Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by Prolor Biotech, Inc.
Sponsor:
Information provided by (Responsible Party):
Prolor Biotech, Inc.
ClinicalTrials.gov Identifier:
NCT01592500
First received: May 2, 2012
Last updated: December 23, 2013
Last verified: December 2013
  Purpose

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.


Condition Intervention Phase
Pediatric Growth Hormone Deficiency
Drug: MOD-4023
Drug: Somatropin
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Prolor Biotech, Inc.:

Primary Outcome Measures:
  • Annual Height Velocity [ Time Frame: 12 months of treatment ] [ Designated as safety issue: No ]
    Annual Height Velocity in cm/year measured after 12 months of treatment


Secondary Outcome Measures:
  • Height velocity at 6 months [ Time Frame: After 6 months of treatment ] [ Designated as safety issue: No ]
  • Change in height Standard Deviation Score (SDS) [ Time Frame: After 6 and 12 months of treatment ] [ Designated as safety issue: No ]
    Change in height Standard Deviation Score (compared to normal population of same age group)

  • IGF-1 serum levels [ Time Frame: Once monthly on day 4 after the last dose ] [ Designated as safety issue: Yes ]
    IGF-1 and IGFBP-3 serum levels measured once monthly on day 4 after the last dose


Estimated Enrollment: 56
Study Start Date: February 2012
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: MOD-4023 low dose
Once weekly subcutaneous injection of MOD-4023
Drug: MOD-4023
Once weekly subcutaneous injection
Experimental: MOD-4023 middle dose
Once weekly subcutaneous injection of MOD-4023
Drug: MOD-4023
Once weekly subcutaneous injection
Experimental: MOD-4023 high dose
Once weekly subcutaneous injection of MOD-4023
Drug: MOD-4023
Once weekly subcutaneous injection
Active Comparator: Genotropin
Once daily subcutaneous injection of Genotropin (r-hGH)
Drug: Somatropin
Once daily subcutaneous injection of Genotropin

  Eligibility

Ages Eligible for Study:   3 Years to 11 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01592500

Contacts
Contact: Richard Sandor +36 (1) 299-0091 ext 204 R.Sandor@accelsiors.com
Contact: Leanne Amitzi, M.Sc. +972-8-9300051 ext 129 leanne@prolor-biotech.com

Locations
Greece
Children's Hospital "P. A. Kyriakou" Recruiting
Athens, Greece
Contact: Elpis Vlachopapadopoulou, MD         
Principal Investigator: Elpis Vlachopapadopoulou, MD         
Hungary
Buda Children's Hospital Recruiting
Budapest, Hungary
Contact: Agota Muzsnai, MD         
Principal Investigator: Agota Muzsnai, MD         
Heim Pal Children's Hospital Recruiting
Budapest, Hungary
Contact: Zsolt Vajda, MD         
Principal Investigator: Zsolt Vajda, MD         
Slovakia
Children's University Hospital Recruiting
Kosice, Slovakia
Contact: Magdalena Paskova, MD         
Principal Investigator: Magdalena Paskova, MD         
Sponsors and Collaborators
Prolor Biotech, Inc.
Investigators
Principal Investigator: Zvi Zadik, MD Kaplan Medical Center, Israel
  More Information

No publications provided

Responsible Party: Prolor Biotech, Inc.
ClinicalTrials.gov Identifier: NCT01592500     History of Changes
Other Study ID Numbers: CP-4-004, 2011-004553-60
Study First Received: May 2, 2012
Last Updated: December 23, 2013
Health Authority: United States: Food and Drug Administration
Belarus: Ministry of Public Health
Bulgaria: Bulgarian Drug Agency
Czech Republic: State Institute for Drug Control
Greece: Ministry of Health and Social Solidarity, National Organization for Medicines (EOF)
Hungary: National Institute of Pharmacy
Israel: Ministry of Health, Pharmaceutical department
Macedonia: Ministry of Health
Poland: Central Registry of Clinical Trials Office for Registration of Medicinal Products, Medical Devices and Biocides
Romania: National Medicines Agency
Russia: Department of Ministry of Health
Slovak Republic: State Institute for Drug Control
Ukraine: The State Expert Center of the Ministry of Health of Ukraine

Keywords provided by Prolor Biotech, Inc.:
GHD
r-hGH
Long-acting

Additional relevant MeSH terms:
Pituitary Diseases
Dwarfism, Pituitary
Endocrine System Diseases
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Hypopituitarism
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases

ClinicalTrials.gov processed this record on July 31, 2014