Idiopathic Short StaturePhase 4NCT01246219
Short Stature Related Distress
A Phase 4 study testing GH treatment (Genotropin) in people with Idiopathic Short Stature and Self Image, run by Rabin Medical Center at 1 site in Israel.
Trial summary
The research aims to provide data on gender-related psychological impact of GH (Growth Hormone)treatment. Implicit measurable psychological metrics of the therapy outcome on males are to be evaluated in a Prospective Randomized Controlled Trial. Read more about this study
- Who is it for?
- Ages 8 to 17 years, men only
- Conditions the study is for
- Idiopathic Short Stature, Self Image
- What is being tested?
- GH treatment (Genotropin)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 60 participants
- Where
- 1 siteIsrael
- Study dates
- Oct 2011to Nov 2017
- 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 8 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 1 Segment 1 :Boys at the age range of 8-13 years 2. Segment 2: Boys at the age range of 12-17 years 3. Tanner stage 1 (segment 1 only) 4. more than 2 SD's (standard deviation) below the average height
Who cannot join
- Mental retardation,
- Psychiatric illness,
- Lack of informed consent\assent.
- Health status that required chronic treatment with steroids
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Influence of Growth Hormone (GH) Therapy on Short Stature Related Distress a Prospective Randomized Controlled Trial
The research aims to provide data on gender-related psychological impact of GH (Growth Hormone)treatment. Implicit measurable psychological metrics of the therapy outcome on males are to be evaluated in a Prospective Randomized Controlled Trial. As a secondary axis of the study, the investigators aim to provide descriptive documentation of the unique distress signal manifestation implicitly shown by Idiopathic Short Stature (ISS) boys and suggest methods of early detection and measurement of such manifestations that can be used to cement guidelines for the treatment in GH.
Read the detailed description
Study Design:
A prospective randomized, double blind, placebo controlled trial design, comparing four research groups, with 4 repeated psychological measures (before treatment, after 3 months,after 1 year, and at the end of treatment, after 4 years).
The study will be divided into two segments:
Segment 1:double blind, placebo controlled, will include the first 60 ISS (Idiopathic Short Stature)recruited patients. Patients in this segment will be randomized to one of the growth hormone treatment groups in a ratio of 2:1. 40 patients will be treated 4 years with GH and 20 patients will be treated with placebo at the first year of the study and will have the option to start GH treatment after one year, for a duration of 3 years.
Segment 2:after the enrollment to segment 1 will end, enrollment to segment 2 will begin.Participants in segment 2 (patients with ISS and short stature) will serve as control groups and therefore will not be treated with growth hormone. Patients in Segment 2 will be evaluated only once.
The 4 repeated psychological evaluations (before treatment,after 3 months, after 1 year, and at the end of treatment, after 4 years)will include the following:
- Implicit Association Test
- Masculine Self Image
- Pictures Projective Test
- The Rosenberg Self Esteem Scale-a self report measure of explicit self- esteem
- Quality of Life Questionnaire
- Silhouette Apperception Technique (SAT)- a self report measure assessing children's and adolescent's perception of their own body size compared with that of their age-related peers
- Child Behavior Checklist
- Conditions
- Idiopathic Short Stature, Self Image
- Being tested
- GH treatment (Genotropin), 1 year treatment with placebo followed by optional 3 years of GH treatment
- Keywords
- Idiopathic Short stature, Self Image, GH therapy, Psychological distress
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GH treatment (Genotropin)Drug
4 years treatment with GH
1 year treatment with placebo followed by optional 3 years of GH treatmentDrug
1 year treatment with placebo followed by optional 3 years of GH treatment
| Study group | Role | What they receive |
|---|---|---|
| GH treatment | Experimental | 4 years of GH treatment Receives: Drug: GH treatment (Genotropin) |
| Placebo | Placebo comparator | 1 year treatment with placebo followed by optional 3 years of GH treatment Receives: Drug: 1 year treatment with placebo followed by optional 3 years of GH treatment |
| Non treatment group | No intervention | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Psychological Distress score
short stature related distress score as measured by the Height Perception Pictures Test
Measured: After 4 years of treatment
Psychological Distress score
short stature related distress score as measured by the Rosenberg Self-Esteem Scale
Measured: After 4 years of treatment
Psychological Distress score
short stature related distress score as measured by the Quality of Life questionnaire
Measured: After 4 years of treatment
Psychological Distress score
short stature related distress score as measured by Silhouette Apperception Technique questionnaire
Measured: After 4 years of treatment
Secondary outcomes (2)
Perceived self image score
Perceived self image score as measured through Single Category Implicit Association Test
Measured: After 4 years of treatment
Perceived self image score
Perceived self image score as measured through the Masculine Self Image questionnaire
Measured: After 4 years of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Nov 2010 (estimated)
- Study startOct 2011
- Primary completionNov 2017
- Study completionNov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Short StatureStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 60 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rabin Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor
- Investigators
- Moshe Phillip, Prof, Principal investigator, Rabin Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01246219
- Sponsor's protocol ID
- rmc004981ctil
- Status last verified
- Dec 2017
- First submitted
- 16 Nov 2010
- Last updated
- 20 Dec 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Shemesh-Iron M, Lazar L, Lebenthal Y, Nagelberg N, Tenenbaum A, Ezra R, Leffler N, Yackobovitch-Gavan M, Schoenberg-Taz M, Phillip M. Growth hormone therapy and short stature-related distress: A randomized placebo-controlled trial. Clin Endocrinol (Oxf). 2019 May;90(5):690-701. doi: 10.1111/cen.13944. Epub 2019 Mar 7.
PubMed 30721549
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Influence of Growth Hormone (GH) Therapy on Short Stature Related Distress a Prospective Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT01246219
- Acronym / sponsor ID
- rmc004981ctil
- Conditions
- Idiopathic Short Stature, Self Image
- Keywords
- Idiopathic Short stature, Self Image, GH therapy, Psychological distress
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 60 participants (actual)
- Lead sponsor
- Rabin Medical CenterUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- Oct 2011 (actual)
- Primary completion
- Nov 2017 (actual)
- Study completion
- Nov 2017 (actual)
- First posted
- 23 Nov 2010 (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 research aims to provide data on gender-related psychological impact of GH (Growth Hormone)treatment. Implicit measurable psychological metrics of the therapy outcome on males are to be evaluated in a Prospective Randomized Controlled Trial. As a secondary axis of the study, the investigators aim to provide descriptive documentation of the unique distress signal manifestation implicitly shown by Idiopathic Short Stature (ISS) boys and suggest methods of early detection and measurement of such manifestations that can be used to cement guidelines for the treatment in GH.
Detailed description
Study Design:
A prospective randomized, double blind, placebo controlled trial design, comparing four research groups, with 4 repeated psychological measures (before treatment, after 3 months,after 1 year, and at the end of treatment, after 4 years).
The study will be divided into two segments:
Segment 1:double blind, placebo controlled, will include the first 60 ISS (Idiopathic Short Stature)recruited patients. Patients in this segment will be randomized to one of the growth hormone treatment groups in a ratio of 2:1. 40 patients will be treated 4 years with GH and 20 patients will be treated with placebo at the first year of the study and will have the option to start GH treatment after one year, for a duration of 3 years.
Segment 2:after the enrollment to segment 1 will end, enrollment to segment 2 will begin.Participants in segment 2 (patients with ISS and short stature) will serve as control groups and therefore will not be treated with growth hormone. Patients in Segment 2 will be evaluated only once.
The 4 repeated psychological evaluations (before treatment,after 3 months, after 1 year, and at the end of treatment, after 4 years)will include the following:
- Implicit Association Test
- Masculine Self Image
- Pictures Projective Test
- The Rosenberg Self Esteem Scale-a self report measure of explicit self- esteem
- Quality of Life Questionnaire
- Silhouette Apperception Technique (SAT)- a self report measure assessing children's and adolescent's perception of their own body size compared with that of their age-related peers
- Child Behavior Checklist
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| GH treatment | Experimental | Drug: GH treatment (Genotropin)4 years of GH treatment |
| Placebo | Placebo comparator | Drug: 1 year treatment with placebo followed by optional 3 years of GH treatment1 year treatment with placebo followed by optional 3 years of GH treatment |
| Non treatment group | No intervention | – |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- 1 Segment 1 :Boys at the age range of 8-13 years 2. Segment 2: Boys at the age range of 12-17 years 3. Tanner stage 1 (segment 1 only) 4. more than 2 SD's (standard deviation) below the average height
Key exclusion criteria (4 listed)
- Mental retardation,
- Psychiatric illness,
- Lack of informed consent\assent.
- Health status that required chronic treatment with steroids
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 |
|---|---|---|
| GH treatment (Genotropin)4 years treatment with GH | Drug | GH treatment |
| 1 year treatment with placebo followed by optional 3 years of GH treatment1 year treatment with placebo followed by optional 3 years of GH treatment | Drug | Placebo |
Primary outcome
Psychological Distress score
Time frame: After 4 years of treatment
short stature related distress score as measured by the Height Perception Pictures Test
Psychological Distress score
Time frame: After 4 years of treatment
short stature related distress score as measured by the Rosenberg Self-Esteem Scale
Psychological Distress score
Time frame: After 4 years of treatment
short stature related distress score as measured by the Quality of Life questionnaire
Psychological Distress score
Time frame: After 4 years of treatment
short stature related distress score as measured by Silhouette Apperception Technique questionnaire
2 secondary outcomes
Perceived self image score
Time frame: After 4 years of treatment
Perceived self image score
Time frame: After 4 years of treatment
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 20 Dec 2017.
- Derived from this study: PubMed 30721549Shemesh-Iron M, Lazar L, Lebenthal Y, Nagelberg N, Tenenbaum A, Ezra R, Leffler N, Yackobovitch-Gavan M, Schoenberg-Taz M, Phillip M. Growth hormone therapy and short stature-related distress: A randomized placebo-controlled trial. Clin Endocrinol (Oxf). 2019 May;90(5):690-701. doi: 10.1111/cen.13944. Epub 2019 Mar 7.
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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.
Moshe Phillip, Prof principal investigator
Rabin Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing GH treatment (Genotropin) in people with Idiopathic Short Stature and Self Image, run by Rabin Medical Center at 1 site in Israel.
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 1 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: finished, as of the sponsor's last update on 20 Dec 2017. 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 Israel. 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. Some participants in this study may receive a placebo. 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 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.
What does the primary outcome measure?
Psychological Distress score (time frame: After 4 years of treatment). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01246219, which also lists 1 linked publication.
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 20 Dec 2017.