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

NCT02968004 | Completed Phase 3 Results

• 1 other identifier: CP-4-006
• Study Type: interventional
• Target: 224
• Locations: 20 countries
• Sites: 81

Brief 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.

Conditions

Pediatric Growth Hormone Deficiency

Outcome Measures

Primary Outcomes (2)

• 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.

  52 weeks

• Annual Height Velocity in OLE Phase

  Annualized Height Velocity (cm/year) in OLE Phase

  Up to 60 months

Secondary Outcomes (6)

• Height Velocity at 6 Months in Main Study Phase

  After 6 months of treatment

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

  After 6 and 12 months

• Change in Bone Maturation (BM) in Main Study Phase

  52 weeks

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

  Baseline and at 12 months

• Change in Height Standard Deviation Score (SDS) in OLE Phase

  60 months

Other Outcomes (2)

• Device

  6 weeks

• Device

  1 week

Study Arms (2)

MOD-4023

EXPERIMENTAL

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

Drug: MOD-4023

Genotropin

ACTIVE COMPARATOR

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

Drug: Somatropin

Interventions

MOD-4023 DRUG

Once weekly subcutaneous injection using pre-filled pen device.

Also known as: Somatrogon

MOD-4023

Somatropin DRUG

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

Also known as: Genotropin

Genotropin

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.
• Confirmed diagnosis of GHD by two different GH provocation tests defined as a peak plasma GH level of ≤10 ng/mL.
• Bone age (BA) is not older than chronological age and should be less than 10 for girls and less than 11 for boys.
• Without prior exposure to any r-hGH therapy (naïve patients).
• 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
• Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
• Normal calculated GFR per updated bedside Schwartz formula for pediatric patients.
• 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.
• Normal 46XX karyotype for girls.
• Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient.
• Completion of the main study (12 months of treatment) with adequate compliance.
• Willing and able to provide written informed consent of the parent or legal guardian and written assent from patient.
• Agree to refrain from sexual activity.

You may not qualify if:

• Children with prior history of leukemia, lymphoma, sarcoma or any other forms of cancer.
• History of radiation therapy or chemotherapy.
• Malnourished children defined as BMI \< -2 SDS for age and sex.
• Children with psychosocial dwarfism.
• Children born small for gestational age (SGA - birth weight and/or birth length \<-2 SDS 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.
• 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.
• Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
• 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).
• Children requiring glucocorticoid therapy (e.g. for asthma) that are taking chronically a dose greater than 400 μg/d of inhaled budesonide or equivalent.
• Major medical conditions and/or presence of contraindication to r-hGH treatment.
• More than one closed epiphyses.
• Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
• Drug, substance, or alcohol abuse.

Sponsors & Collaborators

• OPKO Health, Inc.: lead

Study Sites (81)

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

Orange, California, 92868, United States

Location

Children's Hospital- Colorado

Aurora, Colorado, 80045, United States

Location

Rocky Mountain Pediatrics

Centennial, Colorado, 80112, United States

Location

Nemours Children's Health System

Jacksonville, Florida, 32207, United States

Location

University of Miami Medical Center

Miami, Florida, 33136, United States

Location

St Luke's Children's Specialty Center

Boise, Idaho, 83712, United States

Location

Children's Hospital of Iowa

Iowa City, Iowa, 52242, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

University of Massachusettes

Worcester, Massachusetts, 01655, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55454, United States

Location

The Diabetes & Obesity Clinical Specialist

Las Vegas, Nevada, 89148, United States

Location

Goryeb Children's Hospital

Morristown, New Jersey, 07962, United States

Location

Buffalo Children's Hospital

Buffalo, New York, 14222, United States

Location

Albert Einstein College of Medicine at Yeshiva University

Mineola, New York, 11501, United States

Location

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

Cincinnati, Ohio, 45229, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Mary Bridge Children's Hospital & Health Center

Tacoma, Washington, 98405, United States

Location

Hospital Materno Infantil San Roque

Paraná, Entre Ríos Province, 3100, Argentina

Location

Hospital de Ninos de la Santisima Trinidad Cordoba

Córdoba, 5000, Argentina

Location

John Hunter Children's Hospital

New Lambton Heights, New South Wales, 2305, Australia

Location

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

Location

PM Hospital for Children

Subiaco, Western Australia, 6008, Australia

Location

Health Institution- 2nd City Pediatric Hospital

Minsk, 220020, Belarus

Location

MHAT Sveta Marina

Varna, 9010, Bulgaria

Location

Centre de recherche du CHU Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Dexa Diab

Bogotá, 110221, Colombia

Location

Uniendo

Bogotá, 110221, Colombia

Location

Hospital Pablo Tobon Uribe

Medellín, 050034, Colombia

Location

Maritime Hospital JSC

Batumi, 6010, Georgia

Location

Vere XXI JSC

Tbilisi, 0108, Georgia

Location

Aghia Sophia Children's Hospital

Athens, 11527, Greece

Location

General Children's Hospital of Athens P&A Kyriakou

Athens, 11527, Greece

Location

Manipal Hospital

Bengaluru, Karnataka, 560017, India

Location

Getwell Health & Research Institute

Nagpur, Maharashtra, 440012, India

Location

Jehangir Clinical Development Centre

Pune, Maharashtra, 411001, India

Location

Postgraduate Institute of Medical Education and Research

Chandigarh, Punjab, 160012, India

Location

Care Hospital

Hyderabad, Telangna, 500001, India

Location

Meditrina Institute of Medical Sciences

Nagpur, 440010, India

Location

All India Institute of Medical Sciences

New Delhi, 110029, India

Location

Sir Gangaram Hospital

New Delhi, 110060, India

Location

Haemek Medical Center

Afula, 1834111, Israel

Location

Soroka Medical Center

Beersheba, 84101, Israel

Location

Assaf Harofeh Medical Center

Be’er Ya‘aqov, 7030000, Israel

Location

Bnei Zion Medical Center

Haifa, 3339509, Israel

Location

Shaare Zedek Medical Center

Jerusalem, 9103102, Israel

Location

Scheider Children's Medical Center

Petah Tikva, 49202, Israel

Location

Tel Hashomer Medical Center

Ramat Gan, 5265601, Israel

Location

Kaplan Medical Center

Rehovot, 76100, Israel

Location

Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

Hospital Angeles de Puebla

Puebla City, Reserva Territorial Atilxcayotl, 72190, Mexico

Location

The Liggins Institute

Grafton, Auckland, 1023, New Zealand

Location

Wellington Children's Hospital

Newtown, Wellington Region, 6021, New Zealand

Location

Uniwersyteckie Centrum Kliniczne in Gdansk

Gdansk, 80-211, Poland

Location

Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

Location

Bashkirian State Medical University

Ufa, Bashlortostan Republic, 450008/450106, Russia

Location

Kazan State Medical Univeristy/Pediatric Republic Clinical Hospital

Kazan', Tatarstan Republic, 420012/420138, Russia

Location

Endocrinology Scientific Center

Moscow, 117036, Russia

Location

Pediatric City Clinical Hospital/Russian Medical Academy of Continuous Education

Moscow, 125373, Russia

Location

Saint Petersburg State Pediatric Medical University

Saint Petersburg, 194100, Russia

Location

Voronezh State Medical University

Voronezh, 394024, Russia

Location

Bundang Cha Hospital

Seongnam-si, Gyeonggi-do, 13497, South Korea

Location

Inje University Busan Paik Hospital

Busan, 47392, South Korea

Location

KyungPook National University Hospital

Daegu, 41944, South Korea

Location

Chosun University Hospital

Gwangju, 61453, South Korea

Location

Severance Hospital

Seoul, 03722, South Korea

Location

Hospital Miguel Servet

Zaragoza, Aragon, 50009, Spain

Location

Hospital Josep Trueta

Girona, Catalonia, 17007, Spain

Location

Hospital de Navarra

Pamplona, Navarre, 31008, Spain

Location

Hospital Universitario Central de Asturias

Oviedo, 33011, Spain

Location

Parc Tauli

Sabadell, 15706, Spain

Location

Hospital Universitario de Santiago de Compostela

Santiago de Compostela, 08950, Spain

Location

Taipei Veterans General Hospital

Taipei, 11217, Taiwan

Location

Tri-Service General Hospital

Taipei, 114, Taiwan

Location

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

Kyiv, 01021, Ukraine

Location

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

Kyiv, 04114, Ukraine

Location

Odesa Regional Clinical Children's Hospital

Odesa, 65031, Ukraine

Location

Vinnitsa Regional Clinical Highly Specialized Endocrinology Centre

Vinnitsa, 21010, Ukraine

Location

Zaporizhzhya Regional Clinical Child Hospital

Zaporizhzhya, 69063, Ukraine

Location

Royal Hospital for Children

Glasgow, G514TF, United Kingdom

Location

St. George's University Hospital

London, SW17 OQT, United Kingdom

Location

Related Publications (4)

• 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.

  PMID: 38717038 DERIVED

• 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.

  PMID: 38053515 DERIVED

• 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.

  PMID: 37955005 DERIVED

• 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.

  PMID: 35405011 DERIVED

MeSH Terms

Interventions

MOD-4023; somatrogon; Human Growth Hormone

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: OPKO Health, Inc.
• Organization: OPKO Health, Inc.

contact@opko.com

3055754100

Study Officials

• Tony Cruz

  Sponsor GmbH

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2016
• First Posted: November 18, 2016
• Study Start: December 1, 2016
• Primary Completion: August 1, 2019
• Study Completion: May 1, 2024
• Last Updated: July 20, 2025
• Results First Posted: June 25, 2021

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

NZ (2); UA (5); US (18); IL (9); IN (8); AU (3); GR (2); KR (5); GB (2); AR (2); ES (6); BU (1); GE (2); RU (6); TW (2); CA (1); CO (3); ME (1); BE (1); PL (2)