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Pediatric Growth Hormone DeficiencyPhase 3NCT02968004

Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

A Phase 3 study testing MOD-4023 and Somatropin in people with Pediatric Growth Hormone Deficiency, run by OPKO Health, Inc. at 81 sites in 20 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This is an open-label, randomized, multicenter, efficacy and safety study of weekly MOD-4023 compared to daily Genotropin therapy in pre-pubertal children with growth hormone deficiency. 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
224 participants
Where
81 sitesin 20 countries
Study dates
Dec 2016to May 2024
Phase
Phase 3
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

Main Study Inclusion Criteria:

  1. Pre-pubertal children aged ≥3 years, and not yet 11 years for girls or not yet 12 years for boys with either isolated GHD, or GH insufficiency as part of multiple pituitary hormone deficiency.
  2. Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤10 ng/mL.
  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 (naïve patients).
  5. Impaired height and height velocity defined as:
  • Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS) and gender according to sponsor calculator
  • The interval between 2 height measurements should be at least 6 months, but should not exceed 18 months prior to inclusion
  1. Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
  2. Normal calculated GFR per updated bedside Schwartz formula for pediatric patients.
  3. 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 ICF signing.
  4. Normal 46XX karyotype for girls.
  5. Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient. LT-OLE Inclusion Criteria:
  6. Completion of the main study (12 months of treatment) with adequate compliance.
  7. Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient.
  8. Agree to refrain from sexual activity.

Main Study 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 psychosocial dwarfism.
  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. T2 and T1 diabetic patients, who in the opinion of the investigator are not receiving standard of care treatment or are non-compliant with their prescribed treatment or who are in poor metabolic control.
  9. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
  10. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of ADHD drugs or hormone replacement therapies (thyroxin, hydrocortisone, desmopressin).
  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.
  12. Major medical conditions and/or presence of contraindication to r-hGH treatment.
  13. More than one closed epiphyses.
  14. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
  15. Drug, substance, or alcohol abuse.
  16. Known hypersensitivity to the components of study medication.
  17. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets.
  18. Likely non-compliance in respect to study conduct.
  19. Participation in any other trial of an investigational agent within 30 days prior to consent.
  20. Study enrollment has been met or study is closed by sponsor prior to completion of screening process. LT-OLE Exclusion Criteria:
  21. Concomitant administration of other treatments that may have an effect on growth such as anabolic steroids, or sex steroids, with the exception of ADHD drugs or HRT (thyroxin, hydrocortisone, desmopressin).
  22. Change in medical condition during the treatment period (such as, but not limited to, development of a serious inter-current critical illness, a severe adverse drug reaction, etc.).
  23. Positive pregnancy test.
  24. Unresolved drug related (Genotropin or MOD-4023) SAE from the treatment period as per medical monitor judgement.

Trial locations and contacts

81 sites in 20 countries.

81 sites in 20 countries

United States18 sites

  • Children's Hospital- Colorado

    Aurora, Colorado, 80045

  • St Luke's Children's Specialty Center

    Boise, Idaho, 83712

  • Tufts Medical Center

    Boston, Massachusetts, 02111

  • Buffalo Children's Hospital

    Buffalo, New York, 14222

  • Rocky Mountain Pediatrics

    Centennial, Colorado, 80112

  • Children's Hospital of Cincinnati / Cincinnati Center for Growth Disorders

    Cincinnati, Ohio, 45229

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104

  • Children's Hospital of Iowa

    Iowa City, Iowa, 52242

  • Nemours Children's Health System

    Jacksonville, Florida, 32207

  • The Diabetes & Obesity Clinical Specialist

    Las Vegas, Nevada, 89148

  • University of Miami Medical Center

    Miami, Florida, 33136

  • Albert Einstein College of Medicine at Yeshiva University

    Mineola, New York, 11501

  • University of Minnesota

    Minneapolis, Minnesota, 55454

  • Goryeb Children's Hospital

    Morristown, New Jersey, 07962

  • Children's Hospital of Orange County- Children's Clinic

    Orange, California, 92868

  • Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224

  • Mary Bridge Children's Hospital & Health Center

    Tacoma, Washington, 98405

  • University of Massachusettes

    Worcester, Massachusetts, 01655

Other trials in United States

Israel9 sites

  • Haemek Medical Center

    Afula, 1834111

  • Assaf Harofeh Medical Center

    Be’er Ya‘aqov, 7030000

  • Soroka Medical Center

    Beersheba, 84101

  • Bnei Zion Medical Center

    Haifa, 3339509

  • Shaare Zedek Medical Center

    Jerusalem, 9103102

  • Scheider Children's Medical Center

    Petah Tikva, 49202

  • Tel Hashomer Medical Center

    Ramat Gan, 5265601

  • Kaplan Medical Center

    Rehovot, 76100

  • Sourasky Medical Center

    Tel Aviv, 6423906

Other trials in Israel

India8 sites

  • Manipal Hospital

    Bengaluru, Karnataka, 560017

  • Postgraduate Institute of Medical Education and Research

    Chandigarh, Punjab, 160012

  • Care Hospital

    Hyderabad, Telangna, 500001

  • Getwell Health & Research Institute

    Nagpur, Maharashtra, 440012

  • Meditrina Institute of Medical Sciences

    Nagpur, 440010

  • All India Institute of Medical Sciences

    New Delhi, 110029

  • Sir Gangaram Hospital

    New Delhi, 110060

  • Jehangir Clinical Development Centre

    Pune, Maharashtra, 411001

Other trials in India

Russia6 sites

  • Kazan State Medical Univeristy/Pediatric Republic Clinical Hospital

    Kazan', Tatarstan Republic, 420012/420138

  • Endocrinology Scientific Center

    Moscow, 117036

  • Pediatric City Clinical Hospital/Russian Medical Academy of Continuous Education

    Moscow, 125373

  • Saint Petersburg State Pediatric Medical University

    Saint Petersburg, 194100

  • Bashkirian State Medical University

    Ufa, Bashlortostan Republic, 450008/450106

  • Voronezh State Medical University

    Voronezh, 394024

Other trials in Russia

Spain6 sites

  • Hospital Josep Trueta

    Girona, Catalonia, 17007

  • Hospital Universitario Central de Asturias

    Oviedo, 33011

  • Hospital de Navarra

    Pamplona, Navarre, 31008

  • Parc Tauli

    Sabadell, 15706

  • Hospital Universitario de Santiago de Compostela

    Santiago de Compostela, 08950

  • Hospital Miguel Servet

    Zaragoza, Aragon, 50009

Other trials in Spain

South Korea5 sites

  • Inje University Busan Paik Hospital

    Busan, 47392

  • KyungPook National University Hospital

    Daegu, 41944

  • Chosun University Hospital

    Gwangju, 61453

  • Bundang Cha Hospital

    Seongnam-si, Gyeonggi-do, 13497

  • Severance Hospital

    Seoul, 03722

Other trials in South Korea

Ukraine5 sites

  • Ukrainian Research and Practical Centre of Endocrine Surgery, Transplantation of Endocrine Organs and Tissues of the Ministry of Health of Ukraine

    Kyiv, 01021

  • Institute of Endocrinology and Metabolism/VP Komisarenko of Academy of Medical Science of Ukraine

    Kyiv, 04114

  • Odesa Regional Clinical Children's Hospital

    Odesa, 65031

  • Vinnitsa Regional Clinical Highly Specialized Endocrinology Centre

    Vinnitsa, 21010

  • Zaporizhzhya Regional Clinical Child Hospital

    Zaporizhzhya, 69063

Other trials in Ukraine

Australia3 sites

  • John Hunter Children's Hospital

    New Lambton Heights, New South Wales, 2305

  • PM Hospital for Children

    Subiaco, Western Australia, 6008

  • Children's Hospital at Westmead

    Westmead, New South Wales, 2145

Other trials in Australia

Colombia3 sites

  • Dexa Diab

    Bogotá, 110221

  • Uniendo

    Bogotá, 110221

  • Hospital Pablo Tobon Uribe

    Medellín, 050034

Other trials in Colombia

Argentina2 sites

  • Hospital de Ninos de la Santisima Trinidad Cordoba

    Córdoba, 5000

  • Hospital Materno Infantil San Roque

    Paraná, Entre Ríos Province, 3100

Other trials in Argentina

Georgia2 sites

  • Maritime Hospital JSC

    Batumi, 6010

  • Vere XXI JSC

    Tbilisi, 0108

Other trials in Georgia

Greece2 sites

  • Aghia Sophia Children's Hospital

    Athens, 11527

  • General Children's Hospital of Athens P&A Kyriakou

    Athens, 11527

Other trials in Greece

New Zealand2 sites

  • The Liggins Institute

    Grafton, Auckland, 1023

  • Wellington Children's Hospital

    Newtown, Wellington Region, 6021

Other trials in New Zealand

Poland2 sites

  • Uniwersyteckie Centrum Kliniczne in Gdansk

    Gdansk, 80-211

  • Centrum Zdrowia Matki Polki

    Lodz, 93-338

Other trials in Poland

Taiwan2 sites

  • Taipei Veterans General Hospital

    Taipei, 11217

  • Tri-Service General Hospital

    Taipei, 114

Other trials in Taiwan

United Kingdom2 sites

  • Royal Hospital for Children

    Glasgow, G514TF

  • St. George's University Hospital

    London, SW17 OQT

Other trials in United Kingdom

Belarus1 site

  • Health Institution- 2nd City Pediatric Hospital

    Minsk, 220020

Other trials in Belarus

Bulgaria1 site

  • MHAT Sveta Marina

    Varna, 9010

Other trials in Bulgaria

Canada1 site

  • Centre de recherche du CHU Sainte-Justine

    Montreal, Quebec, H3T 1C5

Other trials in Canada

Mexico1 site

  • Hospital Angeles de Puebla

    Puebla City, Reserva Territorial Atilxcayotl, 72190

Other trials in Mexico

About this study

Official title: A Phase 3, Open-label, Randomized, Multicenter, 12 Months, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin - Therapy in Pre-pubertal Children With Growth Hormone Deficiency

This is an open-label, randomized, multicenter, efficacy and safety study of weekly MOD-4023 compared to daily Genotropin therapy in pre-pubertal children with growth hormone deficiency.

Read the detailed description

The study consists of a 12 month, open-label, randomized, active controlled, parallel group study comparing the efficacy and safety of weekly MOD-4023 to daily growth hormone (GH), Genotropin. After 12 months, subjects will have the option to enter the long term open-label extension.

Being tested
MOD-4023, Somatropin

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MOD-4023Drug

    Also called Somatrogon

    Once weekly subcutaneous injection using pre-filled pen device.

  • SomatropinDrug

    Also called Genotropin

    Once daily subcutaneous injection of Genotropin using pre-filled pen device.

Study groups in NCT02968004
Study groupRoleWhat they receive
MOD-4023ExperimentalOnce weekly subcutaneous injection of long acting r-hGH (MOD-4023)
Receives: Drug: MOD-4023
GenotropinActive comparatorOnce 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.

  1. Annual Height Velocity in Main Study Phase

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

    Measured: 52 weeks

  2. Annual Height Velocity in OLE Phase

    Annualized Height Velocity (cm/year) in OLE Phase

    Measured: Up to 60 months

Secondary outcomes (6)

  1. Height Velocity at 6 Months in Main Study Phase

    Height velocity in cm measured after 6 months of treatment. Annualized Height velocity after 6 months is calculated based on the difference between the heights at 6 months and baseline.

    Measured: After 6 months of treatment

  2. Change in Height Standard Deviation Score (SDS) in Main Study Phase

    Change in height Standard Deviation Score (SDS) after 6 and 12 months is calculated based on the difference between the heights at 6 and 12 months and baseline.

    Measured: After 6 and 12 months

  3. Change in Bone Maturation (BM) in Main Study Phase

    Annual change in bone age measurements as per Gruelich-Pyle method

    Measured: 52 weeks

  4. Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in Main Study Phase

    Via central lab analysis

    Measured: Baseline and at 12 months

  5. Change in Height Standard Deviation Score (SDS) in OLE Phase

    Change in height Standard Deviation Score (SDS) at each time point is calculated based on the difference between the time point and baseline.

    Measured: 60 months

  6. Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in OLE Phase

    Measured: 60 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Nov 2016 (estimated)
  2. Study startDec 2016
  3. Primary completionAug 2019
  4. Study completionMay 2024

Resources

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
224 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02968004
Sponsor's protocol ID
CP-4-006
Status last verified
Jun 2025
First submitted
13 Nov 2016
Last updated
20 Jul 2025
Individual data sharing
No

Publications

  • Gomez R, Khadilkar V, Shembalkar J, Chu DM, Ko CW, Wajnrajch MP, Wang R. Post hoc subgroup analysis of Asian children with paediatric GHD from the global phase 3 efficacy and safety study of once-weekly somatrogon vs. once-daily somatropin. J Pediatr Endocrinol Metab. 2024 May 9;37(6):525-531. doi: 10.1515/jpem-2023-0512. Print 2024 Jun 25.

    PubMed 38717038

  • Loftus J, Quitmann J, Valluri SR. Health-related quality of life in pre-pubertal children with pediatric growth hormone deficiency: 12-month results from a phase 3 clinical trial of once-weekly somatrogon versus once-daily somatropin. Curr Med Res Opin. 2024 Feb;40(2):175-184. doi: 10.1080/03007995.2023.2290623. Epub 2024 Jan 24.

    PubMed 38053515

  • Gomez R, Lamoureux R, Turner-Bowker DM, Loftus J, Maghnie M, Miller BS, Polak M, Yaworsky A. Physician experience with once-weekly somatrogon versus once-daily rhGH regimen in pediatric patients with growth hormone deficiency: a cross-sectional survey of physicians from the global phase 3 study. Front Endocrinol (Lausanne). 2023 Oct 17;14:1254424. doi: 10.3389/fendo.2023.1254424. eCollection 2023.

    PubMed 37955005

  • Deal CL, Steelman J, Vlachopapadopoulou E, Stawerska R, Silverman LA, Phillip M, Kim HS, Ko C, Malievskiy O, Cara JF, Roland CL, Taylor CT, Valluri SR, Wajnrajch MP, Pastrak A, Miller BS. Efficacy and Safety of Weekly Somatrogon vs Daily Somatropin in Children With Growth Hormone Deficiency: A Phase 3 Study. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2717-e2728. doi: 10.1210/clinem/dgac220.

    PubMed 35405011

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing MOD-4023 and Somatropin in people with Pediatric Growth Hormone Deficiency, run by OPKO Health, Inc. at 81 sites in 20 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 20 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 81 sites in United States, Israel, India, Russia, Spain and 15 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 25 Jun 2021). 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 in Main Study Phase (time frame: 52 weeks). 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?

On the ClinicalTrials.gov record for NCT02968004, which also lists 4 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 20 Jul 2025.

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