Growth Hormone Deficiency (GHD)Phase 2NCT02500316
Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
A Phase 2 study testing MOD-4023 in people with Growth Hormone Deficiency (GHD), run by OPKO Health, Inc. at 13 sites in 6 countries.
Trial summary
Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Growth Hormone Deficiency (GHD)
- What is being tested?
- MOD-4023
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 13 sitesin 6 countries
- Study dates
- Feb 2014to Nov 2023
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study.
Who cannot join
- 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 Standard Deviation for age and sex;
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- 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, short stature homeobox-containing gene (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))
- 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.
- Major medical conditions and/or presence of contraindication to r-hGH treatment.
- Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
- Drug, substance, or alcohol abuse.
- Known hypersensitivity to the components of study medication.
- Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
- The patient and/or the parent/legal guardian are likely to be non-compliant in respect to study conduct.
- Participation in any other trial of an investigational agent within 30 days prior to Screening.
Trial locations and contacts
13 sites in 6 countries.
13 sites in 6 countries
Russia5 sites
Endocrinology Scientific Centre, Institute of Child Endocrinology
Moscow
Russian Medical Academy of Postgraduate Education
Moscow
SPGPMA
Saint Petersburg
SamGMU
Samara
SBEIHPE
Ufa
Ukraine4 sites
Donetsk Regional Children Clinical Hospital
Donetsk
Institute of Endocrinology
Kiev
Ukrainian Scientific Center of Endocrine Surgery Moh of Ukraine
Kiev
Odessa Regional Children'S Clinical Hospital
Odesa
United States1 site
Nemours Children's Clinic
Jacksonville, Florida, 32207
About this study
Official title: Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.
- Conditions
- Growth Hormone Deficiency (GHD)
- Being tested
- MOD-4023
- Keywords
- Growth hormone deficiency (GHD), r-hGH, Long-acting
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MOD-4023Drug
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
| Study group | Role | What they receive |
|---|---|---|
| MOD-4023 | Experimental | Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN). Receives: Drug: MOD-4023 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Height Velocity
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).
Measured: 8 years
Delta Height SDS Every 12 Months
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.
Measured: 8 years
Secondary outcomes (1)
Summary of IGF-1 SDS
A summary of IGF-I SDS at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5). IGF-1 SDS (Standard Deviation Score) reflects how an individual's IGF-1 level compares to the average for their age and sex, based on reference values from Bidlingmaier et al. (2014, The Journal of Clinical Endocrinology \& Metabolism, 99(5):1712-1721). A z-score of 0 indicates a level equal to the population mean, while positive scores indicate above-average levels and negative scores reflect below-average levels. An IGF-1 SDS below -2 suggests deficiency, whereas a score above +2 is considered elevated. In the study, when IGF-1 SDS exceeded +2, a confirmatory test was performed, and if elevation was confirmed, the dose was reduced by 15%.
Measured: 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2015 (estimated)
- Study startFeb 2014
- Primary completionNov 2023
- Study completionNov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Growth Hormone Deficiency (GHD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- OPKO Health, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Zvi Zadik, MD, Principal investigator, Kaplan Medical Center, Israel
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02500316
- Sponsor's protocol ID
- CP-4-004-OLE
- Other IDs
- 2011-004553-60
- Status last verified
- Apr 2025
- First submitted
- 12 Jul 2015
- Last updated
- 9 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily R-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
- ClinicalTrials.gov ID
- NCT02500316
- Acronym / sponsor ID
- CP-4-004-OLE
- Conditions
- Growth Hormone Deficiency (GHD)
- Keywords
- Growth hormone deficiency (GHD), r-hGH, Long-acting
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants (actual)
- Lead sponsor
- OPKO Health, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2014
- Primary completion
- Nov 2023 (actual)
- Study completion
- Nov 2023 (actual)
- First posted
- 16 Jul 2015 (estimated)
- Results
- Posted 9 Apr 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MOD-4023 | Experimental | Drug: MOD-4023Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN). |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study.
Key exclusion criteria (23 listed)
- 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:
- Children with psychosocial dwarfism.
- Children born small for gestational age (SGA - birth weight and/or birth length < -2 SD for gestational age).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MOD-4023Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN). | Drug | MOD-4023 |
Primary outcome
Annualized Height Velocity
Time frame: 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
Time frame: 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.
1 secondary outcome
Summary of IGF-1 SDS
Time frame: 8 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Zvi Zadik, MD principal investigator
Kaplan Medical Center, Israel
13 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MOD-4023 in people with Growth Hormone Deficiency (GHD), run by OPKO Health, Inc. at 13 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Russia, Ukraine, United States, Belarus, Greece and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Height Velocity (time frame: 8 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2025.