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MOD-4023

Phase 3

Growth Hormone Deficiency | Small molecule | Endocrine |Opko Health Inc.|Last Updated: Jul 20, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials2
Total Enrollment86

FDA Designations

No designations recorded

Clinical trial landscape

MOD-4023 · 7 trials · 5 indications

Phase 3 2Phase 2 3Phase 1 2
NCT03874013Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone DeficiencyGrowth Hormone Deficiency
COMPLETED44 Analytics
NCT02968004Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient ChildrenPediatric Growth Hormone Deficiency
COMPLETED224 Analytics
PHASE3COMPLETED
Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency
Growth Hormone DeficiencyUnlock trial analytics
PHASE3COMPLETED
Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Pediatric Growth Hormone DeficiencyUnlock trial analytics

Study Endpoints

Primary Endpoints

Annual Height Velocity (HV) After 12 Months
12 months

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

Annual Height Velocity in Main Study Phase
52 weeks

Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.

Annual Height Velocity in OLE Phase
Up to 60 months

Annualized Height Velocity (cm/year) in OLE Phase

Annualized Height Velocity
8 years

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).

Delta Height SDS Every 12 Months
8 years

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.

Annual Height Velocity
12 months of treatment

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

1. Safety and Tolerability
Following 4 weeks of MOD-4023 treatment

Adverse events (AE's), vital signs, electrocardiogram (ECG), laboratory tests, local reaction

Maintenance of normal Insulin-like-Growth-Factor-1 (IGF-1)levels in GHDA
Following 4 weeks MOD-4023 treatment

The primary efficacy endpoint will be the mean time interval of IGF-I levels that lay within ±1.5 SDS after the last dose administration during stage I (4w treatment) expressed in hours.

Composite safety and tolerability parameters as measured by adverse events, electrocardiograms (ECG), laboratory results, vital signs and injection site reactions
30 days

To assess the safety, tolerability and immunogenicity of MOD-4023 in healthy Caucasian and Japanese subjects after single subcutaneous (SC) dose.

Pharmacokinetics (PK) profile of MOD-4023 in healthy Caucasian and Japanese subjects after single SC dose
7 days

MOD-4023 serum levels (T1/2, Area-Under-the-Curve, Cmax, Tmax)

Safety and Tolerability (AE's, vital signs, ECG, laboratory tests, local reaction)
30 days
Pharmacokinetics: AUC, Cmax, Tmax, T1/2
14 days

Secondary Endpoints

Height Velocity at 6 Months
6 months
Change in Height Standard Deviation Score (SDS) Compared to Baseline After 12 Months
12 months
Change in Bone Maturation (BM) After 12 Months
12 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeOTHER

Treatment Arms

ArmTypeDescription
MOD-4023 Treatment ArmEXPERIMENTALMOD-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.
Genotropin Treatment ArmACTIVE_COMPARATORGenotropin® (reference treatment): daily Genotropin® (0.025 mg/kg/day).
MOD-4023EXPERIMENTALOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)
GenotropinACTIVE_COMPARATOROnce daily subcutaneous injection of somatropin (r-hGH; Genotropin)
MOD-4023 low doseEXPERIMENTALOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)
MOD-4023 middle doseEXPERIMENTALOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)
MOD-4023 high doseEXPERIMENTALOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)
Weekly low doseEXPERIMENTALMOD-4023
Weekly middle doseEXPERIMENTALMOD-4023
Weekly high doseEXPERIMENTALMOD-4023
Every-other week doseEXPERIMENTALMOD-4023
Low doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
Mid doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
High doseEXPERIMENTAL7 Japanese and 7 Caucasian subjects (randomized 6:1) administered a single dose of MOD-4023/Placebo
MOD-4023, dose level 1EXPERIMENTAL -
MOD-4023, dose level 2EXPERIMENTAL -
MOD-4023, dose level 3EXPERIMENTAL -

Interventions

NameTypeDescription
MOD-4023DRUGMOD-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.
GenotropinDRUGGenotropin® 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 (Genotropin®) 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).
SomatropinDRUGOnce daily subcutaneous injection of Genotropin using pre-filled pen device.
PlaceboDRUG -
MOD-4023 (long lasting hGH)DRUGSingle subcutaneous dose of MOD-4023
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Eligibility Criteria

Age Range3 Years to 11 Years
SexALL
Healthy VolunteersNo
Study Sites46

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. ...

Countries:JapanUnited StatesArgentinaAustraliaBelarusBulgariaCanadaColombiaGeorgiaGreeceIndiaIsraelMexicoNew ZealandPolandRussiaSouth KoreaSpainTaiwanUkraineUnited KingdomHungarySlovakiaCzechiaSerbiaSlovenia
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Frequently asked questions about MOD-4023

What is MOD-4023 used for?

MOD-4023 is an investigational long-acting human growth hormone product being studied for the treatment of Growth Hormone Deficiency (GHD) in both children and adults. It is also being evaluated in healthy volunteers for pharmacokinetic and pharmacodynamic studies. The drug is currently in clinical development and is not yet approved by regulatory authorities.

Who is developing MOD-4023?

MOD-4023 is being developed by Opko Health Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol OPK. The company is conducting clinical trials to evaluate the safety and efficacy of this long-acting growth hormone product for the treatment of Growth Hormone Deficiency.

What phase is MOD-4023 in?

MOD-4023 has completed Phase 1, Phase 2, and Phase 3 clinical trials. The most advanced trial, NCT03874013, was a Phase 3 study in children with Growth Hormone Deficiency. However, the drug remains investigational and has not received FDA approval. All completed trials are finished, with no active trials currently listed.

What clinical trials has MOD-4023 been in?

MOD-4023 has been studied in four completed clinical trials. These include NCT01060722 and NCT02418767, both Phase 1 studies in healthy volunteers, NCT02500316, a Phase 2 long-term follow-up in children with GHD, and NCT03874013, a Phase 3 safety and efficacy study in children with Growth Hormone Deficiency.

Is MOD-4023 the same as long-acting human growth hormone?

MOD-4023 is a long-acting human growth hormone (hGH) product. It is designed to provide a longer duration of action compared to standard daily growth hormone injections. The drug is being investigated as a potential treatment for Growth Hormone Deficiency, with the goal of reducing the frequency of required dosing.