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

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02968004
Recruitment Status : Active, not recruiting
First Posted : November 18, 2016
Last Update Posted : December 13, 2019
Sponsor:
Information provided by (Responsible Party):
OPKO Health, Inc.

Tracking Information
First Submitted Date  ICMJE November 13, 2016
First Posted Date  ICMJE November 18, 2016
Last Update Posted Date December 13, 2019
Study Start Date  ICMJE December 2016
Actual Primary Completion Date August 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 31, 2018)
Annual Height Velocity [ Time Frame: 52 weeks ]
Annual change in Height Velocity in cm
Original Primary Outcome Measures  ICMJE
 (submitted: November 15, 2016)
Annual Height Velocity [ Time Frame: 12 months of treatment ]
Annual Height Velocity in cm/year measured after 12 months of treatment
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 31, 2018)
  • Height velocity at 6 months [ Time Frame: After 6 months of treatment ]
    Height velocity in cm measured after 6 months of treatment
  • Change in height Standard Deviation Score (SDS) [ Time Frame: After 6 months and then every 52 weeks ]
    Compared to baseline
  • Change in bone maturation (BM) [ Time Frame: 52 weeks ]
    Annual change in bone age measurements as per Gruelich-Pyle method
  • IGF-1 serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis
  • IGF-1 SDS serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis
  • IGFBP-3 levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis
  • IGFBP-3 SDS [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis
Original Secondary Outcome Measures  ICMJE
 (submitted: November 15, 2016)
  • 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 ]
Current Other Pre-specified Outcome Measures
 (submitted: July 23, 2018)
  • 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
  • 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)
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Official Title  ICMJE 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
Brief Summary 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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pediatric Growth Hormone Deficiency
Intervention  ICMJE
  • Drug: MOD-4023
    Once weekly subcutaneous injection using pre-filled pen device.
    Other Name: Somatrogon
  • Drug: Somatropin
    Once daily subcutaneous injection of Genotropin
    Other Name: Genotropin
Study Arms  ICMJE
  • Experimental: MOD-4023
    Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
    Intervention: Drug: MOD-4023
  • Active Comparator: Genotropin
    Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
    Intervention: Drug: Somatropin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: November 7, 2018)
224
Original Estimated Enrollment  ICMJE
 (submitted: November 15, 2016)
220
Estimated Study Completion Date  ICMJE December 2022
Actual Primary Completion Date August 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Main Study 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 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 using Tanner, Prader, and Hermanussen
    • 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
  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. Provide consent/assent

    LT-OLE Inclusion Criteria:

  11. Completion of the main study (12 months of treatment) with adequate compliance.
  12. Provide consent/assent
  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 HRT (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. Possible 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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Argentina,   Australia,   Belarus,   Bulgaria,   Canada,   Colombia,   Georgia,   Greece,   India,   Israel,   Korea, Republic of,   Mexico,   New Zealand,   Poland,   Russian Federation,   Spain,   Taiwan,   Ukraine,   United Kingdom,   United States
Removed Location Countries Germany,   Italy,   Turkey
 
Administrative Information
NCT Number  ICMJE NCT02968004
Other Study ID Numbers  ICMJE CP-4-006
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party OPKO Health, Inc.
Study Sponsor  ICMJE OPKO Health, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Tony Cruz Sponsor GmbH
PRS Account OPKO Health, Inc.
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP