Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Class |
|---|---|
| Pfizer | INDUSTRY |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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).
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Interventional | Active Comparator | Unchanged dose Genotropin |
|
| Interventional 2 | Active Comparator | reduced dose 50% Genotropin |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Genotropin | Drug | Children were randomized to either decreased dose by 50% or unchanged dose after 2-3 years of Catch-up growth |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | twelve months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Changes in Height | Changes in height standard deviation scores (SDS) (calculated as height in cm at start converted to SDS and height in cm after two years in the trial converted to SDS) | start of study to two years after start in the trial |
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Berit Kriström, MD | University Hospital, Umeå | Principal Investigator |
| Nils-Östen Nilsson, MD | Halmstad Department of Pediatrics | Principal Investigator |
| Maria Halldin, MD | Uppsala University Hospital | Principal Investigator |
| Sten Ivarsson, MD, Prof | Malmö Academic Hospital | Principal Investigator |
| Kerstin Albertsson-Wikland, MD, prof | Gothenburg University, Departments of pediatrics | Principal Investigator |
Not provided
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 11004238 | Background | 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. | |
| 19001519 | Result | Kristrom 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. |
Not provided
| ID | Type | URL | Comment |
|---|---|---|---|
| Study Protocol | View IPD |
If glucose levels at the day of inclusion were higher than 7.1 mmol/L, then the child was excluded before assignment.
All children in the fix dose group and children in the individualized dose group who have reached their midparental height SDS (>- 0.6 SDS) during at least two years of GH-treatment within the 'GH-dose-catch up' study (TR 98-0198-003) and still are prepubertal were eligible in the study.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Decreased GH Dose | Decreased GH dose Genotropin Genotropin: Children were randomized to 50% GH dose after 2-3 years of initial GH treatment in previous trial (years depending on achieved Catch-up) |
| FG001 | Unchanged GH Dose | Unchanged GH dose Genotropin: Children were randomized to unchanged dose after 2-3 years of initial GH treatment in previous trial (years depending on achieved Catch-up) |
| FG002 | Control Group | Control Group with informed consent but not randomized |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
98 children participated with two randomized arms (n=27+38) and one control arm with conventional fixed GH treatment (n=33).
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Decreased GH Dose | Reduced dose 50% Genotropin Genotropin: Children were randomized to decreased GH dose by 50% after 2-3 years of Catch-up growth |
| BG001 | Unchanged GH Dose | Unchanged dose Genotropin Genotropin: Children were randomized to unchanged GH dose after 2-3 years of Catch-up growth |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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) | Posted | Count of Participants | Participants | twelve months |
|
Two years or in some cases up to seven prepubertal years
No relevant information according to reviewer.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Decreased GH Dose | Reduced dose 50% Genotropin Children were randomized to decreased GH dose by 50% after 2-3 years of Catch-up growth |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Otitis media | Ear and labyrinth disorders | ICD10 | Systematic Assessment | Otitis media with perforation needed hospitalization |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| virosis | Infections and infestations | ICD10 | Systematic Assessment | Common adverse event was virosis winter-time |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr Jovanna Dahlgren, PI and director of Department of Pediatrics | Queen Silvia Children´s Hospital and University of Gothenburg | +46702750233 | jovanna.dahlgren@gu.se |
Not provided
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| ID | Term |
|---|---|
| D001848 | Bone Diseases, Developmental |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided
| ID | Term |
|---|---|
| D019382 | Human Growth Hormone |
| ID | Term |
|---|---|
| D013006 | Growth Hormone |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D036361 | Peptide Hormones |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 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 | 1-7 years in the trial |
| 39906034 | Derived | Ly 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. |
| 30339244 | Derived | Decker 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. |
The document will be found under "Trials" and then "GH-dose/Maintenance study" |
| Statistical Analysis Plan | View IPD | The document will be found under "Trials" and then "GH-dose/Maintenance study" |
| BG002 | Control Group | Control Group randomized in the previous study to fix dose. This group of children continued with fix dose in the current trial after signing informed consent. |
| BG003 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| OG002 | Control Group | Control Group children with fix dose through the previous and current trial and with signed consent |
|
|
| Secondary | 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. | Drop outs due to puberty or adverse events | Posted | Mean | Standard Deviation | standard deviation scores | start of study to two years after start in the trial |
|
|
|
| Secondary | 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 | All 98 Children were measured at start of puberty | Posted | Mean | Standard Deviation | standard deviation scores | 1-7 years in the trial |
|
|
|
| Other Pre-specified | Changes in Height | Changes in height standard deviation scores (SDS) (calculated as height in cm at start converted to SDS and height in cm after two years in the trial converted to SDS) | Posted | Mean | Standard Deviation | standard deviation scores | start of study to two years after start in the trial |
|
|
|
| 0 |
| 27 |
| 3 |
| 27 |
| 20 |
| 27 |
| EG001 | Unchanged GH Dose | Unchanged dose Children were randomized to unchanged dose after 2-3 years of Catch-up growth | 0 | 38 | 4 | 38 | 25 | 38 |
| EG002 | Control Group | Control Group with fix dose. Children that were randomized to fix dose in the previous and current trial and signed informed consent | 0 | 33 | 3 | 33 | 24 | 33 |
|
| Abdominal pain | Gastrointestinal disorders | ICD10 | Systematic Assessment | Hospitalization for risk of appendicitis |
|
| facial nerve palsy due to tick disease | Infections and infestations | ICD10 | Systematic Assessment | Hospitalization due to need for intravenous antibiotic treatment |
|
| accident | Injury, poisoning and procedural complications | ICD10 | Systematic Assessment | Hospitalization due to car accident |
|
| fracture | Injury, poisoning and procedural complications | ICD10 | Systematic Assessment | Fracture after falling from a tree |
|
| Urinary tract infection (upper) | Infections and infestations | ICD10 | Systematic Assessment | Urinary tract infection with hospitalization |
|
|
Not provided
Not provided
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004700 | Endocrine System Diseases |
| D006728 |
| Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |