Growth Hormone DeficiencyNCT00263445
Constructing an Insulin-Like Growth Factor-based Prediction Model
An observational study of Growth Hormone Deficiency and Idiopathic Short Stature, run by University of California, Los Angeles at 1 site in United States.
Trial summary
Serum insulin-like growth factor-I (IGF-I) measurements have been shown to correlate well with growth hormone action and effect, and recent data show that serum IGF-I may be related to safety and efficacy of growth hormone (GH) treatment in patients. Read more about this study
- Who is it for?
- Ages 3 to 14 years
- Conditions the study is for
- Growth Hormone Deficiency, Idiopathic Short Stature, Small for Gestational Age
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2004to Jul 2012
- Phase
- Observational
- 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 3 to 14 years
- All sexes
- No healthy volunteers
- Child
Study population: GH-treated chlidren
Who may be able to join
- Prepubertal patients,
- Male/female,
- Ages 3-14 yrs,
- Being treated with growth hormone for the conditions of growth hormone deficiency, idiopathic short stature, and small-for-gestational age with failure to catch up to the normal growth curve by age 2 years.
Who cannot join
- Patients being treated with growth hormone for other conditions such as Turner syndrome, chronic renal failure, or Prader-Willi syndrome
Trial locations and contacts
1 site in 1 country.
United States1 site
UCLA Pediatric Endocrinology
Los Angeles, California, 90095
About this study
Serum insulin-like growth factor-I (IGF-I) measurements have been shown to correlate well with growth hormone action and effect, and recent data show that serum IGF-I may be related to safety and efficacy of growth hormone (GH) treatment in patients. Some studies indicate that high IGF-I levels are associated with increased cancer risk, and low IGF-I levels are associated with increased risk for cardiovascular disease. Studies in children also show that the serum IGF-I level is correlated with the change in height score achieved (that is, the higher the IGF-I level, the greater the gain in height). Pediatric endocrinologists have therefore begun to use serum IGF-I levels, in addition to growth rate and weight gain, to adjust the GH dose in treated children.
Although monitoring of serum IGF-I levels is becoming standard of care in patients begin treated with GH, there are few guidelines regarding the actual logistics of adjusting GH dose. As serum IGF-I level has been linked to both safety and efficacy of GH treatment, the ideal practice would be to maintain serum IGF-I levels within a certain target range. The overall goal of our study is to construct a mathematical model which predicts the change in GH dose necessary to achieve a desired change in IGF-I level.
Hypotheses to be tested by our study include the following: IGF-I measurement has a role in optimization of GH therapy; GH dose change to achieve IGF-I changes are predictable; and gender and puberty affect the relationship between dose change and target IGF-I changes.
Read the detailed description
The first phase of our study, previously approved by the UCLA IRB in August 2004, involved a retrospective chart review and collection of preexisting data. We analyzed charts of patients who underwent non-weight-based GH dose adjustments preceded and followed by a serum IGF-I level. Data collected included patient's age, gender, height, weight, linear height velocity, pubertal status, GH dose, IGF-I levels, disease condition (i.e., growth hormone deficiency, idiopathic short stature, small-for-gestational age) and recording of any adverse effects. Based on this data collected, we performed statistical analysis of the relationship between GH dose change and change in IGF-I level achieved, and have found that in prepubertal children, there is a significant relationship between the GH dose change and change in corresponding IGF-I level. We have subsequently constructed a mathematical prediction model that allows us to determine the GH dose change necessary to achieve a desired IGF-I level, and in this second phase of our study, we plan to apply this mathematical prediction model prospectively for guiding GH dose adjustments in prepubertal children being treated with GH therapy.
Children being treated with GH are typically followed in the outpatient Endocrinology clinic every 3-4 months, and our practitioners have routinely been obtaining serum IGF-I levels (via venipuncture in the outpatient laboratory) for monitoring purposes. Validation of our GH dose change/IGF-I change prediction model will require assessment of sequential serum IGF-I level measurements immediately preceding a GH dose change, and again within 1-4 months following the GH dose change. We plan to continue our retrospective and ongoing analysis of patients who have paired values of GH dose adjustments preceded and followed by a serum IGF-I level, in order that we may continue to fine-tune our prediction model for optimizing GH dose adjustments.
- Keywords
- Growth hormone dosing, IGF-I
- Medical subject headings
- Dwarfism, Pituitary
What is being measured
The primary outcome is the main question the study is designed to answer.
Delta IGF-I SDS
1 SDS change per 20% dose change
Measured: 3-months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Dec 2005 (estimated)
- Study startAug 2004
- Primary completionJul 2012
- Study completionJul 2012
Why it stopped: Insufficient recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- More trials run by University of California, Los AngelesThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Pinchas Cohen, University of California, Los Angeles
- Investigators
- Pinchas Cohen, MD, Principal investigator, University of California, Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00263445
- Sponsor's protocol ID
- 04-07-052-02
- Other IDs
- 304-F02/X3270n (Genentech)
- Status last verified
- Dec 2014
- First submitted
- 7 Dec 2005
- Last updated
- 2 Dec 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Constructing an Insulin-Like Growth Factor-based Prediction Model
- ClinicalTrials.gov ID
- NCT00263445
- Acronym / sponsor ID
- 04-07-052-02
- Keywords
- Growth hormone dosing, IGF-I
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Insufficient recruitment
- Enrolment
- 30 participants (actual)
- Lead sponsor
- University of California, Los AngelesUniversity, hospital or other
- Study start
- Aug 2004
- Primary completion
- Jul 2012 (actual)
- Study completion
- Jul 2012 (actual)
- First posted
- 8 Dec 2005 (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.
Serum insulin-like growth factor-I (IGF-I) measurements have been shown to correlate well with growth hormone action and effect, and recent data show that serum IGF-I may be related to safety and efficacy of growth hormone (GH) treatment in patients. Some studies indicate that high IGF-I levels are associated with increased cancer risk, and low IGF-I levels are associated with increased risk for cardiovascular disease. Studies in children also show that the serum IGF-I level is correlated with the change in height score achieved (that is, the higher the IGF-I level, the greater the gain in height). Pediatric endocrinologists have therefore begun to use serum IGF-I levels, in addition to growth rate and weight gain, to adjust the GH dose in treated children.
Although monitoring of serum IGF-I levels is becoming standard of care in patients begin treated with GH, there are few guidelines regarding the actual logistics of adjusting GH dose. As serum IGF-I level has been linked to both safety and efficacy of GH treatment, the ideal practice would be to maintain serum IGF-I levels within a certain target range. The overall goal of our study is to construct a mathematical model which predicts the change in GH dose necessary to achieve a desired change in IGF-I level.
Hypotheses to be tested by our study include the following: IGF-I measurement has a role in optimization of GH therapy; GH dose change to achieve IGF-I changes are predictable; and gender and puberty affect the relationship between dose change and target IGF-I changes.
Detailed description
The first phase of our study, previously approved by the UCLA IRB in August 2004, involved a retrospective chart review and collection of preexisting data. We analyzed charts of patients who underwent non-weight-based GH dose adjustments preceded and followed by a serum IGF-I level. Data collected included patient's age, gender, height, weight, linear height velocity, pubertal status, GH dose, IGF-I levels, disease condition (i.e., growth hormone deficiency, idiopathic short stature, small-for-gestational age) and recording of any adverse effects. Based on this data collected, we performed statistical analysis of the relationship between GH dose change and change in IGF-I level achieved, and have found that in prepubertal children, there is a significant relationship between the GH dose change and change in corresponding IGF-I level. We have subsequently constructed a mathematical prediction model that allows us to determine the GH dose change necessary to achieve a desired IGF-I level, and in this second phase of our study, we plan to apply this mathematical prediction model prospectively for guiding GH dose adjustments in prepubertal children being treated with GH therapy.
Children being treated with GH are typically followed in the outpatient Endocrinology clinic every 3-4 months, and our practitioners have routinely been obtaining serum IGF-I levels (via venipuncture in the outpatient laboratory) for monitoring purposes. Validation of our GH dose change/IGF-I change prediction model will require assessment of sequential serum IGF-I level measurements immediately preceding a GH dose change, and again within 1-4 months following the GH dose change. We plan to continue our retrospective and ongoing analysis of patients who have paired values of GH dose adjustments preceded and followed by a serum IGF-I level, in order that we may continue to fine-tune our prediction model for optimizing GH dose adjustments.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- 3 to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- GH-treated chlidren
Key inclusion criteria (4 listed)
- Prepubertal patients,
- Male/female,
- Ages 3-14 yrs,
- Being treated with growth hormone for the conditions of growth hormone deficiency, idiopathic short stature, and small-for-gestational age with failure to catch up to the normal growth curve by age 2 years.
Key exclusion criteria (1 listed)
- Patients being treated with growth hormone for other conditions such as Turner syndrome, chronic renal failure, or Prader-Willi syndrome
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Delta IGF-I SDS
Time frame: 3-months
1 SDS change per 20% dose change
Results and safety
The study was stopped early (reason given: Insufficient recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Dec 2014.
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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.
Pinchas Cohen, MD principal investigator
University of California, Los Angeles
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Growth Hormone Deficiency and Idiopathic Short Stature, run by University of California, Los Angeles 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 4 inclusion and 1 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 2 Dec 2014. 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.
Are results available for this study?
The study was stopped early (reason given: Insufficient recruitment). 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.
What does the primary outcome measure?
Delta IGF-I SDS (time frame: 3-months). 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 2 Dec 2014.