Terminated due to a patent legal settlement). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2007.
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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.
Kenneth Attie, MD study director
Insmed, Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rhIGF-1/rhIGFBP-3 in people with Noonan Syndrome, run by Insmed Incorporated at 2 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 7 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: stopped early, as of the sponsor's last update on 30 Mar 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 stopped early (reason given: Terminated due to a patent legal settlement). Results are not available in the source record.
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.
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 30 Mar 2007.