Pediatric Growth Hormone DeficiencyPhase 2NCT01592500
Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
A Phase 2 study testing MOD-4023 and Somatropin in people with Pediatric Growth Hormone Deficiency, run by OPKO Health, Inc. at 4 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Pediatric Growth Hormone Deficiency
- What is being tested?
- MOD-4023 and Somatropin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 4 sitesin 3 countries
- Study dates
- Feb 2012to Jul 2015
- 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
- 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.
- 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 .
- Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
- Without prior exposure to any r-hGH therapy.
- Normal birth size, birth weight and length for gestational age
- Impaired height and height velocity defined as:
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- 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).
- 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.
- BMI must be within ±2 SD of mean BMI for the chronological age and sex according to the 2000 CDC standards.
- 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;
- 12. Written informed consent of the parent or legal guardian of the patient and assent of the patient (if the patient can read).
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 SD 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, 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
4 sites in 3 countries.
4 sites in 3 countries
Hungary2 sites
Buda Children's Hospital
Budapest
Heim Pal Children's Hospital
Budapest
About this study
Official title: Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily r-hGH Therapy in Pre-pubertal Growth Hormone Deficient Children
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.
- Conditions
- Pediatric Growth Hormone Deficiency
- Being tested
- MOD-4023, Somatropin
- Keywords
- GHD, r-hGH, Long-acting
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MOD-4023Drug
Once weekly subcutaneous injection
SomatropinDrug
Also called Genotropin
Once daily subcutaneous injection of Genotropin
| Study group | Role | What they receive |
|---|---|---|
| MOD-4023 low dose | Experimental | Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) Receives: Drug: MOD-4023 |
| MOD-4023 middle dose | Experimental | Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) Receives: Drug: MOD-4023 |
| MOD-4023 high dose | Experimental | Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) Receives: Drug: MOD-4023 |
| Genotropin | Active comparator | Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin) Receives: Drug: Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual Height Velocity
Annual Height Velocity in cm/year measured after 12 months of treatment
Measured: 12 months of treatment
Secondary outcomes (3)
Height Velocity at 6 Months
Annualized Height Velocity in cm/year measured after 6 months of treatment
Measured: After 6 months of treatment
Change in Height Standard Deviation Score (SDS)
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
Measured: After 6 and 12 months of treatment
Change in IGF-1 Standard Deviation Score
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.
Measured: Once monthly on day 4 after the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2012 (estimated)
- Study startFeb 2012
- Primary completionJul 2015
- Study completionJul 2015
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.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Pediatric Growth Hormone DeficiencyStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 56 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
- NCT01592500
- Sponsor's protocol ID
- CP-4-004
- Other IDs
- 2011-004553-60
- Status last verified
- Jan 2020
- First submitted
- 2 May 2012
- Last updated
- 23 Jan 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Mauras 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.
PubMed 42372104Zelinska 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.
PubMed 28323965
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2020. 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-hGH Therapy in Pre-pubertal Growth Hormone Deficient Children
- ClinicalTrials.gov ID
- NCT01592500
- Acronym / sponsor ID
- CP-4-004
- Conditions
- Pediatric Growth Hormone Deficiency
- Keywords
- GHD, r-hGH, Long-acting
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- OPKO Health, Inc.Industry (pharma, biotech, devices)
- Study start
- Feb 2012
- Primary completion
- Jul 2015 (actual)
- Study completion
- Jul 2015 (actual)
- First posted
- 7 May 2012 (estimated)
- Results
- Posted 23 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| MOD-4023 low dose | Experimental | Drug: MOD-4023Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) |
| MOD-4023 middle dose | Experimental | Drug: MOD-4023Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) |
| MOD-4023 high dose | Experimental | Drug: MOD-4023Once weekly subcutaneous injection of long acting r-hGH (MOD-4023) |
| Genotropin | Active comparator | Drug: SomatropinOnce daily subcutaneous injection of Somatropin (r-hGH; Genotropin) |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (11 listed)
- 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.
- 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 .
- Bone age (BA) is not older than chronological age and should be no greater than 9 years for girls and 10 years for boys.
- Without prior exposure to any r-hGH therapy.
- Normal birth size, birth weight and length for gestational age
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 subcutaneous injection | Drug | MOD-4023 high dose, MOD-4023 low dose, MOD-4023 middle dose |
| SomatropinAlso: GenotropinOnce daily subcutaneous injection of Genotropin | Drug | Genotropin |
Primary outcome
Annual Height Velocity
Time frame: 12 months of treatment
Annual Height Velocity in cm/year measured after 12 months of treatment
3 secondary outcomes
Height Velocity at 6 Months
Time frame: After 6 months of treatment
Change in Height Standard Deviation Score (SDS)
Time frame: After 6 and 12 months of treatment
Change in IGF-1 Standard Deviation Score
Time frame: Once monthly on day 4 after the last dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42372104Mauras 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.
- Derived from this study: PubMed 28323965Zelinska 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.
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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
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MOD-4023 and Somatropin in people with Pediatric Growth Hormone Deficiency, run by OPKO Health, Inc. at 4 sites in 3 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 11 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 23 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Hungary, Greece, Slovakia. 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 23 Jan 2020). 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?
Annual Height Velocity (time frame: 12 months of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01592500, which also lists 2 linked publications.
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 23 Jan 2020.