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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2024
• Main ID: NCT02968004
• Date of registration: 13/11/2016
• Prospective Registration: Yes
• Primary sponsor: OPKO Health, Inc.
• 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
• Date of first enrolment: December 2016
• Target sample size: 224
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02968004
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belarus, Bulgaria, Canada, Colombia, Georgia, Germany, Greece, India, Israel, Italy, Korea, Republic of, Mexico, New Zealand, Poland, Russian Federation, Spain, Taiwan, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Tony Cruz
• Affiliation: Sponsor GmbH

Key inclusion & exclusion criteria

Main Study Inclusion Criteria:

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 Inclusion Criteria:

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 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 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.



Age minimum: 3 Years
Age maximum: 11 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pediatric Growth Hormone Deficiency

Intervention(s)

Drug: Somatropin

Drug: MOD-4023

Primary Outcome(s)

Annual Height Velocity [Time Frame: 52 weeks]

Secondary Outcome(s)

Height Velocity at 6 Months [Time Frame: After 6 months of treatment]

Change in Bone Maturation (BM) [Time Frame: 52 weeks]

Change in Height Standard Deviation Score (SDS) [Time Frame: After 6 and 12 months]

Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS) [Time Frame: Baseline and at 12 months]

Secondary ID(s)

CP-4-006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 25/06/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02968004