Growth Hormone Insensitivity Syndrome (GHIS)Phase 2/3NCT00368173
IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome
A Phase 2/3 study testing rhIGF-I/rhIGFBP-3 in people with Growth Hormone Insensitivity Syndrome (GHIS) and Laron Syndrome, run by Insmed Incorporated at 15 sites in 14 countries.
Trial summary
STUDY OBJECTIVE To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Growth Hormone Insensitivity Syndrome (GHIS), Laron Syndrome
- What is being tested?
- rhIGF-I/rhIGFBP-3
- How many people
- Not given
- Where
- 15 sitesin 14 countries
- Study dates
- Start not givento not given
- Phase
- Phase 2/3
- 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 18 years
- All sexes
- Child, Adult
Who may be able to join
- A diagnosis of GHIS such as Laron syndrome,
- 2 - 18 years of age,
- Height less than or equal to -3SD for age,
- Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL
Who cannot join
- Children in puberty,
- Diagnosed malignancy,
- A diagnosis of diabetes mellitus
Trial locations and contacts
15 sites in 14 countries.
15 sites in 14 countries
Argentina2 sites
Dr. Alicia Belgorosky
Buenos Aires
Dr. Christina Bazan
San Miguel de Tucumán
About this study
STUDY OBJECTIVE
To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS.
STUDY DESIGN
This study is an open-label, multi-center clinical trial to evaluate the safety and effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS) such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional safety and long-term efficacy data will be assessed in a second 12 month treatment period.
- Being tested
- rhIGF-I/rhIGFBP-3
- Medical subject headings
- Laron Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhIGF-I/rhIGFBP-3Drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2006 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone Insensitivity Syndrome (GHIS)Studies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Sponsor and investigators
- Lead sponsor
- Insmed Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00368173
- Sponsor's protocol ID
- INSM-110-303
- Status last verified
- Sep 2012
- First submitted
- 23 Aug 2006
- Last updated
- 13 Sep 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome
- ClinicalTrials.gov ID
- NCT00368173
- Acronym / sponsor ID
- INSM-110-303
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Lead sponsor
- Insmed IncorporatedIndustry (pharma, biotech, devices)
- First posted
- 24 Aug 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.
STUDY OBJECTIVE
To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS.
STUDY DESIGN
This study is an open-label, multi-center clinical trial to evaluate the safety and effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS) such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional safety and long-term efficacy data will be assessed in a second 12 month treatment period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- A diagnosis of GHIS such as Laron syndrome,
- 2 - 18 years of age,
- Height less than or equal to -3SD for age,
- Pre-pubertal, defined as Tanner breast stage 1 or testis volume <4mL
Key exclusion criteria (3 listed)
- Children in puberty,
- Diagnosed malignancy,
- A diagnosis of diabetes mellitus
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-I/rhIGFBP-3 | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2012.
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.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing rhIGF-I/rhIGFBP-3 in people with Growth Hormone Insensitivity Syndrome (GHIS) and Laron Syndrome, run by Insmed Incorporated at 15 sites in 14 countries.
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 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 13 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Argentina, United States, Australia, Brazil, China and 9 more countries. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 13 Sep 2012.