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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02968004




Registration number
NCT02968004
Ethics application status
Date submitted
13/11/2016
Date registered
18/11/2016

Titles & IDs
Public title
Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
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
Secondary ID [1] 0 0
CP-4-006
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pediatric Growth Hormone Deficiency 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
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.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Annual Height Velocity
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
Height Velocity at 6 Months
Timepoint [1] 0 0
After 6 months of treatment
Secondary outcome [2] 0 0
Change in Height Standard Deviation Score (SDS)
Timepoint [2] 0 0
After 6 and 12 months
Secondary outcome [3] 0 0
Change in Bone Maturation (BM)
Timepoint [3] 0 0
52 weeks
Secondary outcome [4] 0 0
Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)
Timepoint [4] 0 0
Baseline and at 12 months

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

LT-OLE
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
Minimum age
3 Years
Maximum age
11 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

LT-OLE
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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/05/2024
Sample size
Target
Accrual to date
Final
224
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
John Hunter Children's Hospital - New Lambton Heights
Recruitment hospital [2] 0 0
Children's Hospital at Westmead - Westmead
Recruitment hospital [3] 0 0
PM Hospital for Children - Subiaco
Recruitment postcode(s) [1] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
6008 - Subiaco
Recruitment outside Australia
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United States of America
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California
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Colorado
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Florida
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Idaho
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Iowa
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Massachusetts
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Nevada
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New York
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Ohio
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Pennsylvania
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Texas
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Washington
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Argentina
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Entre Rios
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Argentina
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Córdoba
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Belarus
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Minsk
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Bulgaria
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Varna
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Canada
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Quebec
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Colombia
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Bogotá
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Colombia
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Medellín
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Georgia
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Batumi
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Georgia
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Tbilisi
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Greece
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Athens
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India
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Nagpur
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Afula
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Haifa
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Jerusalem
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Israel
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Petach Tikva
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Israel
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Ramat Gan
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Israel
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Re?ovot
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Israel
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Tel Aviv
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Korea, Republic of
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Gyeonggi-do
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Busan
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Daegu
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Gwangju
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Seoul
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Mexico
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Reserva Territorial Atilxcayotl
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New Zealand
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Auckland
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New Zealand
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Wellington
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Poland
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Gdansk
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Poland
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Lódz
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Russian Federation
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Bashlortostan Republic
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Russian Federation
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Tatarstan Republic
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Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Russian Federation
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Voronezh
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Spain
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Aragon
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Cataluna
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Navarra
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Oviedo
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Spain
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Sabadell
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Spain
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Santiago De Compostela
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Taiwan
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Taipei
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Ukraine
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Kyiv
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Ukraine
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Odesa
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Ukraine
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Vinnitsa
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Ukraine
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Zaporizhzhya
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United Kingdom
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Glasgow
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United Kingdom
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London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
OPKO Health, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Tony Cruz
Address 0 0
Sponsor GmbH
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT02968004