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TerminatedNCT00572156Updated Mar 30, 2023Results posted

rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency

A Phase 2 interventional study of NutropinAq® (Somatropin [rDNA origin]) and Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin]) in Insulin-like Growth Factor-1 Deficiency, sponsored by Ipsen. Terminated at 1 site in United States. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2023-03-30.

Sponsored by Ipsen · Phase 2, Interventional, and Treatment

Why this study was terminated
The study was prematurely terminated due to strategic reasons.
Phase
Phase 2
Study type
Interventional
Enrollment
106
Allocation
Randomized
Ages
5 Years and older
Sex
All
01

Study summary

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.

02

Conditions studied

  • Insulin-like Growth Factor-1 Deficiency

Keywords

  • IGF-1 Deficiency
  • growth
  • ISS
  • Constitutional growth delay
  • Primary IGFD
  • Primary IGF Deficiency
03

In context

Lead sponsor

Ipsen is the lead sponsor of 288 studies on the registry; 16 are open to participants now.

Of its 26 completed or terminated interventional studies of FDA-regulated products, 21 (81%) have results posted.

Browse Ipsen studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
5 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
106 participants (actual)

Study arms

  • Active comparator
    1. rhGH Alone

    Drug: NutropinAq® (Somatropin [rDNA origin])

  • Experimental
    2. Combination Dose

    Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

  • Experimental
    3. Combination Dose

    Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

  • Experimental
    4. Combination Dose

    Drug: Increlex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

Interventions

  • DrugNutropinAq® (Somatropin [rDNA origin])

    rhGH (Somatropin) 45µg/kg once daily injection

    Also known as: GH

  • DrugIncrelex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

    Drug page: Mecasermin

    rhGH 45µg/kg and rhIGF-1 150µg/kg once daily injection

    Also known as: Increlex, rhIGF-1, GH

  • DrugIncrelex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

    Drug page: Mecasermin

    rhGH (Somatropin) 45µg/kg and rhIGF-1 (Mecasermin) 50µg/kg once daily injections

    Also known as: Increlex, rhIGF-1, GH

  • DrugIncrelex® (Mecasermin [rDNA origin] injection) + NutropinAq® (Somatropin [rDNA origin])

    Drug page: Mecasermin

    rhGH 45µg/kg and rhIGF-1 100µg/kg once daily injections

    Also known as: Increlex, rhIGF-1, GH

06

What researchers measure

Primary outcomes

  1. Height Velocity

    Time frame: First year of treatment

Secondary outcomes

  1. Height Velocity

    Time frame: Second, third and fourth year

  2. Cumulative Change in Height Standard Deviation Score (SDS)

    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)

    Time frame: First, second, third and fourth year

  3. Predicted Adult Height (PAH)

    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)

    Time frame: At baseline (Day 1), year 1,2,3 and 4

  4. Total Change From Baseline (Day 1) in BMI SDS

    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)

    Time frame: At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1)

  5. Skeletal Maturation

    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)

    Time frame: At baseline(day 1), year 1,2,3 and 4

  6. Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)

    Time frame: At Baseline (Day 1), Year 1,2,3 and 4

  7. 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

  8. 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

  9. 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

  10. 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

  11. 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

  12. Summary of Adverse Events With Number of Occurrences

    A Data Monitoring Committee (DMC) was established to monitor subject safety

    Time frame: Approximately up to 4 years.

07

Results

Posted Dec 15, 2015
Limitations and caveats
The study was prematurely terminated once the last subject had completed three years of treatment (compared to the six years planned in protocol Amendment) for strategic reasons.

Participant flow

Date of first enrolment: December 2007. Date of last completed: March 2012. This was a Phase II, multicenter, randomized, open-label, parallel-group, active treatment controlled, dose selection study conducted at 27 centers in the US.

Participant flow — Overall Study
Milestone45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Started26272726
Completed0101
Not completed26262725
Withdrew: Adverse event3151
Withdrew: Lost to follow-up1110
Withdrew: Protocol violation1202
Withdrew: Patient/parent decision1643
Withdrew: Sponsor decision20161719

Outcome measures

PrimaryHeight Velocity
Time frame:
First year of treatment
Reported as:
Mean · cm/y
Height Velocity
cm/yrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Height Velocity9.3 ± 1.710.1 ± 1.39.7 ± 2.511.2 ± 2.1
SecondaryHeight Velocity
Time frame:
Second, third and fourth year
Reported as:
Mean · cm/y
Height Velocity
cm/yrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Year 2 height velocity [n= 24,25,22,24]8.4 ± 1.39.1 ± 1.29.4 ± 1.610.1 ± 1.8
Year 3 height velocity [n= 22,22,20,22]8.0 ± 1.18.4 ± 1.08.9 ± 1.39.2 ± 1.5
Year 4 height velocity [n= 17,16,15,16]7.7 ± 0.98.1 ± 0.88.4 ± 1.28.3 ± 1.3
SecondaryCumulative Change in Height Standard Deviation Score (SDS)

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)

Time frame:
First, second, third and fourth year
Reported as:
Mean · SDS
Cumulative Change in Height Standard Deviation Score (SDS)
SDSrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
First year [n= 25,27,27,26]0.7 ± 0.30.9 ± 0.20.8 ± 0.41.0 ± 0.4
Second year [n= 24,25,22,24]1.0 ± 0.41.4 ± 0.41.4 ± 0.51.6 ± 0.6
Third year [n=22,22,20,22]1.3 ± 0.51.6 ± 0.41.8 ± 0.71.9 ± 0.6
Fourth year [n=17,16,15,16]1.5 ± 0.51.8 ± 0.52.1 ± 0.82.0 ± 0.7
SecondaryPredicted Adult Height (PAH)

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)

Time frame:
At baseline (Day 1), year 1,2,3 and 4
Reported as:
Mean · SDS
Predicted Adult Height (PAH)
SDSrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Baseline (day 1) [n=25,27,27,26]-1.67 ± 0.51-1.83 ± 0.58-1.69 ± 0.59-1.76 ± 0.73
Year 1 [n= 24,25,22,25]-1.12 ± 0.57-1.22 ± 0.58-0.99 ± 0.55-1.01 ± 0.80
Year 2 [n= 22,21,20,22]-0.96 ± 0.56-0.81 ± 0.55-0.66 ± 0.56-0.69 ± 0.91
Year 3 [n=21,19,19,20]-0.78 ± 0.63-0.65 ± 0.60-0.42 ± 0.62-0.64 ± 0.98
Year 4 [n=3,4,4,3]-1.22 ± 0.59-0.44 ± 0.810.49 ± 0.60-0.07 ± 0.89
Mid-parental target height [n=25,27,27,26]-0.51 ± 0.38-0.64 ± 0.52-0.53 ± 0.50-0.47 ± 0.51
SecondaryTotal Change From Baseline (Day 1) in BMI SDS

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)

Time frame:
At year 1,2,3,4 and end of study (visit 23) versus baseline (day 1)
Reported as:
Mean · SDS
Total Change From Baseline (Day 1) in BMI SDS
SDSrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Change from day 1 to year 1 [n= 24,25,22,25]0.24 ± 0.430.31 ± 0.390.36 ± 0.410.54 ± 0.40
Change from day 1 to year 2 [n= 22,21,20,22]0.31 ± 0.490.57 ± 0.410.49 ± 0.440.59 ± 0.46
Change from day 1 to year 3 [n=21,19,19,20]0.35 ± 0.580.62 ± 0.430.49 ± 0.590.71 ± 0.54
Change from day 1 to year 4 [n=3,5,5,4]0.27 ± 0.520.64 ± 0.580.65 ± 0.901.20 ± 0.91
Change from day 1 to visit 23 [n=25,27,27,26]0.34 ± 0.540.45 ± 0.450.42 ± 0.530.64 ± 0.60
SecondarySkeletal Maturation

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)

Time frame:
At baseline(day 1), year 1,2,3 and 4
Reported as:
Mean · SDS
Skeletal Maturation
SDSrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Baseline [n=25,27,27,26]7.6 ± 1.87.3 ± 1.97.2 ± 2.07.4 ± 2.0
Year 1 [n= 24,25,22,25]9.0 ± 1.98.5 ± 1.98.4 ± 2.28.5 ± 2.1
Year 2 [n= 22,21,20,22]10.3 ± 2.09.6 ± 2.19.8 ± 2.49.9 ± 2.2
Year 3 [n=21,19,19,20]11.4 ± 2.010.9 ± 2.211.4 ± 2.410.9 ± 2.2
Year 4 [n=3,4,5,3]12.4 ± 1.010.9 ± 1.812.2 ± 3.012.5 ± 2.1
SecondaryChanges From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)
Time frame:
At Baseline (Day 1), Year 1,2,3 and 4
Reported as:
Mean · ng/mL
Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone (GH)
ng/mLrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n=23,25,18,25]1.1 ± 2.22.9 ± 8.12.4 ± 4.51.9 ± 2.3
Year 1 (Trough) [n=23,25,18,25]1.7 ± 2.13.5 ± 12.92.1 ± 3.21.4 ± 2.1
Year 2 (Trough) [n=21,20,16,20]4.0 ± 6.02.2 ± 4.22.7 ± 3.91.4 ± 1.2
Year 3 (Trough) [n=20,18,15,20]2.8 ± 5.11.3 ± 1.61.5 ± 1.41.9 ± 3.5
Year 4 (Trough) [n=3,5,4,3]1.2 ± 0.30.5 ± 0.43.0 ± 2.91.1 ± 0.6
SecondaryChanges 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
Reported as:
Mean · ng/mL
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor-1 (IGF-1)
ng/mLrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n= 24,25,22,25]108.3 ± 39.887.1 ± 31.0100.2 ± 41.496.7 ± 38.8
Year 1 (Trough) [n= 24,25,22,25]278.2 ± 95.5326.9 ± 108.9397.8 ± 185.4296.0 ± 126.8
Year 2 (Trough) [n= 22,21,20,22]312.8 ± 97.2427.2 ± 178.5539.3 ± 195.9466.4 ± 178.6
Year 3 (Trough)[n= 21,19,19, 20]335.3 ± 110.0404.9 ± 104.4462.2 ± 156.4441.3 ± 226.0
Year 4 (Trough)[n= 3,5,5,3]347.7 ± 130.7363.6 ± 125.4546.2 ± 265.8607.7 ± 65.0
SecondaryChanges 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
Reported as:
Mean · ng/mL
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-1 (IGFBP-1)
ng/mLrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n= 24,24,21,24]87.8 ± 54.5102.5 ± 37.295.7 ± 50.0122.4 ± 53.7
Year 1 (Trough) [n= 24,24,21,24]45.7 ± 38.063.8 ± 50.168.2 ± 61.688.8 ± 47.4
Year 2 (Trough) [n= 22,21,19,20]59.4 ± 45.658.2 ± 47.753.3 ± 35.371.7 ± 46.1
Year 3 (Trough) [n= 21,19,18,19]46.1 ± 35.260.4 ± 52.553.2 ± 34.884.7 ± 53.5
Year 4 (Trough) [n= 3,5,5,3]100.0 ± 60.446.8 ± 58.049.2 ± 20.749.0 ± 26.9
SecondaryChanges 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
Reported as:
Mean · ng/mL
Changes From Baseline (Day 1) in Serum Concentrations of Insulin-Like Growth Factor Binding Protein-3 (IGFPB-3)
ng/mLrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n= 24,25,22,25]2204.2 ± 387.32160.0 ± 421.32113.6 ± 486.32196.0 ± 612.0
Year 1 (Trough) [n= 24,25,22,25]2954.2 ± 618.52784.0 ± 755.92677.3 ± 548.52624.0 ± 598.8
Year 2 (Trough) [n= 22,21,20,22]2813.6 ± 399.22942.9 ± 552.83060.0 ± 703.72754.5 ± 600.6
Year 3 (Trough) [n= 21,19,19,20]3404.8 ± 476.93431.6 ± 718.13373.7 ± 479.43260.0 ± 908.1
Year 4 (Trough) [n= 3,5,5,3]3733.3 ± 378.63260.0 ± 709.23620.0 ± 957.63466.7 ± 461.9
SecondaryChanges From Baseline (Day 1) in Serum Concentrations of Acid-Labile Subunit (ALS)
Time frame:
At Baseline (Day 1), Year 1,2,3 and 4
Reported as:
Mean · mg/L
Changes From Baseline (Day 1) in Serum Concentrations of Acid-Labile Subunit (ALS)
mg/LrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n=25,26,26,25]10.6 ± 3.010.0 ± 3.410.9 ± 3.011.0 ± 4.1
Year 1 (Trough) [n=24,24,21,24]16.0 ± 4.012.7 ± 2.314.0 ± 3.911.5 ± 2.8
Year 2 (Trough) [n= 22,21,19,20]15.2 ± 4.314.3 ± 5.215.7 ± 4.013.7 ± 4.5
Year 3 (Trough) [n= 21,18,18,19]13.7 ± 2.313.0 ± 3.212.9 ± 2.711.2 ± 4.0
Year 4 (Trough) [n= 3,5,5,3]13.7 ± 1.512.0 ± 2.212.0 ± 3.013.7 ± 2.1
SecondaryChanges 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
Reported as:
Mean · pmol/L
Changes From Baseline (Day 1) in Serum Concentrations of Growth Hormone Binding Protein (GHBP)
pmol/LrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Day 1 (Baseline) [n= 25,26,25,25]785.8 ± 291.1735.8 ± 343.5698.8 ± 212.2719.0 ± 261.5
Year 1 (Trough) [n= 23,24,20,24]729.4 ± 229.5576.9 ± 211.0598.9 ± 187.3530.5 ± 233.5
Year 2 (Trough) [n=22,21,18,21]725.6 ± 308.4589.3 ± 171.6562.5 ± 184.4564.6 ± 171.9
Year 3 (Trough) [n= 21,17,17,19]672.0 ± 224.4646.9 ± 204.1614.3 ± 210.9554.9 ± 197.0
Year 4 (Trough) [n= 3,5,5,3]714.0 ± 38.5516.9 ± 252.5798.2 ± 336.1519.7 ± 141.1
SecondarySummary of Adverse Events With Number of Occurrences

A Data Monitoring Committee (DMC) was established to monitor subject safety

Time frame:
Approximately up to 4 years.
Reported as:
Number · Number of events
Summary of Adverse Events With Number of Occurrences
Number of eventsrhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1
Any Adverse Event381416401518
Any TEAE374406392513
Severe TEAEs5533
Moderate TEAE60555786
Mild TEAE309346332424
Related TEAEs275374119
Related and Severe1110
Related and Moderate541813
Related and Mild214855106
TEAEs Leading to Study Withdrawal1817366
SAEs1621
Deaths0000

Adverse events

Collected over Approximate up to 4 years. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
1. rhGH Alone—1/26 (3.8%)26/26 (100%)
2. 45 rhGH + 50 rhIGF-1—1/27 (3.7%)27/27 (100%)
3. 45 rhGH + 100 rhIGF-1—2/27 (7.4%)27/27 (100%)
4. 45 rhGH + 150 rhIGF-1—1/26 (3.8%)26/26 (100%)
Most frequent serious events
Most frequent serious events
Event1. rhGH Alone2. 45 rhGH + 50 rhIGF-13. 45 rhGH + 100 rhIGF-14. 45 rhGH + 150 rhIGF-1
Gastroenteritis viralInfections and infestations1/260/270/270/26
PapilledemaEye disorders0/260/270/271/26
Intracranial pressure increasedNervous system disorders0/260/271/270/26
Forearm fractureInjury, poisoning and procedural complications0/260/271/270/26
Evans syndromeBlood and lymphatic system disorders0/261/270/270/26
ThrombocytopeniaBlood and lymphatic system disorders0/261/270/270/26
Viral infectionInfections and infestations0/261/270/270/26
HematuriaRenal and urinary disorders0/261/270/270/26
Most frequent other events
Showing 10 of 97
Most frequent other events
Event1. rhGH Alone2. 45 rhGH + 50 rhIGF-13. 45 rhGH + 100 rhIGF-14. 45 rhGH + 150 rhIGF-1
HeadacheNervous system disorders14/2614/2717/2718/26
PyrexiaGeneral disorders9/2615/274/2713/26
CoughRespiratory, thoracic and mediastinal disorders6/2614/279/2710/26
Upper respiratory tract infectionInfections and infestations10/2610/279/2713/26
VomitingGastrointestinal disorders9/265/276/2712/26
Pharyngitis streptococcalInfections and infestations5/2612/277/276/26
Otitis mediaInfections and infestations4/265/273/279/26
Viral infectionInfections and infestations6/263/276/278/26
Pain in extremityMusculoskeletal and connective tissue disorders4/268/277/275/26
Abdominal pain upperInfections and infestations7/265/273/277/26

Baseline characteristics

All Subjects

Age, Continuous
Age, Continuous(years)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean9.2 ± 2.08.4 ± 2.09.0 ± 2.28.8 ± 2.38.8 ± 2.1
Sex: Female, Male
Sex: Female, Male(Participants)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Female557421
Male2122202285
Race
Race(participants)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Asian24118
Black11103
Hispanic413210
White1720192379
Hispanic/White11305
Hawaiian or Pacific Islander/ White10001
Height
Height(Standard Deviation Score (SDS))45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean-2.5 ± 0.5-2.5 ± 0.4-2.6 ± 0.4-2.6 ± 0.4-2.5 ± 0.4
Weight
Weight(SDS)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean-2.0 ± 0.7-2.1 ± 0.6-2.0 ± 0.7-2.2 ± 0.9-2.1 ± 0.7
Body Mass Index (BMI)
Body Mass Index (BMI)(SDS)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean-0.3 ± 0.7-0.5 ± 0.7-0.4 ± 0.7-0.5 ± 0.8-0.4 ± 0.7
IGF-1
IGF-1(SDS)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean-1.8 ± 0.5-2.0 ± 0.7-1.8 ± 0.6-2.0 ± 0.6-1.9 ± 0.6
Mid-Parental target height
Mid-Parental target height(SDS)45 rhGH Alone45 rhGH + 50 rhIGF-145 rhGH + 100 rhIGF-145 rhGH + 150 rhIGF-1Total
Mean-0.5 ± 0.4-0.6 ± 0.5-0.5 ± 0.5-0.5 ± 0.5-0.5 ± 0.5

6 further baseline measures are reported on the registry.

08

Study locations

1 site
  • Ipsen
    Brisbane, California 94005, United States
09

Timeline

Tracking since Sep 25, 2026
Not marked actual or estimatedDates not marked actual or estimated

The record gives a start of Dec 2007, a primary completion of Apr 2010 and a completion of Mar 2012. It does not say whether these dates are actual or estimated.

Results were first posted in Dec 2015, 5 years 8 months after primary completion. A results delay was requested in Apr 2011.

How these figures are worked out, and which studies are left out of them.

Registered
Dec 10, 2007
First posted
Dec 12, 2007
Start
Dec 2007
Primary completionActual
Apr 2010
CompletionActual
Mar 2012
Results delay requested
Apr 18, 2011
Results first posted
Dec 15, 2015
Last update
Mar 30, 2023
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Mar 30, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00572156
Lead sponsor
Ipsen
Responsible party
Sponsor
First posted
Dec 12, 2007
Start date
Dec 2007
Primary completion
Apr 2010
Completion
Mar 2012
Results posted
Dec 15, 2015
Last update
Mar 30, 2023

Study contacts

Ipsen Medical Director
study director · Ipsen (formerly Tercica, Inc.)

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Mar 2023. You cannot join it, but the record below documents what was studied.

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