Phase 4
Completed N=318
Predictive Markers in Growth Hormone Deficiency (GHD) and Turner Syndrome (TS) Children Treated With SAIZEN®
Source: ClinicalTrials.gov NCT00256126 ↗Enrolled (actual)
318
Serious AEs
0.3%
Results posted
Mar 2018
Primary outcomePrimary: Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 — 1.7692; 1.4007 Standard deviation score (SDS) — p=<0.0001
◆ Published Evidence
Established
36citations · ~3 / year
Pharmacogenomics of insulin-like growth factor-I generation during GH treatment in children with GH deficiency or Turner syndrome.
Summary
The study aims at identifying the predictive markers after one month of Saizen therapy in Growth Hormone Deficiency (GHD) and Turner Syndrome children.
Linked Publications (4)
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Pharmacogenomics of insulin-like growth factor-I generation during GH treatment in children with GH deficiency or Turner syndrome.
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Transcriptomics and machine learning predict diagnosis and severity of growth hormone deficiency.
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GH deficiency status combined with GH receptor polymorphism affects response to GH in children.
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Gene expression signatures predict response to therapy with growth hormone.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Insulin Like Growth Factor-1 Standard Deviation Score (IGF-1 SDS) at Month 1 |
1.7692; 1.4007 | <0.0001 sig |
| SECONDARY Change From Baseline in Insulin-like Growth Factor Binding Protein - 3 (IGFBP-3) Level at Month 1 |
0.86; 0.69 | — |
| SECONDARY Change From Baseline in Fasting Glucose Levels at Month 1 |
0.22; 0.13 | — |
| SECONDARY Change From Baseline in Fasting Insulin Levels at Month 1 |
47.7; 26.9 | — |
| SECONDARY Change From Baseline in Homeostasis Model Assessment of Insulin Resistance (HOMA-IR) at Month 1 |
2.132; 1.061 | — |
| SECONDARY Change From Baseline in Bone Alkaline Phosphatase Levels at Month 1 |
21.13; 14.78 | — |
Eligibility Criteria
Inclusion Criteria
- One of the following diagnoses and candidacy for SAIZEN® therapy:
A) GHD: documented pre-established diagnosis of GHD with a growth hormone (GH) peak response of 5th and <95th percentiles) for gender Willingness and ability to comply with the protocol for the duration of the study.
- Parent's or guardian's written informed consent, given before any study related procedure that is not part of the subject's normal medical care, with the understanding that the subject or parent/guardian may withdraw consent at any time without prejudice to future medical care. If the child is old enough to read and write, a separate assent form will be given.
Exclusion Criteria
- Acquired GHD due to central nervous system tumour, trauma, infection, infiltration (documented by imaging), and history of irradiation or cranial surgery
- Previous treatment with GH, growth hormone-releasing hormone (GHRH), anabolic steroids or any treatment affecting growth.
- Previous treatment with corticosteroids, except in case of topical or inhaled corticosteroid administration for atopic disease. Corticosteroids for hormonal substitution are also allowed if the condition and the treatment regimen have been stable for at least 3 months.
- Severe associated pathology affecting growth such as malnutrition, malabsorption, or bone dysplasia.
- Chronic severe kidney disease.
- Chronic severe liver disease.
- Chronic infectious disease.
- Acute or severe illness during the previous 6 months.
- Significant concomitant illness that would interfere with participation or assessment in this study.
- Active malignancy (except non-melanomatous skin malignancies that have undergone surgical excision and/or biopsy, diagnosis and treatment to resolution)
- History or active Idiopathic intra-cranial hypertension (benign intracranial hypertension or pseudo-tumor cerebri).
- Diabetes Mellitus type I & II.
- Any autoimmune disease.
- Previous screening failure in this study.
- Use of an investigational drug or participation in another clinical study within the last three months.
Data sourced from ClinicalTrials.gov (NCT00256126) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.