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Trial · Interventional

CompletedPhase 2Results postedRegistry data

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

NCTNCT01592500 (opens ClinicalTrials.gov in a new tab)Other idCP-4-004Other id2011-004553-60

Overview

Status
Completed
Phase
Phase 2
Enrollment
56 participants (actual)
Sponsor
OPKO Health, Inc. · Industry
Design
Randomized · Open label (no masking)
Countries
3 countries

Greece, Hungary, Slovakia

Start
Feb 2012
Primary completion
Jul 2015
Completion
Jul 2015
First posted
7 May 2012 (estimated)
Last update posted
23 Jan 2020
Conditions
Pediatric Growth Hormone Deficiency

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20122013201420152016
  1. Ph 2

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

    NCT01592500

    Feb 2012 to Jul 2015

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • MOD-4023Drug
  • SomatropinDrug

Arms4

  • Experimental

    MOD-4023 low dose

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

  • Experimental

    MOD-4023 middle dose

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

  • Experimental

    MOD-4023 high dose

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

  • Active comparator

    Genotropin

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

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Annual Height Velocity

    Time frame · 12 months of treatment

    Registry description

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

Secondary outcomes(3)
  1. Height Velocity at 6 Months

    Time frame · After 6 months of treatment

    Registry description

    Annualized Height Velocity in cm/year measured after 6 months of treatment

  2. Change in Height Standard Deviation Score (SDS)

    Time frame · After 6 and 12 months of treatment

    Registry description

    Change in height standard deviation score from baseline (compared to normal population of same age group and sex). Height SDS was calculated as height minus reference mean height divided by SD of the reference mean height

  3. Change in IGF-1 Standard Deviation Score

    Time frame · Once monthly on day 4 after the last dose

    Registry description

    Change in IGF-1 standard deviation score from reference population mean of same age group and sex (WHO source). IGF-1 SDS was calculated as IGF-1 result minus reference mean IGF-1 result divided by SD of the reference mean IGF-1 value.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMean· 12 months of treatment

Annual Height Velocity

GroupValue (cm/year)Spread
MOD-4023 Low Dose10.42.6
MOD-4023 Middle Dose11.02.3
MOD-4023 High Dose11.93.5
Genotropin12.52.1
Secondary and other outcome results(3)
Secondary outcomeMean· After 6 months of treatment

Height Velocity at 6 Months

GroupValue (cm/year)Spread
MOD-4023 Low Dose11.83.6
MOD-4023 Middle Dose12.52.4
MOD-4023 High Dose13.55.0
Genotropin15.02.9
Secondary outcomeMean· After 6 and 12 months of treatment

Change in Height Standard Deviation Score (SDS)

GroupValue (score on a scale)Spread
MOD-4023 Low Dose0.650.36
MOD-4023 Middle Dose0.750.25
MOD-4023 High Dose0.900.39
Genotropin1.000.35
Secondary outcomeMean· Once monthly on day 4 after the last dose

Change in IGF-1 Standard Deviation Score

GroupValue (score on a scale)Spread
MOD-4023 Low Dose-1.860.817
MOD-4023 Middle Dose-1.940.899
MOD-4023 High Dose-2.130.887
Genotropin-2.120.813

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
MOD-4023 Low DoseOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)–0 / 1310 / 13
MOD-4023 Middle DoseOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)–0 / 1510 / 15
MOD-4023 High DoseOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)–0 / 1410 / 14
GenotropinOnce daily subcutaneous injection of Somatropin (r-hGH; Genotropin)–0 / 118 / 11

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • DerivedMauras N, Skorodok Y, Iotova V, Rosenfeld R, Wajnrajch MP, Malievskiy O, Zelinska N, Valluri SR, Zadik Z. Open-Label 9-Year Follow-Up Extension Phase 2 Study of Once-Weekly Somatrogon in Children With Growth Hormone Deficiency. J Clin Endocrinol Metab. 2026 Jun 29:dgag260. doi: 10.1210/clinem/dgag260. Online ahead of print. PMID 42372104
  • DerivedZelinska 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

Eligibility

Who could join, as posted.

Sex
All
Age
from 3 Years up to 11 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. 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. 2. 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 . 3. Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys. 4. Without prior exposure to any r-hGH therapy. 5. Normal birth size, birth weight and length for gestational age 6. Impaired height and height velocity defined as: 1. 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). 2. Annualized height velocity (HV) below the 25th percentile for CA (HV \<-0.7 SDS) and gender according to the standards of Prader et al (1989). The interval between two height measurements should be at least 6 months before inclusion. 7. BMI must be within ±2 SD of mean BMI for the chronological age and sex according to the 2000 CDC standards. 8. 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; 9. 12\. Written informed consent of the parent or legal guardian of the patient and assent of the patient (if the patient can read). 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 SD 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, 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.

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