Overview

This trial is active, not recruiting.

Condition somatotropin deficiency
Treatment mod-4023
Phase phase 2
Sponsor Opko Biologics
Start date February 2013
End date December 2020
Trial size 47 participants
Trial identifier NCT02500316, 2011-004553-60, CP-4-004-extension

Summary

Protocol CP-4-004-EXT is designed as an open-label extension study assessing long-term MOD-4023 administration. It is open to subjects completing Phase 2 study in children with growth hormone deficiency (CP-4-004).

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
mod-4023
Once weekly subcutaneous injection
(Experimental)
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
mod-4023
Once weekly subcutaneous injection
(Experimental)
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
mod-4023
Once weekly subcutaneous injection

Primary Outcomes

Measure
Annual Height Velocity
time frame: 5 Years

Eligibility Criteria

Male or female participants from 3 years up to 11 years old.

Inclusion Criteria: Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study. Exclusion Criteria: 1. Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast). 2. History of radiation therapy or chemotherapy. 3. Malnourished children defined as: 1. Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory; 2. Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory; 3. BMI < -2 Standard Deviation for age and sex; 4. Children with psychosocial dwarfism. 5. Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age). 6. Presence of anti-hGH antibodies at screening. 7. Any clinically significant abnormality likely to affect growth or the ability to evaluate growth, such as, but not limited to, chronic diseases like renal insufficiency, spinal cord irradiation, etc. 8. Patients with diabetes mellitus. 9. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or 6.1 mmol/l) after repeated blood analysis. 10. Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, short stature homeobox-containing gene (SHOX) mutations/deletions and skeletal dysplasias), with the exception of septo-optic dysplasia. 11. Closed epiphyses. 12. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder (ADHD), with the exception of hormone replacement therapies (thyroxine, hydrocortisone, desmopressin (DDAVP)) 13. Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month during a calendar year. 14. Major medical conditions and/or presence of contraindication to r-hGH treatment. 15. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. 16. Drug, substance, or alcohol abuse. 17. Known hypersensitivity to the components of study medication. 18. Other causes of short stature such as coeliac disease, hypothyroidism and rickets. 19. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. 20. Participation in any other trial of an investigational agent within 30 days prior to Screening.

Additional Information

Official title Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
Principal investigator Zvi Zadik, MD
Trial information was received from ClinicalTrials.gov and was last updated in August 2016.
Information provided to ClinicalTrials.gov by Opko Biologics.