Turner's SyndromePhase 4NCT01066052
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
A Phase 4 study testing r-hGH in people with Turner's Syndrome, run by Merck KGaA, Darmstadt, Germany at 1 site in France.
Trial summary
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome… Read more about this study
- Who is it for?
- Ages Up to 4 years, women only
- Conditions the study is for
- Turner's Syndrome
- What is being tested?
- r-hGH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 115 participants
- Where
- 1 siteFrance
- Study dates
- 29 Feb 1992to 31 Aug 2010
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 4 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- Young girls with turner syndrome proved by karyotype
- Growth hormone secretion confirmed with ornithin stimulation test
- Normal glucidic metabolism confirmed by assessment of HbA1c
- None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
- No previous or associated treatment with anabolic or sexual steroids
- Known parental height
Who cannot join
- Severe associated pathology with impact on growth
- Concomitant treatment with impact on growth
- Previous or associated treatment with anabolic steroids
- Associated growth hormone deficiency
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Collaborative Study to Assess the Effects of Treatment With Recombinant Growth Hormone Saizen® in the Prevention of Short Stature in Young Girls Suffering From Turner Syndrome Before the Age of 4 Years. Original French Title: Etude Collaborative Pour apprécier Les Effets du Traitement Par l'Hormone de Croissance Recombinante SAIZEN® Dans le Retard de Croissance de la Fillette Atteinte de Syndrome de Turner Avant l'âge de 4 Ans
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.
- Conditions
- Turner's Syndrome
- Being tested
- r-hGH
- Keywords
- Growth hormone, Turner syndrome, r-hGH, early treatment
- Medical subject headings
- Turner Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
r-hGHDrug
Also called Saizen®
Subcutaneous administration.
| Study group | Role | What they receive |
|---|---|---|
| r-hGH | Experimental | Participants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years. Receives: Drug: r-hGH |
| Historical Control | No intervention | This arm will include matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Height SDS at Year 4
Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
Measured: Year 4
Secondary outcomes (5)
Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported.
Measured: Baseline up to Year 2
Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)
BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported.
Measured: Baseline, Year 1, Year 2
Number of Participants With Anti r-hGH Antibodies
Measured: Baseline up to Year 2
Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported.
Measured: Baseline up to Year 2
Number of Participants Who Reached Normal Height at Year 4
Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
Measured: Year 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2010 (estimated)
- Study start29 Feb 1992
- Primary completion31 Mar 1999
- Study completion31 Aug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Turner's 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 115 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck KGaA, Darmstadt, Germany
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Responsible, Study director, Merck KGaA, Darmstadt, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01066052
- Sponsor's protocol ID
- GF 5834
- Status last verified
- Jun 2018
- First submitted
- 5 Feb 2010
- Last updated
- 14 Dec 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Linglart A, Cabrol S, Berlier P, Stuckens C, Wagner K, de Kerdanet M, Limoni C, Carel JC, Chaussain JL; French Collaborative Young Turner Study Group. Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome. Eur J Endocrinol. 2011 Jun;164(6):891-7. doi: 10.1530/EJE-10-1048. Epub 2011 Mar 11.
PubMed 21398400
Study at a glance
From the registry record, last updated by the sponsor on 14 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Collaborative Study to Assess the Effects of Treatment With Recombinant Growth Hormone Saizen® in the Prevention of Short Stature in Young Girls Suffering From Turner Syndrome Before the Age of 4 Years. Original French Title: Etude Collaborative Pour apprécier Les Effets du Traitement Par l'Hormone de Croissance Recombinante SAIZEN® Dans le Retard de Croissance de la Fillette Atteinte de Syndrome de Turner Avant l'âge de 4 Ans
- ClinicalTrials.gov ID
- NCT01066052
- Acronym / sponsor ID
- GF 5834
- Conditions
- Turner's Syndrome
- Keywords
- Growth hormone, Turner syndrome, r-hGH, early treatment
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 115 participants (actual)
- Lead sponsor
- Merck KGaA, Darmstadt, GermanyIndustry (pharma, biotech, devices)
- Study start
- 29 Feb 1992 (actual)
- Primary completion
- 31 Mar 1999 (actual)
- Study completion
- 31 Aug 2010 (actual)
- First posted
- 10 Feb 2010 (estimated)
- Results
- Posted 14 Dec 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| r-hGH | Experimental | Drug: r-hGHParticipants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years. |
| Historical Control | No intervention | –This arm will include matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated. |
Who may qualify
- Ages
- Up to 4 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Young girls with turner syndrome proved by karyotype
- Growth hormone secretion confirmed with ornithin stimulation test
- Normal glucidic metabolism confirmed by assessment of HbA1c
- None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
- No previous or associated treatment with anabolic or sexual steroids
Key exclusion criteria (4 listed)
- Severe associated pathology with impact on growth
- Concomitant treatment with impact on growth
- Previous or associated treatment with anabolic steroids
- Associated growth hormone deficiency
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 |
|---|---|---|
| r-hGHAlso: Saizen®Subcutaneous administration. | Drug | r-hGH |
Primary outcome
Height SDS at Year 4
Time frame: Year 4
Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.
5 secondary outcomes
Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
Time frame: Baseline up to Year 2
Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)
Time frame: Baseline, Year 1, Year 2
Number of Participants With Anti r-hGH Antibodies
Time frame: Baseline up to Year 2
Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
Time frame: Baseline up to Year 2
Number of Participants Who Reached Normal Height at Year 4
Time frame: Year 4
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Dec 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 21398400Linglart A, Cabrol S, Berlier P, Stuckens C, Wagner K, de Kerdanet M, Limoni C, Carel JC, Chaussain JL; French Collaborative Young Turner Study Group. Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome. Eur J Endocrinol. 2011 Jun;164(6):891-7. doi: 10.1530/EJE-10-1048. Epub 2011 Mar 11.
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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.
Medical Responsible study director
Merck KGaA, Darmstadt, Germany
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 r-hGH in people with Turner's Syndrome, run by Merck KGaA, Darmstadt, Germany at 1 site in France.
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 6 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 14 Dec 2018. 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 France. 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 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?
Yes. Results are posted in the registry (first posted 14 Dec 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Height SDS at Year 4 (time frame: Year 4). 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 NCT01066052, 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 14 Dec 2018.