Phase 3
N=90
A Phase 3 Study to Evaluate the Safety and Efficacy of Saizen® in Children With Idiopathic Short Stature (ISS)
Idiopathic Short Stature
Bottom Line
View on ClinicalTrials.gov: NCT01746862 ↗Enrolled (actual)
90
Serious AEs
4.5%
Results posted
Oct 2016
Primary outcome: Primary: Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method — 5.63; 4.94; 4.45; 1.01 centimeter/year (cm/yr) — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Saizen® (Drug)
- Age
- Pediatric, Adult, Older Adult · 5+ yrs
- Sex
- All
- Sponsor
- Merck KGaA, Darmstadt, Germany
- Primary completion
- Aug 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Height Velocity at Month 6 Using Last Observation Carried Forward (LOCF) Method |
5.63; 4.94; 4.45; 1.01 | <0.0001 sig |
| SECONDARY Change From Baseline in Height Velocity at Month 12 |
3.77; 2.86 | — |
| SECONDARY Change From Baseline in Height at Month 6 and 12 |
108.27; 108.07; 5.41; 3.10; 9.78; 8.24 | — |
| SECONDARY Change From Baseline in Height Standard Deviation Score (SDS) at Month 6 and 12 |
-2.26; -2.34; 0.59; 0.08; 0.96; 0.62 | — |
| SECONDARY Change From Baseline in Serum Concentration of Insulin-like Growth Factor-I (IGF-I) at Month 6 and 12 |
112.62; 125.01; 122.57; 8.30; 164.48; 142.17 | — |
| SECONDARY Change From Baseline in Serum Concentration of Insulin Like Growth Factor Binding Protein-3 (IGFBP-3) at Month 6 and 12 |
3,783.33; 3,935.67; 857.64; -38.89; 932.31; 545.56 | — |
| SECONDARY Percentage of Adherence to Study Treatment |
94.38; 95.69; 93.27; NA | — |
| SECONDARY Number of Subjects With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs |
42; 18; 3; 1 | — |
Summary
This is an open-label, multi-center, randomized, two-arm parallel, no-treatment group controlled (only for the first 6 months), Phase 3 study in children with ISS. The subjects will be treated with 0.067 milligram/kilogram/day (mg/kg/day) of Saizen®, weight base dose, for 12 months (12 months of treatment in the test group, and 6 months of no treatment and then 6 months of treatment in the control group).
Eligibility Criteria
Inclusion Criteria
- Age greater than or equal to 5 years
- Pre-pubertal; testicular volume less than 4 milliliter (in males) and breast Stage 1 (in females)
- The official records of height (for example records measured in hospitals or schools) during previous 6 months or more preceding inclusion in the study (self-measurement of the height at home will not be considered as a valid record)
- Height less than or equal to 3rd percentile compared to same sex, same age
- Peak serum growth hormone (GH) greater than 10 microgram per liter (mcg/L) in GH stimulation test (results of peak serum GH greater than 10 mcg/L in GH stimulation test within 1 year can be used instead)
- Naive to GH therapy
- Normal birth weight (that is greater than or equal to 3rd percentile when compared to same sex)
- Normal thyroid function
- Normal karyotype in girls
- Written informed consent from parent/guardian
- Written informed consent from the subject who speaks, understand, read, and write Korean
- Bone age less than 10 years in boys and less than 9 years in girls, whose difference between the bone and chronological age is no more than 3 years
Exclusion Criteria
- Puberty development (Tanner stage greater than or equal to 2)
- Skeletal dysplasia or abnormal body proportions
- Chronic systemic illness
- Dysmorphic syndrome
- Growth Hormone Deficiency
- Small for Gestational Age (SGA)
- Current medication for Attention deficit hyperactivity disorder (ADHD) or hyperactivity disorder
- Current medication with drugs that may influence secretion or action of growth hormone (such as estrogen, androgen, anabolic steroid, corticosteroid, thyroxine, aromatase inhibitors)
- Diabetes mellitus
- Kidney transplantation
- Acute critical illness, including complications following open heart surgery, abdominal surgery or multiple accidental trauma
- Acute respiratory failure
- Malignancy or previous therapy for malignancy
- Known hypersensitivity to somatotropin or any of its excipients including cresol or glycerol
- Closed epiphyses, progression or recurrence of an underlying intracranial tumor, chronic renal disease
- Endocrinologic or metabolic disorders such as Prader-Willi syndrome; Russel-Silver syndrome; Seckel syndrome; Down syndrome; Cushing syndrome; Noonan syndrome; short stature caused by other chromosomal abnormalities
- The disorders that explain short stature such as psychiatric disorders, nutritional disorders, and chronic debilitating diseases
- Participation in another clinical trial within the past 3 months
- Status of legal incapacity or limited legal capacity of the parents or legal guardian
Data sourced from ClinicalTrials.gov (NCT01746862). 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.