Brief Title
Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Official Title
Safety and Dose Finding Study of Different MOD-4023 Dose Levels Compared to Daily r-hGH Therapy in Pre-pubertal Growth Hormone Deficient Children
Brief Summary
This is a phase II, open label, active-controlled, randomized safety and dose finding study
of different MOD-4023 dose levels compared to daily r-hGH therapy in pre-pubertal growth
hormone deficient children.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Annual Height Velocity
Secondary Outcome
Height Velocity at 6 Months
Condition
Pediatric Growth Hormone Deficiency
Intervention
MOD-4023
Study Arms / Comparison Groups
MOD-4023 low dose
Description: Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
56
Start Date
February 2012
Completion Date
July 2015
Primary Completion Date
July 2015
Eligibility Criteria
Inclusion Criteria:
1. Pre-pubertal child aged ≥ 3 yrs old and not above 10 years for girls or 11 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 ≤7 ng/ml, determined by central laboratory using a validated assay
.
3. Bone age (BA) is not older than chronological age and should be no greater than 9
years for girls and 10 years for boys.
4. Without prior exposure to any r-hGH therapy.
5. Normal birth size, birth weight and length for gestational age
6. Impaired height and height velocity defined as:
1. Height (HT) of at least 2.0 standard deviations (SD) below the mean height for
chronological age (CA) and gender according to the standards from Prader et. al,
1989 , (HT SDS ≤ -2.0).
2. Annualized height velocity (HV) below the 25th percentile for CA (HV <-0.7 SDS)
and gender according to the standards of Prader et al (1989). The interval
between two height measurements should be at least 6 months before inclusion.
7. BMI must be within ±2 SD of mean BMI for the chronological age and sex according to
the 2000 CDC standards.
8. Baseline IGF-I level of at least 1 SD below the mean IGF-I level standardized for age
and sex (IGF-I SDS ≤ -1.0) according to the central laboratory reference values;
9. 12. Written informed consent of the parent or legal guardian of the patient and assent
of the patient (if the patient can read).
Exclusion Criteria:
1. Children with past or present intracranial tumor growth as confirmed by an MRI scan
(with contrast).
2. History of radiation therapy or chemotherapy.
3. Malnourished children defined as:
1. Serum albumin below the lower limit of normal (LLN) according to the reference
ranges of central laboratory;
2. Serum iron below the lower limit of normal (LLN) according to the reference
ranges of central laboratory;
3. BMI < -2 SD for age and sex;
4. Children with psychosocial dwarfism.
5. Children born small for gestational age (SGA - birth weight and/or birth length < -2
SD 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. Patients with diabetes mellitus.
9. Patients with impaired fasting sugar (based on WHO; fasting blood sugar >110 mg/dl or
6.1 mmol/l) after repeated blood analysis.
10. Chromosomal abnormalities and medical "syndromes" (Turner's syndrome, Laron syndrome,
Noonan syndrome, Prader-Willi Syndrome, Russell-Silver Syndrome, SHOX
mutations/deletions and skeletal dysplasias), with the exception of septo-optic
dysplasia.
11. Closed epiphyses.
12. Concomitant administration of other treatments that may have an effect on growth such
as anabolic steroids and methylphenidate for attention deficit hyperactivity disorder
(ADHD), with the exception of hormone replacement therapies (thyroxine,
hydrocortisone, desmopressin (DDAVP))
13. Children requiring glucocorticoid therapy (e.g. asthma) who are taking a dose of
greater than 400 µg/d of inhaled budesonide or equivalents for longer than 1 month
during a calendar year.
14. Major medical conditions and/or presence of contraindication to r-hGH treatment.
15. Known or suspected HIV-positive patient, or patient with advanced diseases such as
AIDS or tuberculosis.
16. Drug, substance, or alcohol abuse.
17. Known hypersensitivity to the components of study medication.
18. Other causes of short stature such as coeliac disease, hypothyroidism and rickets.
19. The patient and/or the parent/legal guardian are likely to be non-compliant in respect
to study conduct.
20. Participation in any other trial of an investigational agent within 30 days prior to
Screening.
Gender
All
Ages
3 Years - 11 Years
Accepts Healthy Volunteers
No
Contacts
Zvi Zadik, MD, ,
Location Countries
Greece
Location Countries
Greece
Administrative Informations
NCT ID
NCT01592500
Organization ID
CP-4-004
Secondary IDs
2011-004553-60
Responsible Party
Sponsor
Study Sponsor
OPKO Health, Inc.
Study Sponsor
Zvi Zadik, MD, Principal Investigator, Kaplan Medical Center, Israel
Verification Date
January 2020