CP-4-006: A Phase 3, Open-Label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin Therapy in Pre-pubertal Children with Growth Hormone Deficiency
Clinical Trial Information
Trial Contact: Tilme, Linda; Parrimon, Yozandra
IRB No: 16.131.10
Principal Investigator: Paul M. Desrosiers, MD
Sub Investigators: Banks, Richard MD; Yang, Joshua MD; Villar, Jennifer MD
Phase: Drug: Phase III
Age Group: Pediatric
Secondary Protocol No: OPKO CP-4-006
Applicable Disease Sites: Growth Hormone Deficiency, Endocrinology
Therapies Involved: MOD-4023 and Genotropin
To demonstrate that weekly MOD-4023 administration is non-inferior to daily Genotropin administration in terms of safety and efficacy outcomes
1. Pre-pubertal children aged ≥3 yrs old and not yet 11 years for girls (10 years and 364 days) or not yet 12 years (11 years and 364 days) for boys (on the date of ICF signature) with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiencies.
2. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤10 ng/ml, determined by local (if done prior to signing the ICF) or central laboratory using a validated assayb. Prior local laboratory results will be accepted subject to pre-approval by Sponsor Global Medical Monitor and if the tests were conducted according to one of the protocols
3. Bone age (BA) is not older than chronological age and should be less than 10 for females and less than 11 for males.
4. Without prior exposure to any r-hGH therapy.
5. Impaired height and height velocity defined as:
Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS) and gender according to one of the following standards: Prader HV standard tables, Tanner HV curves, or the standard in use by local primary care provider.
The interval between two height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion.
6. BMI must be within ±2 SDS of mean BMI for the chronological age and sex.
7. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS ≤ -1)a according to the central laboratory reference values. A single re-test will be allowed (subject to discussion with medical monitor) if all other criteria are met.
8. Normal calculated GFR based on updated “bedside” Schwartz formula for pediatric patients (recommended calculation is provided below):
CrCL (mL/min/1.73 m2) =0.413 * Ht / Scr
Ht: height in cm;
Scr: serum creatinine in mg/dL;
9. Children with multiple hormonal deficiencies must be on stable replacement therapies (no change in dose) for other hypothalamo-pituitary-organ axes for at least 3 months prior ICF signing
10. Normal 46XX karyotype for girls.
11. Willing and able to provide written informed consent of the parent or legal guardian of the patient and written assent from pediatric patients (where applicable based on age and country regulation).