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MOD-4023

Phase 3

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

MOD-4023 development status

Highest phase Phase 3
Registered trials 10 across 2 sponsors since Oct 2010

MOD-4023 target and mechanism

Molecular targetGH receptor
Target classReceptor
ModalitySmall molecule

Also known as MOD-4023 (long lasting hGH), Somatrogon

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials7
Total Enrollment515

FDA Designations

No designations recorded

MOD-4023 clinical trials

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.

Area under the curve (AUC) of the serum concentration-time from time 0 to a specific time (AUCt)
30 days
Area under the curve (AUC) of the serum concentration-time from time 0 to infinity (AUC∞)
30 days
Serum concentration from of maximum concentration (Cmax) of somatrogon levels in serum
30 days
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)

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
Somatrogon pre-filled PENEXPERIMENTAL -
Somatrogon frozen liquid formulationACTIVE_COMPARATOR -
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

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.
SomatrogonDRUGA single dose of 12 mg of somatrogon will be injected as one sc injection to the arm on each dosing day (Day 0 and Day 14).
PlaceboDRUG -
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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?

MOD-4023 is an investigational long-acting human growth hormone (hGH) therapy being developed by OPKO Health Inc. (NASDAQ: OPK) for growth hormone deficiency. It is designed to reduce the frequency of injections compared with daily growth hormone therapy. It is currently in Phase 2 development.

What is MOD-4023 used for?

MOD-4023 is being developed for the treatment of growth hormone deficiency in adults and children. It is intended to replace endogenous growth hormone in patients who do not produce enough. The drug is investigational and has not been approved for any indication.

What does MOD-4023 target?

MOD-4023 targets the growth hormone receptor (GHR). By binding to this receptor, it mimics the action of endogenous growth hormone, stimulating growth and metabolic effects. The target is a cell surface receptor, and the drug is a long-acting form of human growth hormone.

Who is developing MOD-4023?

MOD-4023 is being developed by OPKO Health Inc., a biopharmaceutical company traded on the NASDAQ under the ticker OPK. OPKO has conducted clinical trials of MOD-4023 in both adult and pediatric growth hormone deficiency.

What phase is MOD-4023 in?

MOD-4023 is in Phase 2 development. It has completed Phase 1, Phase 2, and Phase 3 clinical trials, but the overall development program is currently at Phase 2. It is investigational and has not been approved by the FDA or any other regulatory agency.

What clinical trials is MOD-4023 in?

MOD-4023 has been studied in several completed clinical trials, including NCT03874013 (Phase 3 in Japan), NCT02968004 (Phase 3 global pediatric study), NCT02500316 (Phase 2 long-term follow-up), and NCT02418767 (Phase 1 pharmacokinetics in healthy volunteers). All trials are listed as completed.

Is MOD-4023 the same as long-acting hGH?

MOD-4023 is also known as long-acting hGH (long-lasting human growth hormone). This alternative name reflects its extended duration of action compared with daily growth hormone injections. It is the same investigational product developed by OPKO Health.