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CompletedNCT02968004Updated Jul 20, 2025Results posted

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

A Phase 3 interventional study of MOD-4023 and Somatropin in Pediatric Growth Hormone Deficiency, sponsored by OPKO Health, Inc.. Completed at 81 sites in 20 countries. Open to participants aged 3 Years to 11 Years. Per ClinicalTrials.gov, last updated 2025-07-20.

Sponsored by OPKO Health, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
224
Allocation
Randomized
Ages
3 Years to 11 Years
Sex
All
01

Study 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 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.

02

Conditions studied

  • Pediatric Growth Hormone Deficiency
03

In context

Lead sponsor

OPKO Health, Inc. is the lead sponsor of 48 studies on the registry; 2 are open to participants now.

Of its 8 completed or terminated interventional studies of FDA-regulated products, 6 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 11 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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
  6. Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
  7. Normal calculated GFR per updated bedside Schwartz formula for pediatric patients.
  8. 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.
  9. Normal 46XX karyotype for girls.

  10. Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient.

    LT-OLE Inclusion Criteria:

  11. Completion of the main study (12 months of treatment) with adequate compliance.
  12. Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient.
  13. 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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
224 participants (actual)

Study arms

  • Experimental
    MOD-4023

    Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

    Drug: MOD-4023

  • Active comparator
    Genotropin

    Once daily subcutaneous injection of somatropin (r-hGH; Genotropin)

    Drug: Somatropin

Interventions

  • DrugMOD-4023

    Once weekly subcutaneous injection using pre-filled pen device.

    Also known as: Somatrogon

  • DrugSomatropin

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

    Also known as: Genotropin

06

What researchers measure

Primary outcomes

  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.

    Time frame: 52 weeks

  2. Annual Height Velocity in OLE Phase

    Annualized Height Velocity (cm/year) in OLE Phase

    Time frame: Up to 60 months

Secondary outcomes

  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.

    Time frame: 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.

    Time frame: 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

    Time frame: 52 weeks

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

    Via central lab analysis

    Time frame: 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.

    Time frame: 60 months

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

    Time frame: 60 Months

Other outcomes

  1. Device

    Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA

    Time frame: 6 weeks

  2. Device

    Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA at Week 1 based on the Observer Assessment Tool (OAT)

    Time frame: 1 week

07

Results

Posted Jun 25, 2021

Participant flow

Main Study Phase
Participant flow — Main Study Phase
MilestoneMOD-4023Genotropin
Started109115
Completed108114
Not completed11
Withdrew: Adverse event10
Withdrew: Withdrawal by subject01
Open Label Extension (OLE) Year 1
Participant flow — Open Label Extension (OLE) Year 1
MilestoneMOD-4023Genotropin
Started104108
Completed10299
Not completed29
Withdrew: Adverse event06
Withdrew: Withdrawal by subject22
Withdrew: Miscellaneous01
Open Label Extension (OLE) Year 2
Participant flow — Open Label Extension (OLE) Year 2
MilestoneMOD-4023Genotropin
Started9186
Completed8882
Not completed34
Withdrew: Adverse event11
Withdrew: Withdrawal by subject22
Withdrew: Miscellaneous01
Open Label Extension (OLE) Year 3
Participant flow — Open Label Extension (OLE) Year 3
MilestoneMOD-4023Genotropin
Started8882
Completed8480
Not completed42
Withdrew: Adverse event10
Withdrew: Withdrawal by subject21
Withdrew: Prohibited treatments01
Withdrew: Local crisis10
Open Label Extension (OLE) Year 4
Participant flow — Open Label Extension (OLE) Year 4
MilestoneMOD-4023Genotropin
Started8379
Completed3843
Not completed4536
Withdrew: Withdrawal by subject34
Withdrew: Non-compliance with study drug01
Withdrew: Sponsor discontinuation of study2721
Withdrew: Bone age reached31
Withdrew: Local crisis109
Withdrew: Miscellaneous20
Open Label Extension (OLE) Year 5
Participant flow — Open Label Extension (OLE) Year 5
MilestoneMOD-4023Genotropin
Started2428
Completed1822
Not completed66
Withdrew: Withdrawal by subject10
Withdrew: Sponsor discontinuation of study26
Withdrew: Bone age reached,10
Withdrew: Closure of epiphyseal plates10
Withdrew: Local crisis10
Open Label Extension (OLE) Year 6
Participant flow — Open Label Extension (OLE) Year 6
MilestoneMOD-4023Genotropin
Started02
Completed00
Not completed02

Outcome measures

PrimaryAnnual 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.

Time frame:
52 weeks
Reported as:
Least squares mean · cm/year
Annual Height Velocity in Main Study Phase
cm/yearMOD-4023Genotropin
Annual Height Velocity in Main Study Phase10.10 (9.58 to 10.63)9.78 (9.29 to 10.26)
PrimaryAnnual Height Velocity in OLE Phase

Annualized Height Velocity (cm/year) in OLE Phase

Time frame:
Up to 60 months
Reported as:
Mean · cm/year
Annual Height Velocity in OLE Phase
cm/yearMOD-4023Genotropin
OLE Month 010.27 ± 2.389.72 ± 2.50
OLE Month 128.03 ± 1.818.21 ± 1.87
OLE Month 247.78 ± 1.918.05 ± 1.77
OLE Month 366.90 ± 1.807.14 ± 1.69
OLE Month 486.19 ± 1.626.86 ± 2.08
OLE Month 60—5.05 ± 1.10
SecondaryHeight 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.

Time frame:
After 6 months of treatment
Reported as:
Least squares mean · cm/year
Height Velocity at 6 Months in Main Study Phase
cm/yearMOD-4023Genotropin
Height Velocity at 6 Months in Main Study Phase10.59 (9.96 to 11.22)10.04 (9.47 to 10.62)
SecondaryChange 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.

Time frame:
After 6 and 12 months
Reported as:
Mean · Standard Deviation Score
Change in Height Standard Deviation Score (SDS) in Main Study Phase
Standard Deviation ScoreMOD-4023Genotropin
SDS at 6 months0.54 (0.48 to 0.61)0.48 (0.42 to 0.54)
SDS at 12 months0.92 (0.82 to 1.02)0.87 (0.78 to 0.96)
SecondaryChange in Bone Maturation (BM) in Main Study Phase

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

Time frame:
52 weeks
Reported as:
Mean · ratio of bone age to chronologic age
Change in Bone Maturation (BM) in Main Study Phase
ratio of bone age to chronologic ageMOD-4023Genotropin
Change in Bone Maturation (BM) in Main Study Phase0.05 ± 0.090.06 ± 0.1
SecondaryInsulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in Main Study Phase

Via central lab analysis

Time frame:
Baseline and at 12 months
Reported as:
Mean · Standard Deviation Score (SDS)
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in Main Study Phase
Standard Deviation Score (SDS)MOD-4023Genotropin
Baseline-1.95 ± 0.89-1.72 ± 0.90
At 12 months0.65 ± 1.32-0.69 ± 1.09
SecondaryChange 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.

Time frame:
60 months
Reported as:
Mean · Standard Deviation Score
Change in Height Standard Deviation Score (SDS) in OLE Phase
Standard Deviation ScoreMOD-4023Genotropin
Baseline (from Main Study Phase)-2.95 ± 1.30-2.79 ± 1.31
OLE Month 00.95 ± 0.550.85 ± 0.52
OLE Month 121.38 ± 0.781.34 ± 0.71
OLE Month 241.57 ± 0.771.66 ± 0.78
OLE Month 361.81 ± 0.851.93 ± 0.85
OLE Month 481.96 ± 0.672.04 ± 0.81
OLE Month 60—2.14 ± 0.38
SecondaryInsulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in OLE Phase
Time frame:
60 Months
Reported as:
Mean · Standard Deviation Score
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) in OLE Phase
Standard Deviation ScoreMOD-4023Genotropin
Baseline (from Main Study Phase)77.91 ± 46.1183.10 ± 41.17
OLE Month 0185.9 ± 107.169.96 ± 59.92
OLE Month 12255.9 ± 120.0248.6 ± 111.9
OLE Month 24293.4 ± 132.8295.3 ± 137.5
OLE Month 36315.6 ± 139.7330.7 ± 138.0
OLE Month 48354.8 ± 133.1368.9 ± 124.9
OLE Month 60—397.5 ± 85.27
Other pre-specifiedDevice

Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA

Time frame:
6 weeks

Results for this outcome have not been posted.

Other pre-specifiedDevice

Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA at Week 1 based on the Observer Assessment Tool (OAT)

Time frame:
1 week
Reported as:
Count of participants · Participants
Device
ParticipantsMOD-4023
Device17

Adverse events

Collected over 1 year for Main Study Phase. Up to 6 years for OLE Phase.. Non-serious events are listed at a 2% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
MOD-40230/109 (0%)3/109 (2.8%)92/109 (84.4%)
Genotropin0/115 (0%)2/115 (1.7%)90/115 (78.3%)
MOD-4023 in OLE Phase0/212 (0%)14/212 (6.6%)175/212 (82.5%)
Most frequent serious events
Showing 10 of 19
Most frequent serious events
EventMOD-4023GenotropinMOD-4023 in OLE Phase
AppendicitisInfections and infestations0/1090/1154/212
Chronic tonsillitisInfections and infestations1/1090/1150/212
GastroenteritisInfections and infestations1/1090/1150/212
PneumoniaInfections and infestations1/1090/1151/212
TonsillitisInfections and infestations0/1091/1151/212
UreterolithiasisRenal and urinary disorders0/1091/1150/212
LymphadenitisBlood and lymphatic system disorders0/1090/1151/212
Adrenal InsufficiencyEndocrine disorders0/1090/1151/212
Delayed PubertyEndocrine disorders0/1090/1151/212
Abdominal PainGastrointestinal disorders0/1090/1151/212
Most frequent other events
Showing 10 of 79
Most frequent other events
EventMOD-4023GenotropinMOD-4023 in OLE Phase
Injection Site PainGeneral disorders43/10929/11566/212
NasopharyngitisInfections and infestations25/10929/11566/212
HeadacheNervous system disorders18/10925/11541/212
PyrexiaGeneral disorders18/10916/11540/212
Corona Virus InfectionInfections and infestations0/1090/11537/212
CoughRespiratory, thoracic and mediastinal disorders9/1099/11524/212
VomitingGastrointestinal disorders8/1099/11523/212
InfluenzaInfections and infestations5/1093/11522/212
Oropharyngeal PainRespiratory, thoracic and mediastinal disorders6/1094/11518/212
Injection Site ErythemaGeneral disorders9/1090/11511/212

Baseline characteristics

Age, Continuous
Age, Continuous(years)MOD-4023GenotropinTotal
Mean7.83 ± 2.667.61 ± 2.377.72 ± 2.51
Sex: Female, Male
Sex: Female, Male(Participants)MOD-4023GenotropinTotal
Female273663
Male8279161
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)MOD-4023GenotropinTotal
Hispanic or Latino111324
Not Hispanic or Latino98102200
Unknown or Not Reported000
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)MOD-4023GenotropinTotal
White8186167
Black or African American022
Asian242145
American Indian or Alaska Native101
Native Hawaiian or Other Pacific Islander011
Other358
Unknown000
Multiracial000
Not reported000
Region of Enrollment
Region of Enrollment(participants)MOD-4023GenotropinTotal
Colombia448
Argentina123
United States172542
Ukraine131124
United Kingdom033
Belarus112
India121426
Russia81220
Spain10919
Greece303
New Zealand314
Canada325
South Korea8412
Turkey101
Taiwan022
Poland71219
Mexico202
Georgia516
Israel7815
Australia224
Bulgaria224
08

Study locations

81 sites
  • Children's Hospital of Orange County- Children's Clinic
    Orange, California 92868, United States
  • Children's Hospital- Colorado
    Aurora, Colorado 80045, United States
  • Rocky Mountain Pediatrics
    Centennial, Colorado 80112, United States
  • Nemours Children's Health System
    Jacksonville, Florida 32207, United States
  • University of Miami Medical Center
    Miami, Florida 33136, United States
  • St Luke's Children's Specialty Center
    Boise, Idaho 83712, United States
  • Children's Hospital of Iowa
    Iowa City, Iowa 52242, United States
  • Tufts Medical Center
    Boston, Massachusetts 02111, United States
  • University of Massachusettes
    Worcester, Massachusetts 01655, United States
  • University of Minnesota
    Minneapolis, Minnesota 55454, United States
  • The Diabetes & Obesity Clinical Specialist
    Las Vegas, Nevada 89148, United States
  • Goryeb Children's Hospital
    Morristown, New Jersey 07962, United States
  • Buffalo Children's Hospital
    Buffalo, New York 14222, United States
  • Albert Einstein College of Medicine at Yeshiva University
    Mineola, New York 11501, United States
  • Children's Hospital of Cincinnati / Cincinnati Center for Growth Disorders
    Cincinnati, Ohio 45229, United States
  • Children's Hospital of Pittsburgh
    Pittsburgh, Pennsylvania 15224, United States
  • Cook Children's Medical Center
    Fort Worth, Texas 76104, United States
  • Mary Bridge Children's Hospital & Health Center
    Tacoma, Washington 98405, United States
  • Hospital Materno Infantil San Roque
    Paraná, Entre Ríos Province 3100, Argentina
  • Hospital de Ninos de la Santisima Trinidad Cordoba
    Córdoba, 5000, Argentina
  • John Hunter Children's Hospital
    New Lambton Heights, New South Wales 2305, Australia
  • Children's Hospital at Westmead
    Westmead, New South Wales 2145, Australia
  • PM Hospital for Children
    Subiaco, Western Australia 6008, Australia
  • Health Institution- 2nd City Pediatric Hospital
    Minsk, 220020, Belarus
  • MHAT Sveta Marina
    Varna, 9010, Bulgaria
  • Centre de recherche du CHU Sainte-Justine
    Montreal, Quebec H3T 1C5, Canada
  • Dexa Diab
    Bogotá, 110221, Colombia
  • Uniendo
    Bogotá, 110221, Colombia
  • Hospital Pablo Tobon Uribe
    Medellín, 050034, Colombia
  • Maritime Hospital JSC
    Batumi, 6010, Georgia
  • Vere XXI JSC
    Tbilisi, 0108, Georgia
  • Aghia Sophia Children's Hospital
    Athens, 11527, Greece
  • General Children's Hospital of Athens P&A Kyriakou
    Athens, 11527, Greece
  • Manipal Hospital
    Bengaluru, Karnataka 560017, India
  • Getwell Health & Research Institute
    Nagpur, Maharashtra 440012, India
  • Jehangir Clinical Development Centre
    Pune, Maharashtra 411001, India
  • Postgraduate Institute of Medical Education and Research
    Chandigarh, Punjab 160012, India
  • Care Hospital
    Hyderabad, Telangna 500001, India
  • Meditrina Institute of Medical Sciences
    Nagpur, 440010, India
  • All India Institute of Medical Sciences
    New Delhi, 110029, India
  • Sir Gangaram Hospital
    New Delhi, 110060, India
  • Haemek Medical Center
    Afula, 1834111, Israel
  • Soroka Medical Center
    Beersheba, 84101, Israel
  • Assaf Harofeh Medical Center
    Be’er Ya‘aqov, 7030000, Israel
  • Bnei Zion Medical Center
    Haifa, 3339509, Israel
  • Shaare Zedek Medical Center
    Jerusalem, 9103102, Israel
  • Scheider Children's Medical Center
    Petah Tikva, 49202, Israel
  • Tel Hashomer Medical Center
    Ramat Gan, 5265601, Israel
  • Kaplan Medical Center
    Rehovot, 76100, Israel
  • Sourasky Medical Center
    Tel Aviv, 6423906, Israel
  • Hospital Angeles de Puebla
    Puebla City, Reserva Territorial Atilxcayotl 72190, Mexico
  • The Liggins Institute
    Grafton, Auckland 1023, New Zealand
  • Wellington Children's Hospital
    Newtown, Wellington Region 6021, New Zealand
  • Uniwersyteckie Centrum Kliniczne in Gdansk
    Gdansk, 80-211, Poland
  • Centrum Zdrowia Matki Polki
    Lodz, 93-338, Poland
  • Bashkirian State Medical University
    Ufa, Bashlortostan Republic 450008/450106, Russia
  • Kazan State Medical Univeristy/Pediatric Republic Clinical Hospital
    Kazan', Tatarstan Republic 420012/420138, Russia
  • Endocrinology Scientific Center
    Moscow, 117036, Russia
  • Pediatric City Clinical Hospital/Russian Medical Academy of Continuous Education
    Moscow, 125373, Russia
  • Saint Petersburg State Pediatric Medical University
    Saint Petersburg, 194100, Russia
  • Voronezh State Medical University
    Voronezh, 394024, Russia
  • Bundang Cha Hospital
    Seongnam-si, Gyeonggi-do 13497, South Korea
  • Inje University Busan Paik Hospital
    Busan, 47392, South Korea
  • KyungPook National University Hospital
    Daegu, 41944, South Korea
  • Chosun University Hospital
    Gwangju, 61453, South Korea
  • Severance Hospital
    Seoul, 03722, South Korea
  • Hospital Miguel Servet
    Zaragoza, Aragon 50009, Spain
  • Hospital Josep Trueta
    Girona, Catalonia 17007, Spain
  • Hospital de Navarra
    Pamplona, Navarre 31008, Spain
  • Hospital Universitario Central de Asturias
    Oviedo, 33011, Spain
  • Parc Tauli
    Sabadell, 15706, Spain
  • Hospital Universitario de Santiago de Compostela
    Santiago de Compostela, 08950, Spain
  • Taipei Veterans General Hospital
    Taipei, 11217, Taiwan
  • Tri-Service General Hospital
    Taipei, 114, Taiwan
  • Ukrainian Research and Practical Centre of Endocrine Surgery, Transplantation of Endocrine Organs and Tissues of the Ministry of Health of Ukraine
    Kyiv, 01021, Ukraine
  • Institute of Endocrinology and Metabolism/VP Komisarenko of Academy of Medical Science of Ukraine
    Kyiv, 04114, Ukraine
  • Odesa Regional Clinical Children's Hospital
    Odesa, 65031, Ukraine
  • Vinnitsa Regional Clinical Highly Specialized Endocrinology Centre
    Vinnitsa, 21010, Ukraine
  • Zaporizhzhya Regional Clinical Child Hospital
    Zaporizhzhya, 69063, Ukraine
  • Royal Hospital for Children
    Glasgow, G514TF, United Kingdom
  • St. George's University Hospital
    London, SW17 OQT, United Kingdom
09

References and documents

Publications

  • Stawerska R, Choe J, Wang R, Wajnrajch MP, Cara JF, Thomas M, La Torre D, Taylor CT, Phillip M, Steelman J, Deal CL, Silverman LA. Extension of the Phase 3 Trial of Somatrogon in Children with Growth Hormone Deficiency: 3 Years of Safety and Efficacy. Horm Res Paediatr. 2026 Jun 1:1-19. doi: 10.1159/000552183. Online ahead of print. PubMed 42224388 ↗
  • 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 ↗

Study documents

  • Study protocol · Apr 27, 2018
  • Statistical analysis plan · Jan 3, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 20, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02968004
Lead sponsor
OPKO Health, Inc.
Responsible party
Sponsor
First posted
Nov 18, 2016
Start date
Dec 2016
Primary completion
Aug 2019
Completion
May 2024
Results posted
Jun 25, 2021
Last update
Jul 20, 2025

Study contacts

Tony Cruz
study director · Sponsor GmbH
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Jun 2025. You cannot join it, but the record below documents what was studied.

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Discussion

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Other studies from this sponsor

OPKO Health, Inc.

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