Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03874013
• Date of registration: 01/10/2018
• Prospective Registration: No
• Primary sponsor: OPKO Health, Inc.
• Public title: Safety and Efficacy Study of MOD-4023 to Treat Children With Growth Hormone Deficiency
• Scientific title: A Phase 3, Open-Label, Randomized, Multicenter, 12-month, Efficacy and Safety Study of Weekly MOD-4023 Compared to Daily Genotropin® Therapy in Japanese Pre-pubertal Children With Growth Hormone Deficiency
• Date of first enrolment: December 7, 2017
• Target sample size: 44
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03874013
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Other. Masking: Single (Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Japan

Contacts

• Name: Reiko Horikawa, M.D, Ph. D.
• Affiliation: National Center for Child Health and Development

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Pre-pubertal child aged = 3 years old, and not yet 10 years for girls (9 years and 364
days) or not yet 11 years for boys (10 years and 364 days), on the date of ICF
signature, with either isolated GHD, or GH insufficiency as part of multiple pituitary
hormone deficiency.

2. Confirmed diagnosis of GHD by 2 different types of GH provocation tests (standardized
on growth foundation data): defined as a peak serum GH level of = 6.0 ng/mL or = 16
ng/mL when conducting GHRP-2 provocation test.

Prior local laboratory results will be accepted subject to pre-approval by the study
medical monitor and if the tests were conducted as specified in the protocol.

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.

5. Height SD score = -2.0 at screening

6. Impaired height velocity defined as:

- Annualized height velocity (HV) below the 25th percentile for CA (HV < -0.7 SDS)
and gender according to the local primary care provider standard.

- The interval between two height measurements should be at least 6 months, but
should not exceed 18 months prior to inclusion.

7. BMI must be within ±2 SDS of mean BMI for the chronological age and sex.

8. Baseline IGF-1 level of at least 1 SDS below the mean IGF-1 level standardized for age
and sex (IGF-1 SDS = -1) according to the central laboratory reference values. A
single re-test will be allowed (subject to discussion with the study medical monitor)
if all other criteria are met.

9. Normal creatinine levels according to common practice reference ranges per age.

10. 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 to ICF signing

11. Normal 46 XX karyotype for girls.

12. Willing and able to provide written informed consent of the parent or legal guardian
of the patient and written assent from pediatric patients (when applicable based on
age and Japan regulation).

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 suspected psychosocial dwarfism by the discretion of the investigator

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. Children with diabetes mellitus

9. Chromosomal abnormalities including Turner's syndrome, Laron syndrome, Noonan
syndrome, Prader-Willi syndrome, Russell-Silver syndrome, SHOX (short stature
homeobox) mutations/deletions and skeletal dysplasia's, with the exception of
septo-optic dysplasia.

10. Concomitant administration of other treatments that may have an effect on growth such
as anabolic steroids, sex steroids, with the exception of ADHD drugs or hormone
replacement therapies (thyroxin, hydrocortisone, desmopressin [DDAVP])

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 as
provided in Appendix J.

12. Major medical conditions and/or presence of contraindication to r-hGH treatment.

13. Known or suspected HIV-positive patient, or patient with advanced diseases such as
AIDS or tuberculosis.

14. Drug substance or alcohol abuse.

15. Known hypersensitivity to the components of study medication.

16. Other causes of short stature such as celiac disease, uncontrolled primary
hypothyroidism and rickets.

17. The patient and/or the parent/legal guardian are likely to be non-compliant in respect
to study conduct.

18. Participation in any other clinical trial within 30 days prior to screening and
throughout the entire study period (including administration of investigational
agent).



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

Health Condition(s) or Problem(s) studied

Growth Hormone Deficiency

Intervention(s)

Drug: Genotropin

Drug: MOD-4023

Primary Outcome(s)

Annual Height Velocity (HV) after 12 months [Time Frame: 12 months]

Secondary Outcome(s)

Change in height standard deviation score (SDS) compared to baseline [Time Frame: 12 months]

Change in bone maturation (BM) [Time Frame: 12 months]

Height Velocity at 6 months [Time Frame: 6 months]

Secondary ID(s)

CP-4-009

Source(s) of Monetary Support

Please refer to primary and secondary sponsors