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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02500316
Recruitment Status : Active, not recruiting
First Posted : July 16, 2015
Last Update Posted : December 13, 2019
Sponsor:
Information provided by (Responsible Party):
OPKO Health, Inc.

Brief Summary:
Protocol CP-4-004-EXT is designed as a long-term, open-label extension using single patient use, multi-dose, disposable pre-filled pen.

Condition or disease Intervention/treatment Phase
Growth Hormone Deficiency (GHD) Drug: MOD-4023 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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 Start Date : February 2013
Actual Primary Completion Date : July 2015
Estimated Study Completion Date : December 2021


Arm Intervention/treatment
Experimental: MOD-4023
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).
Drug: MOD-4023
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).




Primary Outcome Measures :
  1. Annual Height Velocity [ Time Frame: 8 years ]
  2. Delta height SDS every 12 months [ Time Frame: 8 years ]

Secondary Outcome Measures :
  1. Absolute IGF-I levels on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]
  2. IGF-I SDS on day 4 after MOD-4023 dosing [ Time Frame: 8 years ]

Other Outcome Measures:
  1. IGFBP-3 levels on day 3 or 4 after MOD-4023 dosing [ Time Frame: 8 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   3 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02500316


Locations
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United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Belarus
2DKB
Minsk, Belarus
Greece
Children's Hospital "P. A. Kyriakou"
Athens, Greece
Hungary
Buda Children's Hospital
Budapest, Hungary
Russian Federation
Endocrinology Scientific Centre, Institute of Child Endocrinology
Moscow, Russian Federation
Russian Medical Academy of Postgraduate Education
Moscow, Russian Federation
SPGPMA
Saint Petersburg, Russian Federation
SamGMU
Samara, Russian Federation
SBEIHPE
Ufa, Russian Federation
Ukraine
Donetsk Regional Children Clinical Hospital
Donetsk, Ukraine
Institute of Endocrinology
Kiev, Ukraine
Ukrainian Scientific Center of Endocrine Surgery Moh of Ukraine
Kiev, Ukraine
Odessa Regional Children'S Clinical Hospital
Odessa, Ukraine
Sponsors and Collaborators
OPKO Health, Inc.
Investigators
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Principal Investigator: Zvi Zadik, MD Kaplan Medical Center, Israel
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Responsible Party: OPKO Health, Inc.
ClinicalTrials.gov Identifier: NCT02500316    
Other Study ID Numbers: CP-4-004-extension
2011-004553-60 ( EudraCT Number )
First Posted: July 16, 2015    Key Record Dates
Last Update Posted: December 13, 2019
Last Verified: December 2019
Keywords provided by OPKO Health, Inc.:
Growth hormone deficiency (GHD)
r-hGH
Long-acting