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

This study is currently recruiting participants.
Verified October 2016 by Opko Biologics
Sponsor:
ClinicalTrials.gov Identifier:
NCT02968004
First Posted: November 18, 2016
Last Update Posted: September 7, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Opko Biologics
  Purpose
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.

Condition Intervention Phase
Pediatric Growth Hormone Deficiency Drug: MOD-4023 Drug: Somatropin Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Opko Biologics:

Primary Outcome Measures:
  • Annual Height Velocity [ Time Frame: 12 months of treatment ]
    Annual Height Velocity in cm/year measured after 12 months of treatment


Secondary Outcome Measures:
  • 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 bone maturation (BM) [ Time Frame: After 12 months of treatment ]
  • IGF-1 serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
  • IGF-1 SDS serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
  • IGFBP-3 levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
  • IGFBP-3 SDS [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]

Estimated Enrollment: 220
Study Start Date: December 2016
Estimated Primary Completion Date: February 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.
Active Comparator: Genotropin
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
Drug: Somatropin
Once daily subcutaneous injection of Genotropin
Other Name: Genotropin

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Pre-pubertal children aged ≥3 years , and not yet 11 years for girls or not yet 12 years 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 rhGH therapy (naïve patients).
  5. Impaired height velocity defined as:

    o Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS) and gender according

  6. BMI must be within ±2 SDS of mean BMI for the chronological age and sex.
  7. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age and sex (IGF-I SDS ≤-1)
  8. Normal calculated GFR
  9. Normal 46XX karyotype for girls.

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. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX mutations/deletions and skeletal dysplasias.
  8. Concomitant administration of other treatments that may have an effect on growth
  9. Major medical conditions and/or presence of contraindication to r-hGH treatment.
  10. Closed epiphyses
  11. Known or suspected HIV-positive patient, or patient with advanced diseases such as AIDS or tuberculosis.
  12. Drug, substance, or alcohol abuse.
  13. Known hypersensitivity to the components of study medication.
  14. Other causes of short stature such as celiac disease, uncontrolled primary hypothyroidism and rickets.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02968004


Contacts
Contact: Gili Hart, PhD 972-8-9300051 ghart@opko.com

Locations
United States, New York
Paul Saenger Recruiting
Mineola, New York, United States, 11501
Contact: Paul saenger, MD, PhD       phsaenger@gmail.com   
Colombia
Hospital Universitario San Vicente de Paul Not yet recruiting
Medellin, Antioquia, Colombia
Contact: Nora Zuluaga    (57) 3122582340      
Hospital Pablo Tobon Uribe Not yet recruiting
Medellín, Antioquia, Colombia
Contact: Veronica Abad, MD.       abadvero@yahoo.com   
Fundación CardioInfantil Not yet recruiting
Bogotá, Colombia
Contact: Jeisson Arevalo       jarevalo@cardioinfantil.org   
Sponsors and Collaborators
Opko Biologics
  More Information

Responsible Party: Opko Biologics
ClinicalTrials.gov Identifier: NCT02968004     History of Changes
Other Study ID Numbers: CP-4-006
First Submitted: November 13, 2016
First Posted: November 18, 2016
Last Update Posted: September 7, 2017
Last Verified: October 2016

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs