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Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
EMD Serono
ClinicalTrials.gov Identifier:
NCT00109733
First received: May 2, 2005
Last updated: August 4, 2013
Last verified: August 2013
  Purpose

The primary objective is to evaluate the efficacy and safety of two different dose regimens of r-hGH (Saizen®) in subjects with childhood-onset growth hormone deficiency (COGHD) during the transition phase from childhood to adulthood.


Condition Intervention Phase
Childhood-onset Growth Hormone Deficiency
Pituitary Dwarfism
Biological: recombinant human growth hormone
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase IIIb, Prospective, Multicenter, Randomized, Open-label Study to Determine the Safety and Efficacy of Two Different Dosing Regimens of Saizen® (Recombinant Human Growth Hormone (r-hGH), Using Cool.Click™ in Subjects With Childhood-onset Growth Hormone Deficiency During the Adolescent Transition Phase (CATS)

Resource links provided by NLM:


Further study details as provided by EMD Serono:

Primary Outcome Measures:
  • Percent Change From Baseline to Week 24 in Trunk Fat [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percent Change From Baseline to Week 24 in Lean Body Mass [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]
  • Percent Change From Baseline to Week 24 in Total Body Fat [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]
  • Percent Change From Baseline to Week 24 in Limb Fat [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]
  • Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio [ Time Frame: Baseline to Week 24 ] [ Designated as safety issue: No ]

Enrollment: 31
Study Start Date: January 2005
Study Completion Date: July 2006
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Standard dose group
0.005 mg/kg/day recombinant human growth hormone (r-hGH) for 30 days then increasing, with the Investigator's approval, to 0.010 mg/kg/day from Day 31 to Week 24.
Biological: recombinant human growth hormone
0.005 mg/kg/day for 30 days then increasing, with the Investigator's approval, to 0.010 mg/kg/day from Day 31 to Week 24.
Other Names:
  • Saizen
  • r-hGH
  • cool click
Experimental: High dose group
0.010 mg/kg/day recombinant human growth hormone for 14 days with the opportunity to dose escalate, with the Investigator's approval, on Day 15 to 0.02 mg/kg/day and Day 29 to 0.03 mg/kg/day.
Biological: recombinant human growth hormone
0.010 mg/kg/day for 14 days with the opportunity to dose escalate, with the Investigator's approval, on Day 15 to 0.02 mg/kg/day and Day 29 to 0.03 mg/kg/day.
Other Names:
  • Saizen
  • r-hGH
  • cool click

Detailed Description:

This is a phase IIIb, prospective, multicenter, randomised, open label study to determine the safety and efficacy of two different dose regimens of r-hGH with a dose escalation scheme. Screening assessments must be completed 30 days prior to SD1 (Study Day 1). Eligible subjects ages 13 to 25 years will be randomised in equal allocation in a 1:1 ratio to one of two treatment groups (30 subjects/group). Daily subcutaneous injections will be self-administered or received from a designated individual using cool.click™, the needle-free growth hormone (GH) delivery device. The study consists of three periods: screening (up to 30 days prior to Study Day 1), active treatment (up to 24 weeks), and follow-up (4 week safety evaluation after the last dose of study medication).

Each subject will be required to complete a daily treatment diary to assess dosing compliance, adverse events, and concomitant medications. Each subject will receive one treatment diary at SD1, weeks 8, 12, and 24. Subjects will be required to record daily diary entries that will capture dosing compliance, adverse events, and concomitant medications. Depending upon treatment allocation and subject tolerability, dose titration will be increased as follows:

  • Group A: 0.005 mg/kg/day for 30 days then increasing, with the Investigator's approval, to 0.010 mg/kg/day from day 31 to week 24.
  • Group B: 0.010 mg/kg/day for 14 days with the opportunity to dose escalate, with the Investigator's approval, on day 15 to 0.02 mg/kg/day and day 29 to 0.03 mg/kg/day.

Scheduled study visits include screening, baseline, and weeks 8, 12, and 24. Dosage adjustments will be based on subject tolerability and telephone assessments from study drug initiation through week 6. Trunk fat will be measured at SD1, weeks 12 and 24 (or early termination visit). Routine clinical laboratory assessments (hematology, blood chemistries, and urinalysis) will be performed pre-treatment (-30 to -1 SD1) and post-treatment on week 24 (or early termination visit). Special laboratory assessments include the central analysis of lipid panel, fasting insulin, fasting glucose, insulin-like growth factor I (IGF-I), insulin-like growth factor binding protein 3 (IGFBP-3), free thyroxine (T4) , total T4, C-reactive protein (CRP). Physical exams will be performed at screening, weeks 12 and 24. Safety evaluations will occur during scheduled study visits, through telephone assessments, and by the review of adverse events and concomitant events on the subject treatment diary.

  Eligibility

Ages Eligible for Study:   13 Years to 25 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

The day of entry or Study Day 1 is defined as the first day of study treatment. To be eligible for inclusion into this study, the subjects must fulfill all of the following criteria within 30 days prior to Study Day 1.

  • Male or female from 13 to 25 years of age, inclusive
  • Diagnosis of childhood onset growth hormone deficiency (GHD) and prior completed growth hormone (GH) treatment as evidenced by bone age greater than 14 years for girls and 16 years for boys or no height increase > 0.5 cm in the 6 months prior to Screen.
  • Have documented GH deficiency (acquired or idiopathic), established by a standard provocative test, such as insulin (<5 ng/mL) or growth hormone releasing hormone plus arginine (<9 ng/mL) at least 30 days after GH has been discontinued. If a subject is hypopituitary with two or more pituitary disorders and has a low IGF-1, the stimulation test does not need to be performed to confirm GHD.
  • If hypopituitary, must have been on adequate replacement therapy (if required) of glucocorticosteroids, thyroid and sex hormones (hormone levels on replacement being in normal/mildly elevated range) for at least 6 months prior to Screen.
  • Be willing and able to comply with the protocol for the duration of the study.
  • Have given written informed consent before any study-related procedure not part of the subject's normal medical care, with the understanding that the subject may withdraw consent at any time without prejudice to future medical care.
  • Female subjects of childbearing potential must use a hormonal contraceptive, intra-uterine device, diaphragm with spermicide or condom with spermicide for the duration of the study. Confirmation that a female patient is not pregnant must be established by a negative human chorionic gonadotrophin (hCG) pregnancy test (urine or serum) within 7 days of study enrolment (SD1).

Exclusion Criteria:

To be eligible for inclusion in this study the subjects must not meet any of the following criteria:

  • Known allergy or hypersensitivity to growth hormone or diluent.
  • Previous treatment with GH within six months prior to Screen.
  • Severe illness during the previous six months.
  • Active malignancy (except non-melanomatous skin malignancies).
  • Diabetes mellitus (type I or II).
  • Seropositivity for human immunodeficiency virus (HIV), Hepatitis B surface antigen (HbsAg) and/or Hepatitis C Virus (HCV) serology.
  • Pregnancy or lactation.
  • History of drug and/or alcohol abuse or use of drugs for non-therapeutic purposes.
  • Any medical condition that, in the opinion of the Investigator, would jeopardize the patient's safety following exposure to study drug.
  • Clinically significant abnormal hematology, chemistry or urinalysis results at screening in the judgment of the Investigator.
  • Have taken another investigational drug or had any experimental procedure in the six months preceding study entry.
  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: NCT00109733

Locations
United States, California
Children's Hospital of Orange County
Orange, California, United States, 92868
United States, Florida
Nemours Children's Clinic
Jacksonville, Florida, United States, 32226
Nemours Children's Clinic
Orlando, Florida, United States, 32806
Pediatric Endocrinology Children's Clinic
Tallahassee, Florida, United States, 32308
United States, Georgia
Pediatric Endocrine Associates
Atlanta, Georgia, United States, 30342
United States, New York
Women's and Children's Hospital of Buffalo
Buffalo, New York, United States, 14222
Columbia University
New York, New York, United States, 10032
United States, Pennsylvania
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
EMD Serono
Investigators
Study Director: Sanja Dragnic, MD EMD Serono
  More Information

Additional Information:
No publications provided

Responsible Party: EMD Serono
ClinicalTrials.gov Identifier: NCT00109733     History of Changes
Other Study ID Numbers: 25253
Study First Received: May 2, 2005
Results First Received: May 19, 2010
Last Updated: August 4, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by EMD Serono:
Childhood-onset growth hormone deficiency (COGHD)
Saizen®

Additional relevant MeSH terms:
Dwarfism, Pituitary
Endocrine System Diseases
Bone Diseases
Bone Diseases, Developmental
Bone Diseases, Endocrine
Brain Diseases
Central Nervous System Diseases
Dwarfism
Hypopituitarism
Hypothalamic Diseases
Musculoskeletal Diseases
Nervous System Diseases
Pituitary Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014