Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

NCT01592500 | Completed Phase 2 Results DMC

• 2 other identifiers: CP-4-0042011-004553-60
• Study Type: interventional
• Target: 56
• Locations: 3 countries
• Sites: 4

Brief Summary

This is a phase II, open label, active-controlled, randomized safety and dose finding study of different MOD-4023 dose levels compared to daily r-hGH therapy in pre-pubertal growth hormone deficient children.

Conditions

Pediatric Growth Hormone Deficiency

Keywords

GHD; r-hGH; Long-acting

Outcome Measures

Primary Outcomes (1)

• Annual Height Velocity

  Annual Height Velocity in cm/year measured after 12 months of treatment

  12 months of treatment

Secondary Outcomes (3)

• Height Velocity at 6 Months

  After 6 months of treatment

• Change in Height Standard Deviation Score (SDS)

  After 6 and 12 months of treatment

• Change in IGF-1 Standard Deviation Score

  Once monthly on day 4 after the last dose

Study Arms (4)

MOD-4023 low dose

EXPERIMENTAL

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

Drug: MOD-4023

MOD-4023 middle dose

EXPERIMENTAL

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

Drug: MOD-4023

MOD-4023 high dose

EXPERIMENTAL

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

Drug: MOD-4023

Genotropin

ACTIVE COMPARATOR

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Drug: Somatropin

Interventions

MOD-4023 DRUG

Once weekly subcutaneous injection

MOD-4023 high dose; MOD-4023 low dose; MOD-4023 middle dose

Somatropin DRUG

Once daily subcutaneous injection of Genotropin

Also known as: Genotropin

Genotropin

Eligibility Criteria

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

You may qualify if:

• Pre-pubertal child aged ≥ 3 yrs old and not above 10 years for girls or 11 years for boys with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
• Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤7 ng/ml, determined by central laboratory using a validated assay .
• Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
• Without prior exposure to any r-hGH therapy.
• Normal birth size, birth weight and length for gestational age
• Impaired height and height velocity defined as:
• Height (HT) of at least 2.0 standard deviations (SD) below the mean height for chronological age (CA) and gender according to the standards from Prader et. al, 1989 , (HT SDS ≤ -2.0).
• BMI must be within ±2 SD of mean BMI for the chronological age and sex according to the 2000 CDC standards.
• Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS ≤ -1.0) according to the central laboratory reference values;
• \. Written informed consent of the parent or legal guardian of the patient and assent of the patient (if the patient can read).

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 SD 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, 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 (4)

Children's Hospital "P. A. Kyriakou"

Athens, Greece

Location

Buda Children's Hospital

Budapest, Hungary

Location

Heim Pal Children's Hospital

Budapest, Hungary

Location

Children's University Hospital

Košice, Slovakia

Location

Related Publications (1)

• Zelinska N, Iotova V, Skorodok J, Malievsky O, Peterkova V, Samsonova L, Rosenfeld RG, Zadik Z, Jaron-Mendelson M, Koren R, Amitzi L, Raduk D, Hershkovitz O, Hart G. Long-Acting C-Terminal Peptide-Modified hGH (MOD-4023): Results of a Safety and Dose-Finding Study in GHD Children. J Clin Endocrinol Metab. 2017 May 1;102(5):1578-1587. doi: 10.1210/jc.2016-3547.

  PMID: 28323965 DERIVED

MeSH Terms

Interventions

MOD-4023; Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

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: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2012
• First Posted: May 7, 2012
• Study Start: February 1, 2012
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: January 23, 2020
• Results First Posted: January 23, 2020

Record last verified: 2020-01

Locations

GR (1); HU (2); SL (1)