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Trial · Interventional

CompletedPhase 3Results postedRegistry data

Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency

Overview

Status
Completed
Phase
Phase 3
Enrollment
44 participants (actual)
Sponsor
OPKO Health, Inc. · Industry
Design
Randomized · Single masking
Countries
1 country

Japan

Start
7 Dec 2017
Primary completion
6 Mar 2020
Completion
6 Mar 2020
First posted
14 Mar 2019
Last update posted
12 Aug 2021
Conditions
Growth Hormone Deficiency

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
20172018201920202021
  1. Ph 3

    Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency

    NCT03874013

    7 Dec 2017 to 6 Mar 2020

Registry summary

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

Treatment of children with growth failure due to growth hormone deficiency (GHD). Primary • To evaluate the efficacy and safety of weekly MOD-4023 administration compared to daily Genotropin® administration in Japanese pre-pubertal children with GHD. Secondary • To evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) profiles of three different doses of MOD-4023 in Japanese pre-pubertal children with GHD.

Interventions

  • MOD-4023Drug
  • GenotropinDrug

Arms2

  • Experimental

    MOD-4023 Treatment Arm

    MOD-4023 (investigational treatment): weekly MOD-4023 SC injections for 12 months; initially over the first 6 weeks, MOD-4023 will be administered in 3 stepwise escalating doses (0.25 mg/kg/week, 0.48 mg/kg/week and 0.66 mg/kg/week), each for two weeks sequentially. For the remaining 46 weeks, patients will continue to receive MOD-4023 at a dose of 0.66 mg/kg/week.

  • Active comparator

    Genotropin Treatment Arm

    Genotropin® (reference treatment): daily Genotropin® (0.025 mg/kg/day).

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Annual Height Velocity (HV) After 12 Months

    Time frame · 12 months

    Registry description

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

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

    Time frame · 6 months

    Registry description

    Annualized height velocity (cm/year) for the MOD-4023 and the daily hGH treatment groups

  2. Change in Height Standard Deviation Score (SDS) Compared to Baseline After 12 Months

    Time frame · 12 months

    Registry description

    Change in height standard deviation score (SDS) for the MOD-4023 and the daily hGH treatment groups Height Standard Deviation Score (SDS) is the number of standard deviations above or below the mean height for age and sex. Height in cm is converted to height standard deviation score (SDS) by subtracting the mean and dividing by the SD. A higher Height Standard Deviation Score (SDS) indicates a better outcome

  3. Change in Bone Maturation (BM) After 12 Months

    Time frame · 12 months

    Registry description

    Change in bone maturation (bone age / chronological age) with the method of TW2 using a central bone age reader.

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

Annual Height Velocity (HV) After 12 Months

GroupValue (cm/year)Spread
MOD-4023 Treatment Arm9.781.59
Genotropin Treatment Arm7.781.14
Secondary and other outcome results(4)
Secondary outcomeMean· 6 months

Height Velocity at 6 Months

GroupValue (cm/year)Spread
MOD-4023 Treatment Arm10.542.04
Genotropin Treatment Arm8.341.84
Secondary outcomeMean· 12 months

Change in Height Standard Deviation Score (SDS) Compared to Baseline After 12 Months

GroupValue (Standard Deviation Score (SDS))Spread
MOD-4023 Treatment Arm0.970.41
Genotropin Treatment Arm0.500.23
Secondary outcomeMean· 12 months

Change in Bone Maturation (BM) After 12 Months

GroupValue (ratio of bone age to chronologic age)Spread
MOD-4023 Treatment Arm0.050.06
Genotropin Treatment Arm0.030.06
Other pre specified outcomeMedian· Baseline, Visit 6 (Month 3), Visit 7 (Month 6), Visit 8 (Month 9) and Visit 9 (12 months)

Biochemical

GroupValue (Standard Deviation Score)Spread
MOD-4023 Treatment Arm-1.4600.902
Genotropin Treatment Arm-1.4200.843

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
MOD-4023 Treatment ArmMOD-4023 (investigational treatment): weekly MOD-4023 SC injections for 12 months; initially over the first 6 weeks, MOD-4023 will be administered in 3 stepwise escalating doses (0.25 mg/kg/week, 0.48 mg/kg/week and 0.66 mg/kg/week), each for two weeks sequentially. For the remaining 46 weeks, patients will continue to receive MOD-4023 at a dose of 0.66 mg/kg/week. MOD-4023: MOD-4023 is a long-acting modified recombinant human growth hormone (r-hGH) which utilizes C-terminal peptide (CTP) technology. It will be provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a multi-dose disposable pre-filled PEN. MOD-4023 will be administered as a SC injection once weekly, using a delivery device.0 / 222 / 2222 / 22
Genotropin Treatment ArmGenotropin® (reference treatment): daily Genotropin® (0.025 mg/kg/day). Genotropin: Genotropin® is dispensed in a 2-chamber cartridge. The front compartment contains recombinant somatropin, glycine, mannitol, sodium dihydrogen phosphate anhydrous and disodium phosphate anhydrous. The rear compartment contains m-Cresol and mannitol in water for injections. A delivery device (GenotropinÒ) will be used for daily (evening/bedtime) SC administration of Genotropin® into the region of the upper arms, buttocks, thighs or abdomen (8 locations). Injection sites should be rotated. Dose regimen for Genotropin®: 0.025 mg/kg/day (or 0.175 mg/kg/w divided equally to 7 injections over a week).0 / 222 / 2217 / 22

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.

  • DerivedHorikawa R, Tanaka T, Hasegawa Y, Yorifuji T, Ng D, Rosenfeld RG, Hoshino Y, Okayama A, Ebata N, Hosoi M, Nakamuta S, Gomez R, Pastrak A, Castellanos O. Efficacy and safety of once-weekly somatrogon following up to 4 years of treatment in Japanese children with growth hormone deficiency: results from an open-label extension of a phase 3 study. Endocr J. 2026 Jan 6;73(1):21-32. doi: 10.1507/endocrj.EJ24-0625. Epub 2025 Oct 31. PMID 41183961

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 years old, and not yet 10 years for girls (9 years and 364 days) or not yet 11 years for boys (10 years and 364 days), on the date of ICF signature, with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency. 2. Confirmed diagnosis of GHD by 2 different types of GH provocation tests (standardized on growth foundation data): defined as a peak serum GH level of ≤ 6.0 ng/mL or ≤ 16 ng/mL when conducting GHRP-2 provocation test. Prior local laboratory results will be accepted subject to pre-approval by the study medical monitor and if the tests were conducted as specified in the protocol. 3. Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys. 4. Without prior exposure to any r-hGH therapy. 5. Height SD score ≤ -2.0 at screening 6. Impaired height velocity defined as: * Annualized height velocity (HV) below the 25th percentile for CA (HV \< -0.7 SDS) and gender according to the local primary care provider standard. * The interval between two height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion. 7. BMI must be within ±2 SDS of mean BMI for the chronological age and sex. 8. Baseline IGF-1 level of at least 1 SDS below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤ -1) according to the central laboratory reference values. A single re-test will be allowed (subject to discussion with the study medical monitor) if all other criteria are met. 9. Normal creatinine levels according to common practice reference ranges per age. 10. 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 to ICF signing 11. Normal 46 XX karyotype for girls. 12. Willing and able to provide written informed consent of the parent or legal guardian of the patient and written assent from pediatric patients (when applicable based on age and Japan regulation). Exclusion Criteria: 1. Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer. 2. History of radiation therapy or chemotherapy 3. Malnourished children defined as BMI \< -2 SDS for age and sex 4. Children with suspected psychosocial dwarfism by the discretion of the investigator 5. Children born small for gestational age (SGA - birth weight and/or birth length \< -2 SDS 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. Children with diabetes mellitus 9. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX (short stature homeobox) mutations/deletions and skeletal dysplasia's, with the exception of septo-optic dysplasia. 10. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, sex steroids, with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin \[DDAVP\]) 11. Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 µg/d of inhaled budesonide or equivalent as provided in Appendix J. 12. Major medical conditions and/or presence of contraindication to r-hGH treatment. 13. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis. 14. Drug substance or alcohol abuse. 15. Known hypersensitivity to the components of study medication. 16. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets. 17. The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct. 18. Participation in any other clinical trial within 30 days prior to screening and throughout the entire study period (including administration of investigational agent).

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