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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT02968004
Registration number
NCT02968004
Ethics application status
Date submitted
13/11/2016
Date registered
18/11/2016
Date last updated
9/02/2024
Titles & IDs
Public title
Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
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Scientific 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
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Secondary ID [1]
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CP-4-006
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pediatric Growth Hormone Deficiency
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Condition category
Condition code
Metabolic and Endocrine
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Other endocrine disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - MOD-4023
Treatment: Drugs - Somatropin
Experimental: MOD-4023 - Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
Active Comparator: Genotropin - Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
Treatment: Drugs: MOD-4023
Once weekly subcutaneous injection using pre-filled pen device.
Treatment: Drugs: Somatropin
Once daily subcutaneous injection of Genotropin using pre-filled pen device.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Annual Height Velocity
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Assessment method [1]
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Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.
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Timepoint [1]
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52 weeks
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Secondary outcome [1]
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Height Velocity at 6 Months
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Assessment method [1]
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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.
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Timepoint [1]
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After 6 months of treatment
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Secondary outcome [2]
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Change in Height Standard Deviation Score (SDS)
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Assessment method [2]
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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.
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Timepoint [2]
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After 6 and 12 months
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Secondary outcome [3]
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Change in Bone Maturation (BM)
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Assessment method [3]
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Annual change in bone age measurements as per Gruelich-Pyle method
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Timepoint [3]
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52 weeks
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Secondary outcome [4]
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Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)
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Assessment method [4]
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Via central lab analysis
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Timepoint [4]
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Baseline and at 12 months
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Eligibility
Key inclusion criteria
Main Study
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
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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
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Minimum age
3
Years
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Maximum age
11
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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.
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/12/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2024
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Actual
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Sample size
Target
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Accrual to date
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Final
224
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Recruitment in Australia
Recruitment state(s)
NSW,WA
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Recruitment hospital [1]
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John Hunter Children's Hospital - New Lambton Heights
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Recruitment hospital [2]
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Children's Hospital at Westmead - Westmead
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Recruitment hospital [3]
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PM Hospital for Children - Subiaco
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Recruitment postcode(s) [1]
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2305 - New Lambton Heights
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Recruitment postcode(s) [2]
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2145 - Westmead
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Recruitment postcode(s) [3]
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6008 - Subiaco
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Recruitment outside Australia
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
OPKO Health, Inc.
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Address
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Ethics approval
Ethics application status
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Summary
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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT02968004
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Tony Cruz
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Address
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Sponsor GmbH
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT02968004
Download to PDF