Primary outcomeMean· First year of treatment
Height Velocity
| Group | Value (cm/y) | Spread |
|---|---|---|
| rhGH Alone | 9.3 | 1.7 |
| 45 rhGH + 50 rhIGF-1 | 10.1 | 1.3 |
| 45 rhGH + 100 rhIGF-1 | 9.7 | 2.5 |
| 45 rhGH + 150 rhIGF-1 | 11.2 | 2.1 |
Trial · Interventional
TerminatedPhase 2Results postedRegistry datarhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
NCTNCT00572156 (opens ClinicalTrials.gov in a new tab)Other idMS316Other id2019-000843-29Other id2019
This trial was stopped early.
Reason given at the registry: The study was prematurely terminated due to strategic reasons.
Results were posted for this trial.
Overview
- Status
- Terminated
- Phase
- Phase 2
- Enrollment
- 106 participants (actual)
- Sponsor
- Ipsen · Industry
- Design
- Randomized · Open label (no masking)
- Countries
1 country
United States
- Start
- Dec 2007
- Primary completion
- Apr 2010
- Completion
- Mar 2012
- First posted
- 12 Dec 2007 (estimated)
- Last update posted
- 30 Mar 2023
- Conditions
- Insulin-like Growth Factor-1 Deficiency
Timeline
1 trial · 1 stopped early · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
2007200820092010201120122013
- Ph 2Terminated
rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency
NCT00572156Dec 2007 to Mar 2012
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence. Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally. This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.
Interventions
- NutropinAq® (Somatropin [rDNA origin])Drug
- Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])Drug
- Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])Drug
- Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])Drug
Arms4
Active comparator
1. rhGH Alone
Experimental
2. Combination Dose
Experimental
3. Combination Dose
Experimental
4. Combination Dose
Primary outcomes1
Outcomes the sponsor listed as primary.
Height Velocity
Time frame · First year of treatment
Secondary outcomes(12)
Height Velocity
Time frame · Second, third and fourth year
Cumulative Change in Height Standard Deviation Score (SDS)
Time frame · First, second, third and fourth year
Registry description
Height was measured standing and without shoes, and recorded as the mean of three measurements (the subject being repositioned each time) by the same observer using a Harpenden or other wall-mounted stadiometer which was to be calibrated prior to measurement of each subject and a calibration log kept. The SDS was calculated as: SDS=\[(value /M)\^L - 1\] / LS; using power (L), Mean (M) and coefficient of variation (S). The reference values were dependent on gender in addition to age and were selected at the age the closest below subject's age. SDS scores were calculated using L, M and S as defined in the National Center for Health Statistics 2000 data as provided by the Center for Disease Control (Kuczmarski, Ogden et al. 2002)
Predicted Adult Height (PAH)
Time frame · At baseline (Day 1), year 1,2,3 and 4
Registry description
Predicted Adult Height calculated by method, Roche-Wainer-Thissen (RWT) and mid-parental target height SDS. The SDS was calculated as: SDS=\[(value /M)\^L - 1\] / LS; using power (L), Mean (M) and coefficient of variation (S). The reference values were dependent on gender in addition to age and were selected at the age the closest below subject's age. SDS scores were calculated using L, M and S as defined in the National Center for Health Statistics 2000 data as provided by the Center for Disease Control (Kuczmarski, Ogden et al. 2002)
Total Change From Baseline (Day 1) in BMI SDS
Time frame · At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1)
Registry description
BMI was calculated by weight divided by height squared and measured as kilogram per square meter (kg/m\^2). The SDS was calculated as: SDS=\[(value /M)\^L - 1\] / LS; using power (L), Mean (M) and coefficient of variation (S). The reference values were dependent on gender in addition to age and were selected at the age the closest below subject's age. SDS scores were calculated using L, M and S as defined in the National Center for Health Statistics 2000 data as provided by the Center for Disease Control (Kuczmarski, Ogden et al. 2002)
Skeletal Maturation
Time frame · At baseline(day 1), year 1,2,3 and 4
Registry description
Assessed by bone age. Bone age was determined by the radiograph. The SDS was calculated as: SDS=\[(value /M)\^L - 1\] / LS; using power (L), Mean (M) and coefficient of variation (S). The reference values were dependent on gender in addition to age and were selected at the age the closest below subject's age. SDS scores were calculated using L, M and S as defined in the National Center for Health Statistics 2000 data as provided by the Center for Disease Control (Kuczmarski, Ogden et al. 2002)
Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor-1 (IGF-1)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-1 (IGFBP-1)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-3 (IGFPB-3)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Acid-Labile Subunit (ALS)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone Binding Protein (GHBP)
Time frame · At Baseline (Day 1), Year 1,2,3 and 4
Summary of Adverse Events With Number of Occurrences
Time frame · Approximately up to 4 years.
Registry description
A Data Monitoring Committee (DMC) was established to monitor subject safety
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeMean· Second, third and fourth year
Height Velocity
| Group | Value (cm/y) | Spread |
|---|---|---|
| rhGH Alone | 8.4 | 1.3 |
| 45 rhGH + 50 rhIGF-1 | 9.1 | 1.2 |
| 45 rhGH + 100 rhIGF-1 | 9.4 | 1.6 |
| 45 rhGH + 150 rhIGF-1 | 10.1 | 1.8 |
Secondary outcomeMean· First, second, third and fourth year
Cumulative Change in Height Standard Deviation Score (SDS)
| Group | Value (SDS) | Spread |
|---|---|---|
| rhGH Alone | 0.7 | 0.3 |
| 45 rhGH + 50 rhIGF-1 | 0.9 | 0.2 |
| 45 rhGH + 100 rhIGF-1 | 0.8 | 0.4 |
| 45 rhGH + 150 rhIGF-1 | 1.0 | 0.4 |
Secondary outcomeMean· At baseline (Day 1), year 1,2,3 and 4
Predicted Adult Height (PAH)
| Group | Value (SDS) | Spread |
|---|---|---|
| rhGH Alone | -1.67 | 0.51 |
| 45 rhGH + 50 rhIGF-1 | -1.83 | 0.58 |
| 45 rhGH + 100 rhIGF-1 | -1.69 | 0.59 |
| 45 rhGH + 150 rhIGF-1 | -1.76 | 0.73 |
Secondary outcomeMean· At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1)
Total Change From Baseline (Day 1) in BMI SDS
| Group | Value (SDS) | Spread |
|---|---|---|
| rhGH Alone | 0.24 | 0.43 |
| 45 rhGH + 50 rhIGF-1 | 0.31 | 0.39 |
| 45 rhGH + 100 rhIGF-1 | 0.36 | 0.41 |
| 45 rhGH + 150 rhIGF-1 | 0.54 | 0.40 |
Secondary outcomeMean· At baseline(day 1), year 1,2,3 and 4
Skeletal Maturation
| Group | Value (SDS) | Spread |
|---|---|---|
| rhGH Alone | 7.6 | 1.8 |
| 45 rhGH + 50 rhIGF-1 | 7.3 | 1.9 |
| 45 rhGH + 100 rhIGF-1 | 7.2 | 2.0 |
| 45 rhGH + 150 rhIGF-1 | 7.4 | 2.0 |
Secondary outcomeMean· At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)
| Group | Value (ng/mL) | Spread |
|---|---|---|
| rhGH Alone | 1.1 | 2.2 |
| 45 rhGH + 50 rhIGF-1 | 2.9 | 8.1 |
| 45 rhGH + 100 rhIGF-1 | 2.4 | 4.5 |
| 45 rhGH + 150 rhIGF-1 | 1.9 | 2.3 |
Secondary outcomeMean· At Baseline (Day 1), Year 1,2,3 and 4
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor-1 (IGF-1)
| Group | Value (ng/mL) | Spread |
|---|---|---|
| rhGH Alone | 108.3 | 39.8 |
| 45 rhGH + 50 rhIGF-1 | 87.1 | 31.0 |
| 45 rhGH + 100 rhIGF-1 | 100.2 | 41.4 |
| 45 rhGH + 150 rhIGF-1 | 96.7 | 38.8 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| 1. rhGH AloneNutropin AQ® (Somatropin \[rDNA origin\]): rhGH (Somatropin) 45µg/kg once daily injection | – | 1 / 26 | 26 / 26 |
| 2. 45 rhGH + 50 rhIGF-1Increlex® (Mecasermin \[rDNA origin\] injection) + Nutropin AQ® (Somatropin \[rDNA origin\]): rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections | – | 1 / 27 | 27 / 27 |
| 3. 45 rhGH + 100 rhIGF-1Increlex® (Mecasermin \[rDNA origin\] injection) + Nutropin AQ® (Somatropin \[rDNA origin\]): rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections | – | 2 / 27 | 27 / 27 |
| 4. 45 rhGH + 150 rhIGF-1Increlex® (Mecasermin \[rDNA origin\] injection) + Nutropin AQ® (Somatropin \[rDNA origin\]): rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection | – | 1 / 26 | 26 / 26 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 5 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
* Parents or legally authorized representatives must give signed informed consent before any trial-related activities
* IGF-1 SDS of ≤ -1 for age and gender
* Short stature, as defined by a height SDS of ≤ -2 for age and gender
* Chronological age ≥ 5 years
* Bone age ≤ 11 years in boys and ≤ 9 years in girls
* GH sufficiency, defined as a maximal stimulated GH response of greater than or equal to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a prior GH stimulation test may satisfy this requirement).
* Prepubertal status
* Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to the 5th percentile for age and gender
Exclusion Criteria:
* Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response greater than or equal to 10 ng/mL)
* Prior or current use of medications with the potential to alter growth patterns including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e.g., Lupron), aromatase inhibitors, androgens and estrogens
* Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations
* Current use of medications for attention deficit disorder
* A chronic health condition that requires anti-inflammatory steroids or daily medication unless approved by the Medical Monitor
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