Clinical trial NCT02500316: Long Term Follow up 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-human Growth Hormone (hGH) Therapy in Pre-pubertal Growth Hormone Deficient Children
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Growth Hormone Deficiency (GHD)
Keywords: Growth hormone deficiency (GHD)
r-hGH
Long-acting
Lead sponsor: OPKO Health, Inc.
Sponsor class: INDUSTRY
First posted: 2015-07-16
Enrollment: 48
Sex: ALL
Minimum age (years): 3
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Protocol CP-4-004-OLE (Open Label Extension) is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.


Eligibility criteria: Inclusion Criteria:

Patients who completed the first year of treatment in the main study are allowed to enter the (Long Term) Open Label Extension (OLE) study.

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 Standard Deviation 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, short stature homeobox-containing gene (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.

Primary outcomes:
measure: Annualized Height Velocity
timeFrame: 8 years
measure: Delta Height SDS Every 12 Months
timeFrame: 8 years
Secondary outcomes:
measure: Summary of IGF-1 SDS
timeFrame: 8 years
Other outcomes:
Locations:
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Minsk
country: Belarus
facility: 2DKB
city: Athens
country: Greece
facility: Children's Hospital "P. A. Kyriakou"
city: Budapest
country: Hungary
facility: Buda Children's Hospital
city: Moscow
country: Russian Federation
facility: Endocrinology Scientific Centre, Institute of Child Endocrinology
city: Moscow
country: Russian Federation
facility: Russian Medical Academy of Postgraduate Education
city: Saint Petersburg
country: Russian Federation
facility: SPGPMA
city: Samara
country: Russian Federation
facility: SamGMU
city: Ufa
country: Russian Federation
facility: SBEIHPE
city: Donetsk
country: Ukraine
facility: Donetsk Regional Children Clinical Hospital
city: Kiev
country: Ukraine
facility: Institute of Endocrinology
city: Kiev
country: Ukraine
facility: Ukrainian Scientific Center of Endocrine Surgery Moh of Ukraine
city: Odessa
country: Ukraine
facility: Odessa Regional Children'S Clinical Hospital
Study officials:
affiliation: Kaplan Medical Center, Israel
name: Zvi Zadik, MD
role: PRINCIPAL_INVESTIGATOR
References: