Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

NCT02500316 | Completed Phase 2 Results DMC

• 2 other identifiers: CP-4-004-OLE2011-004553-60
• Study Type: interventional
• Target: 48
• Locations: 6 countries
• Sites: 13

Brief Summary

Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.

Conditions

Growth Hormone Deficiency (GHD)

Keywords

Growth hormone deficiency (GHD); r-hGH; Long-acting

Outcome Measures

Primary Outcomes (2)

• Annualized Height Velocity

  A summary of the annualized HV at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5).

  8 years

• Delta Height SDS Every 12 Months

  A summary of annual change in height SDS at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5). Height SDS (Standard Deviation Score) reflects how an individual's height compares to the average for their age and sex, calculated using Growth Analyzer and based on Switzerland 1989 (Prader) reference. A z-score of 0 indicates a height equal to the population mean, while positive scores indicate above-average heights and negative scores reflect below-average heights.

  8 years

Secondary Outcomes (1)

• Summary of IGF-1 SDS

  8 years

Study Arms (1)

MOD-4023

EXPERIMENTAL

Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).

Drug: MOD-4023

Interventions

MOD-4023 DRUG

Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).

MOD-4023

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study.

You may not qualify if:

• Children with past or present intracranial tumor growth as confirmed by an MRI scan (with contrast).
• History of radiation therapy or chemotherapy.
• Malnourished children defined as:
• Serum albumin below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
• Serum iron below the lower limit of normal (LLN) according to the reference ranges of central laboratory;
• BMI \< -2 Standard Deviation for age and sex;
• Children with psychosocial dwarfism.
• Children born small for gestational age (SGA - birth weight and/or birth length \< -2 SD for gestational age).
• Presence of anti-hGH antibodies at screening.
• 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.
• Patients with diabetes mellitus.
• Patients with impaired fasting sugar (based on WHO; fasting blood sugar \>110 mg/dl or 6.1 mmol/l) after repeated blood analysis.
• 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.
• Closed epiphyses.
• 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))

Sponsors & Collaborators

• OPKO Health, Inc.: lead

Study Sites (13)

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

Location

2DKB

Minsk, Belarus

Location

Children's Hospital "P. A. Kyriakou"

Athens, Greece

Location

Buda Children's Hospital

Budapest, Hungary

Location

Endocrinology Scientific Centre, Institute of Child Endocrinology

Moscow, Russia

Location

Russian Medical Academy of Postgraduate Education

Moscow, Russia

Location

SPGPMA

Saint Petersburg, Russia

Location

SamGMU

Samara, Russia

Location

SBEIHPE

Ufa, Russia

Location

Donetsk Regional Children Clinical Hospital

Donetsk, Ukraine

Location

Institute of Endocrinology

Kiev, Ukraine

Location

Ukrainian Scientific Center of Endocrine Surgery Moh of Ukraine

Kiev, Ukraine

Location

Odessa Regional Children'S Clinical Hospital

Odesa, Ukraine

Location

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

MOD-4023

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Results Point of Contact

• Title: OPKO Health Inc
• Organization: OPKO Health Inc

contact@opko.com

3055754100

Study Officials

• Zvi Zadik, MD

  Kaplan Medical Center, Israel

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 12, 2015
• First Posted: July 16, 2015
• Study Start: February 1, 2014
• Primary Completion: November 1, 2023
• Study Completion: November 1, 2023
• Last Updated: April 9, 2025
• Results First Posted: April 9, 2025

Record last verified: 2025-04

Locations

US (1); BE (1); HU (1); RU (5); UA (4); GR (1)