Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

OPKO Health

Status and phase: Completed; Phase 2

Conditions: Growth Hormone Deficiency (GHD)

Treatments: Drug: MOD-4023

Study type: Interventional

Funder types: Industry

Identifiers

NCT02500316

CP-4-004-OLE

2011-004553-60 (EudraCT Number)

Details and patient eligibility

About

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.

Enrollment: 48 patients

Sex: All

Ages: 3 to 11 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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48 participants in 1 patient group

MOD-4023

Experimental group

Description:

Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).

Treatment:

Drug: MOD-4023