Cool.Clickâ¢ Adolescent Transition Study: Study of SaizenÂ® in Subjects With Childhood-Onset Growth Hormone Deficiency
Information source: EMD Serono
Information obtained from ClinicalTrials.gov on June 20, 2008
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Childhood-Onset Growth Hormone Deficiency; Pituitary Dwarfism
Intervention: SaizenÂ® (Drug)
Phase: Phase 3
Sponsored by: EMD Serono
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.
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)
Study design: Treatment, Randomized, Open Label, Dose Comparison, Single Group Assignment, Efficacy Study
Primary outcome: Increase in percent of trunk fat in COGHD
Secondary outcome: Changes in lean body mass and body composition parameters
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 21 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. clickâ¢, 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 Investigatorâs 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
Investigatorâs approval, on day 15 to 0. 02 mg/kg/day and day 30 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, IGF-I, IGFBP-3, free T4 , total T4, 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.
Minimum age: 13 Years.
Maximum age: 21 Years.
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 21 years of age, inclusive
- Diagnosis of childhood onset GH deficiency and prior completed 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 SD1.
- 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) within the past 6 months.
- 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 study entry.
- 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
subjectâs 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 hCG pregnancy test (urine or serum) within 7 days of study
To be eligible for inclusion in this study the subjects must not meet any of the following
- Known allergy or hypersensitivity to growth hormone or diluent.
- Previous treatment with GH within six months prior to study entry.
- 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
patientâs 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.
Locations and Contacts
Childrenâs Hospital of Orange County, Orange, California 92868, United States
Pediatric Endocrinology Childrenâs Clinic, Tallahassee, Florida 32308, United States
Nemours Childrenâs Clinic, Orlando, Florida 32806, United States
Nemours Childrenâs Clinic, Jacksonville, Florida 32226, United States
Pediatric Endocrine Associates, Atlanta, Georgia 30342, United States
Womenâs and Childrenâs Hospital of Buffalo, Buffalo, New York 14222, United States
Columbia University, New York, New York 10032, United States
Childrenâs Hospital of Pittsburgh, Pittsburgh, Pennsylvania 15213, United States
Childrenâs Hospital of Wisconsin, Milwaukee, Wisconsin 53226, United States
Full FDA approved prescribing information can be found here
Starting date: August 2004
Ending date: July 2006
Last updated: June 11, 2007