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

February 6, 2024 updated by: OPKO Health, Inc.

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 will be 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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The study will consist 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.

Study Type

Interventional

Enrollment (Actual)

224

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 11 years (Child)

Accepts Healthy Volunteers

No

Description

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-I 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: MOD-4023
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
Drug: MOD-4023
Once weekly subcutaneous injection using pre-filled pen device.
Other Names:
  • Somatrogon
Active Comparator: Genotropin
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
Drug: Somatropin
Once daily subcutaneous injection of Genotropin using pre-filled pen device.
Other Names:
  • Genotropin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annual Height Velocity
Time Frame: 52 weeks
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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Height Velocity at 6 Months
Time Frame: After 6 months of treatment
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.
After 6 months of treatment
Change in Height Standard Deviation Score (SDS)
Time Frame: After 6 and 12 months
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.
After 6 and 12 months
Change in Bone Maturation (BM)
Time Frame: 52 weeks
Annual change in bone age measurements as per Gruelich-Pyle method
52 weeks
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)
Time Frame: Baseline and at 12 months
Via central lab analysis
Baseline and at 12 months

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Device
Time Frame: 6 weeks
Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA
6 weeks
Device
Time Frame: 1 week
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)
1 week

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

OPKO Health, Inc.

Investigators

  • Study Director: Tony Cruz, Sponsor GmbH

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2016

Primary Completion (Actual)

August 1, 2019

Study Completion (Estimated)

June 1, 2024

Study Registration Dates

First Submitted

November 13, 2016

First Submitted That Met QC Criteria

November 15, 2016

First Posted (Estimated)

November 18, 2016

Study Record Updates

Last Update Posted (Estimated)

February 9, 2024

Last Update Submitted That Met QC Criteria

February 6, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CP-4-006

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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