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

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ClinicalTrials.gov Identifier: NCT02968004
Recruitment Status : Recruiting
First Posted : November 18, 2016
Last Update Posted : June 4, 2018
Sponsor:
Information provided by (Responsible Party):
Opko Biologics

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.

Condition or disease Intervention/treatment Phase
Pediatric Growth Hormone Deficiency Drug: MOD-4023 Drug: Somatropin Phase 3

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  (Clinical Trial)
Estimated Enrollment : 220 participants
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
Study Start Date : December 2016
Estimated Primary Completion Date : September 2019
Estimated Study Completion Date : September 2019


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 Name: Somatrogon

Active Comparator: Genotropin
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
Drug: Somatropin
Once daily subcutaneous injection of Genotropin
Other Name: Genotropin




Primary Outcome Measures :
  1. Annual Height Velocity [ Time Frame: 52 weeks ]
    Annual change in Height Velocity in cm


Secondary Outcome Measures :
  1. Height velocity at 6 months [ Time Frame: After 6 months of treatment ]
    Height velocity in cm measured after 6 months of treatment

  2. Change in height Standard Deviation Score (SDS) [ Time Frame: After 6 months and then every 52 weeks ]
    Compared to baseline

  3. Change in bone maturation (BM) [ Time Frame: 52 weeks ]
    Annual change in bone age measurements as per Gruelich-Pyle method

  4. IGF-1 serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis

  5. IGF-1 SDS serum levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis

  6. IGFBP-3 levels [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis

  7. IGFBP-3 SDS [ Time Frame: On day 4(-1) after MOD-4023 dosing across study visits ]
    Via central lab analysis


Other Outcome Measures:
  1. 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

  2. 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)

  3. Quality of Life [ Time Frame: 12 months ]
    Total score measured by the QoLISSY questionnaire



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

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.

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: Shelly Vander 972 8 9543100 ext 116 svander@opko.com

  Show 139 Study Locations
Sponsors and Collaborators
Opko Biologics
Investigators
Study Director: Tony Cruz Sponsor GmbH

Responsible Party: Opko Biologics
ClinicalTrials.gov Identifier: NCT02968004     History of Changes
Other Study ID Numbers: CP-4-006
First Posted: November 18, 2016    Key Record Dates
Last Update Posted: June 4, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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