A Phase 4 interventional study of Nutropin AQ in Growth Hormone Deficiency, Septo-Optic Dysplasia and Hypopituitarism, sponsored by Children's Hospital Los Angeles. Completed at 1 site in United States. Open to participants aged Up to 5 Years. Per ClinicalTrials.gov, last updated 2015-04-01.
Sponsored by Children's Hospital Los Angeles · Phase 4, Interventional, and Treatment
Hypotheses:
Aims:
Subjects for this study will be recruited from active and newly enrolled subjects in our larger ONH study. The study duration is three years and we anticipate 38 subjects will enroll. Subjects will be recruited for this study if they present with either growth deceleration or at least one subnormal result for IGF-1 or IGFBP-3.
Baseline information collected includes: height, weight, head circumference, examinations by an endocrinologist and ophthalmologist, endocrine laboratory testing, fundus photography, electrophysiology testing, head MRI, and a developmental assessment. A glucagon stimulation test will be performed and subjects who are deemed GH-deficient and who have delayed growth will be assigned to GH treatment, in line with standard clinical practice. Those with normal growth but determined to be GH-deficient by a glucagon stimulation test will be randomized to treatment with GH vs control (no intervention; observation only).
Subjects assigned or randomized to treatment with GH will be provided with GH for the duration of their participation in the study. Enrolled subjects will return every four months to monitor progress. Subjects will undergo a physical examination at each visit, including height, weight, head circumference, and body fat. In addition, subjects assigned or randomized to growth hormone will have laboratory testing of thyroid, IGF-1 and IGFBP-3 hormones, and fasting lipid levels.
252 studies on the registry are indexed under Dwarfism, Pituitary; 30 are open to participants now.
This study's enrollment of 20 is below the median of 60 across 169 interventional studies indexed under Dwarfism, Pituitary.
Browse Dwarfism, Pituitary studies →Children's Hospital Los Angeles is the lead sponsor of 152 studies on the registry; 46 are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 6 (67%) have results posted.
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Inclusion Criteria:
Treatment group assignment was based on subject's stature SDS relative to the mid-parental target height (MPTH) at baseline and subsequent classification as growth deceleration or normal growth. Subjects with growth deceleration were assigned to the GH treatment group in accordance with standard of care. Subjects with normal growth were randomized to treatment or to control (no intervention). The intervention was Nutropin AQ. The starting dose was calculated as 0.3 mg/kg/wk and subsequently modified based on observed length/height velocity and serum IGF-I levels.
Drug: Nutropin AQ
Treatment group assignment was based on subject's stature SDS relative to the mid-parental target height (MPTH) at baseline and subsequent classification as growth deceleration or normal growth. Subjects with normal growth were randomized to treatment or to control. The control group received no intervention; however, control subjects were switched (crossed over) to the GH replacement group if, during the course of the study, they met criteria for growth deceleration.
Daily injection. Dosage dependent on weight.
Change in Anthropometric Measures Over Time
Primary outcome measures included change in stature, weight, BMI, and weight-for-stature z-scores over the course of the study. Z-score indicates how many standard deviations an element is from the mean. It is calculated as z = (x - µ) σ, where µ is the mean of the population, and σ is the standard deviation of the population. A positive z-score indicates a datum above the mean, while a negative z-score indicates a datum below the mean. All z-scores were obtained using Epi Info ™ 3.5.4. (Centers for Disease Control, Atlanta, GA).
Time frame: Baseline and 36 months
Participants were recruited from an IRB-approved prospective optic nerve hypoplasia (ONH) registry study at Children's Hospital Los Angeles, between January 2005 through March 2011.
| Milestone | Treatment Group 1: Receiving Growth Hormone Treatment | Treatment Group 2: Control |
|---|---|---|
| Started | 13 | 7 |
| Received at least one dose of gh | 13 | 3 |
| Completed | 10 | 7 |
| Not completed | 3 | 0 |
| Withdrew: Lost to follow-up | 1 | 0 |
| Withdrew: Withdrawal by subject | 2 | 0 |
Primary outcome measures included change in stature, weight, BMI, and weight-for-stature z-scores over the course of the study. Z-score indicates how many standard deviations an element is from the mean. It is calculated as z = (x - µ) σ, where µ is the mean of the population, and σ is the standard deviation of the population. A positive z-score indicates a datum above the mean, while a negative z-score indicates a datum below the mean. All z-scores were obtained using Epi Info ™ 3.5.4. (Centers for Disease Control, Atlanta, GA).
| Z-score | Treatment Group 1: Receiving Growth Hormone Treatment | Treatment Group 2: Control | Treatment Group 3: Control Crossed Over to Treatment |
|---|---|---|---|
| Change in stature z-score | 1.97 (0.56 to 2.29) | -0.61 (-0.9625 to -0.305) | 1.39 (0.62 to 1.43) |
| Change in weight z-score | 1.22 (0.49 to 2.16) | -0.38 (-0.715 to -0.195) | 0.58 (-0.06 to 1.92) |
| Change in weight-for-stature z-score | 0.52 (-0.21 to 0.705) | -0.37 (-0.825 to -0.1075) | 0.95 (0.1 to 1.8) |
| Change in body mass index (BMI) z-score | -0.06 (-0.24 to 0.39) | -0.505 (-0.7025 to -0.3075) | -1.035 (-1.1525 to -0.9175) |
Collected over 36 months (study duration). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment Group 1: Receiving Growth Hormone Treatment | — | 0/13 (0%) | 1/13 (7.7%) |
| Treatment Group 2: Control | — | 0/3 (0%) | 0/3 (0%) |
| Event | Treatment Group 1: Receiving Growth Hormone Treatment | Treatment Group 2: Control |
|---|---|---|
| HeadacheNervous system disorders | 1/13 | 0/3 |
Please note, 3 children were unenrolled from the study and were therefore excluded from our analysis of results. Therefore, while the participant flow section lists 20 participants, the baseline analysis and results are based on 17 participants.
| Age, Categorical(Participants) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| <=18 years | 5 | 5 | 7 | 17 |
| Between 18 and 65 years | 0 | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 | 0 |
| Age, Continuous(Months) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Median | 28 (24 to 30) | 41 (33 to 61) | 26 (23 to 50) | 30 (24 to 50) |
| Sex: Female, Male(Participants) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Female | 3 | 2 | 4 | 9 |
| Male | 2 | 3 | 3 | 8 |
| Weight z-score(Z-score) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Median | -1.76 (-2.48 to -1.6) | 0.6 (-0.47 to 1.49) | -1.36 (-1.73 to 0.41) | -1.36 (-1.73 to 0.6) |
| Stature z-score(Z-score) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Median | -2.16 (-2.35 to -1.94) | -0.95 (-1.73 to -0.22) | -1.3 (-1.79 to -0.91) | -1.73 (-2.16 to -0.95) |
| Weight-for-stature z-score(Z-score) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Median | -0.38 (-0.99 to -0.23) | 1.94 (0.88 to 2.49) | 1.34 (0.22 to 1.73) | 0.82 (-0.38 to 1.91) |
| Body Mass Index (BMI) z-score(Z-score) | Treatment Group 1A: Assigned to GH | Treatment Group 1B: Randomized to GH | Treatment Group 2: Control | Total |
|---|---|---|---|---|
| Median | -0.2 (-0.5 to 0.11) | 2.06 (1.14 to 2.62) | 1.35 (0.38 to 1.7) | 1.14 (0.05 to 2.06) |
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Studies of the drug tested here: Human Growth Hormone
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