Short StaturePhase 2/3MAINTENANCENCT02879747
Individualizing Dose of Growth Hormone to Maintain Normal Growth Velocity After Fulfilled Catch up Growth in Children
A Phase 2/3 study testing Genotropin in people with Short Stature, run by Göteborg University.
Trial summary
The objective was to study whether normal growth velocity can be maintained with adapted GH dosage in GH treated prepubertal children who have responded to GH treatment with fulfilled catch up growth (=difference to target height reached, less than - 0.6 SDS). Read more about this study
- Who is it for?
- Ages 4 to 18 years
- Conditions the study is for
- Short Stature
- What is being tested?
- Genotropin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 99 participants
- Where
- No sites listed yet
- Study dates
- Dec 2003to Dec 2017
- Phase
- Phase 2/3
- 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 4 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participated in the 'GH-dose catch-up study' 98- 0198-003.
- Midparental height reached (difference less than 0.6 SDS)
- Prepubertal at start of the study (girls =B 1, boys: testes :<; 3ml).
- Signed written informed consent from the patient's parents (and the child if old enough)
Who cannot join
- Disease affecting growth other than correctly treated hypothyroidism.
- Incapable of following the study protocol (i.e. bad compliance in the previous study).
- Puberty (> breast stage 2, or testes > 4ml).
- Poor compliance.
About this study
Official title: Individualizing the Dose of Growth Hormone (GH) to Maintain Normal Growth Velocity After Fulfilled Catch up Growth in Children Within TR 98-0198-003
The objective was to study whether normal growth velocity can be maintained with adapted GH dosage in GH treated prepubertal children who have responded to GH treatment with fulfilled catch up growth (=difference to target height reached, less than - 0.6 SDS).
Read the detailed description
The aim of the trial is to study the effect of adapted GH treatment in order to find an individualized GH dose maintaining normal growth velocity close to target height SDS and normal metabolism after fulfilled catch up growth in prepubertal children treated with individual doses of GH within the trial 98-0198-003 (1). The overall aim is to find for the individual the lowest effective GH dose during maintenance period, maintaining normal growth velocity and metabolism, i.e. a satisfactory biological active dose.
- Conditions
- Short Stature
- Being tested
- Genotropin
- Keywords
- treatment, growth hormone, childhood, short stature
- Medical subject headings
- Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GenotropinDrug
Children were randomized to either decreased dose by 50% or unchanged dose after 2-3 years of Catch-up growth
| Study group | Role | What they receive |
|---|---|---|
| Interventional | Active comparator | Unchanged dose Genotropin Receives: Drug: Genotropin |
| Interventional 2 | Active comparator | reduced dose 50% Genotropin Receives: Drug: Genotropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Proportion of Children Maintaining Normal Growth Velocity
The proportion of children in the intervention 1 group (=reduced dose) that maintained an individual ΔheightSDS within ±0.3 during the first year of the Maintenance trial, compared to the proportion of children in the intervention 2 group (non-reduced dose)
Measured: twelve months
Secondary outcomes (2)
IGF-I
Delta Insulin-like growth factor-I (24 months after start compared to start of study) expressed as ng/ml and converted to standard deviation scores (SDS) to adjust for gender and age.
A standard deviation score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. A mean value within +-0.5 SDS and a range within +-1.0 SDS is a favorable outcome.
Measured: start of study to two years after start in the trial
Height SDS at Start of Puberty
Height at start of puberty measured as cm and expressed as standard deviation score (SDS) to adjust for age and gender
Measured: 1-7 years in the trial
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2016 (estimated)
- Study startDec 2003
- Primary completionMar 2012
- Study completionDec 2017
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 (4)Papers the sponsor has linked to this study.
- Other trials for Short StatureStudies of the same condition, open and under way.
- More trials run by Göteborg UniversityThe 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
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 99 participants (actual)
Sponsor and investigators
- Lead sponsor
- Göteborg University
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Sponsor
- Investigators
- Berit Kriström, MD, Principal investigator, University Hospital, UmeåNils-Östen Nilsson, MD, Principal investigator, Halmstad Department of PediatricsMaria Halldin, MD, Principal investigator, Uppsala University HospitalSten Ivarsson, MD, Prof, Principal investigator, Malmö Academic HospitalKerstin Albertsson-Wikland, MD, prof, Principal investigator, Gothenburg University, Departments of pediatrics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02879747
- Sponsor's protocol ID
- NRA 6280003
- Acronym
- MAINTENANCE
- Status last verified
- Apr 2019
- First submitted
- 10 Aug 2016
- Last updated
- 30 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Wikland KA, Kristrom B, Rosberg S, Svensson B, Nierop AF. Validated multivariate models predicting the growth response to GH treatment in individual short children with a broad range in GH secretion capacities. Pediatr Res. 2000 Oct;48(4):475-84. doi: 10.1203/00006450-200010000-00010.
PubMed 11004238Kristrom B, Aronson AS, Dahlgren J, Gustafsson J, Halldin M, Ivarsson SA, Nilsson NO, Svensson J, Tuvemo T, Albertsson-Wikland K. Growth hormone (GH) dosing during catch-up growth guided by individual responsiveness decreases growth response variability in prepubertal children with GH deficiency or idiopathic short stature. J Clin Endocrinol Metab. 2009 Feb;94(2):483-90. doi: 10.1210/jc.2008-1503. Epub 2008 Nov 11.
PubMed 19001519Ly HJ, Ankarberg-Lindgren C, Fors H, Nilsson S, Dahlgren J. Interpreting IGF-1 in children treated with recombinant growth hormone: challenges during early puberty. Front Endocrinol (Lausanne). 2025 Jan 21;15:1514935. doi: 10.3389/fendo.2024.1514935. eCollection 2024.
PubMed 39906034Decker R, Albertsson-Wikland K, Kristrom B, Halldin M, Gustafsson J, Nilsson NO, Dahlgren J. GH Dose Reduction Maintains Normal Prepubertal Height Velocity After Initial Catch-Up Growth in Short Children. J Clin Endocrinol Metab. 2019 Mar 1;104(3):835-844. doi: 10.1210/jc.2018-01006.
PubMed 30339244
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individualizing the Dose of Growth Hormone (GH) to Maintain Normal Growth Velocity After Fulfilled Catch up Growth in Children Within TR 98-0198-003
- ClinicalTrials.gov ID
- NCT02879747
- Acronym / sponsor ID
- MAINTENANCE / NRA 6280003
- Conditions
- Short Stature
- Keywords
- treatment, growth hormone, childhood, short stature
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 99 participants (actual)
- Lead sponsor
- Göteborg UniversityUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- Dec 2003 (actual)
- Primary completion
- Mar 2012 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 26 Aug 2016 (estimated)
- Results
- Posted 30 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective was to study whether normal growth velocity can be maintained with adapted GH dosage in GH treated prepubertal children who have responded to GH treatment with fulfilled catch up growth (=difference to target height reached, less than - 0.6 SDS).
Detailed description
The aim of the trial is to study the effect of adapted GH treatment in order to find an individualized GH dose maintaining normal growth velocity close to target height SDS and normal metabolism after fulfilled catch up growth in prepubertal children treated with individual doses of GH within the trial 98-0198-003 (1). The overall aim is to find for the individual the lowest effective GH dose during maintenance period, maintaining normal growth velocity and metabolism, i.e. a satisfactory biological active dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Interventional | Active comparator | Drug: GenotropinUnchanged dose Genotropin |
| Interventional 2 | Active comparator | Drug: Genotropinreduced dose 50% Genotropin |
Who may qualify
- Ages
- 4 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Participated in the 'GH-dose catch-up study' 98- 0198-003.
- Midparental height reached (difference less than 0.6 SDS)
- Prepubertal at start of the study (girls =B 1, boys: testes :<; 3ml).
- Signed written informed consent from the patient's parents (and the child if old enough)
Key exclusion criteria (4 listed)
- Disease affecting growth other than correctly treated hypothyroidism.
- Incapable of following the study protocol (i.e. bad compliance in the previous study).
- Puberty (> breast stage 2, or testes > 4ml).
- Poor compliance.
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 |
|---|---|---|
| GenotropinChildren were randomized to either decreased dose by 50% or unchanged dose after 2-3 years of Catch-up growth | Drug | Interventional, Interventional 2 |
Primary outcome
The Proportion of Children Maintaining Normal Growth Velocity
Time frame: twelve months
The proportion of children in the intervention 1 group (=reduced dose) that maintained an individual ΔheightSDS within ±0.3 during the first year of the Maintenance trial, compared to the proportion of children in the intervention 2 group (non-reduced dose)
2 secondary outcomes
IGF-I
Time frame: start of study to two years after start in the trial
Height SDS at Start of Puberty
Time frame: 1-7 years in the trial
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 11004238Wikland KA, Kristrom B, Rosberg S, Svensson B, Nierop AF. Validated multivariate models predicting the growth response to GH treatment in individual short children with a broad range in GH secretion capacities. Pediatr Res. 2000 Oct;48(4):475-84. doi: 10.1203/00006450-200010000-00010.
- Results publication: PubMed 19001519Kristrom B, Aronson AS, Dahlgren J, Gustafsson J, Halldin M, Ivarsson SA, Nilsson NO, Svensson J, Tuvemo T, Albertsson-Wikland K. Growth hormone (GH) dosing during catch-up growth guided by individual responsiveness decreases growth response variability in prepubertal children with GH deficiency or idiopathic short stature. J Clin Endocrinol Metab. 2009 Feb;94(2):483-90. doi: 10.1210/jc.2008-1503. Epub 2008 Nov 11.
- Derived from this study: PubMed 39906034Ly HJ, Ankarberg-Lindgren C, Fors H, Nilsson S, Dahlgren J. Interpreting IGF-1 in children treated with recombinant growth hormone: challenges during early puberty. Front Endocrinol (Lausanne). 2025 Jan 21;15:1514935. doi: 10.3389/fendo.2024.1514935. eCollection 2024.
- Derived from this study: PubMed 30339244Decker R, Albertsson-Wikland K, Kristrom B, Halldin M, Gustafsson J, Nilsson NO, Dahlgren J. GH Dose Reduction Maintains Normal Prepubertal Height Velocity After Initial Catch-Up Growth in Short Children. J Clin Endocrinol Metab. 2019 Mar 1;104(3):835-844. doi: 10.1210/jc.2018-01006.
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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.
Berit Kriström, MD principal investigator
University Hospital, Umeå
Nils-Östen Nilsson, MD principal investigator
Halmstad Department of Pediatrics
Maria Halldin, MD principal investigator
Uppsala University Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Genotropin in people with Short Stature, run by Göteborg University.
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 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 30 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
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?
Yes. Results are posted in the registry (first posted 30 Apr 2019). 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?
The Proportion of Children Maintaining Normal Growth Velocity (time frame: twelve months). 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 NCT02879747, which also lists 4 linked publications.
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 Apr 2019.