ClinicalTrials.gg
Sign In
CompletedNCT00109733Updated Aug 7, 2013Results posted

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

A Phase 3 interventional study of recombinant human growth hormone and recombinant human growth hormone in Childhood-onset Growth Hormone Deficiency and Pituitary Dwarfism, sponsored by EMD Serono. Completed at 9 sites in United States. Open to participants aged 13 Years to 25 Years. Per ClinicalTrials.gov, last updated 2013-08-07.

Sponsored by EMD Serono (part of Merck KGaA, Darmstadt, Germany) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
31
Allocation
Randomized
Ages
13 Years to 25 Years
Sex
All
01

Study summary

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.

Read the 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.

02

Conditions studied

  • Childhood-onset Growth Hormone Deficiency
  • Pituitary Dwarfism

Browse trials for

Keywords

  • Childhood-onset growth hormone deficiency (COGHD)
  • Saizen®
03

In context

Dwarfism, Pituitary

171 studies on the registry are indexed under Dwarfism, Pituitary; 25 are open to participants now.

This study's enrollment of 31 is below the median of 69 across 127 interventional studies indexed under Dwarfism, Pituitary.

Browse Dwarfism, Pituitary studies →

Lead sponsor

EMD Serono is the lead sponsor of 91 studies on the registry; none are open to participants now.

First submitted before EMD Serono became part of Merck KGaA, Darmstadt, Germany.

Browse EMD Serono studies →All Merck KGaA, Darmstadt, Germany studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
13 Years to 25 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
31 participants (actual)

Study arms

  • 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

  • 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

Interventions

  • Biologicalrecombinant 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.

    Also known as: Saizen, r-hGH, cool click

  • Biologicalrecombinant 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.

    Also known as: Saizen, r-hGH, cool click

06

What researchers measure

Primary outcomes

  1. Percent Change From Baseline to Week 24 in Trunk Fat

    Time frame: Baseline to Week 24

Secondary outcomes

  1. Percent Change From Baseline to Week 24 in Lean Body Mass

    Time frame: Baseline to Week 24

  2. Percent Change From Baseline to Week 24 in Total Body Fat

    Time frame: Baseline to Week 24

  3. Percent Change From Baseline to Week 24 in Limb Fat

    Time frame: Baseline to Week 24

  4. Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio

    Time frame: Baseline to Week 24

07

Results

Posted Jun 22, 2010

Participant flow

Study Initiation Date: 06 Jan 2005 (date of first subject, first dose) Study Completion Date 11 Jun 2006 (date of last subject, last visit) 19 study centres in the United States received IRB approval to participate in this study; 13 of the centres enrolled at least one subject into the study.

Participant flow — Overall Study
MilestoneStandard Dose GroupHigh Dose Group
Started1516
Completed1312
Not completed24
Withdrew: Protocol violation01
Withdrew: Stopped taking study drug11
Withdrew: Non-compliant01
Withdrew: Could not commit time for study11

Outcome measures

PrimaryPercent Change From Baseline to Week 24 in Trunk Fat
Time frame:
Baseline to Week 24
Reported as:
Mean · percent change
Percent Change From Baseline to Week 24 in Trunk Fat
percent changeStandard Dose GroupHigh Dose Group
Percent Change From Baseline to Week 24 in Trunk Fat-6.27 ± 12.72-8.36 ± 17.69
Statistical analysis
  • Standard Dose Group · ANOVA · p = 0.177
  • High Dose Group · ANOVA · p = 0.004
SecondaryPercent Change From Baseline to Week 24 in Lean Body Mass
Time frame:
Baseline to Week 24
Reported as:
Mean · percent change
Percent Change From Baseline to Week 24 in Lean Body Mass
percent changeStandard Dose GroupHigh Dose Group
Percent Change From Baseline to Week 24 in Lean Body Mass3.85 ± 4.057.10 ± 5.02
Statistical analysis
  • Standard Dose Group · ANOVA · p = 0.004
  • High Dose Group · ANOVA · p = <0.001
SecondaryPercent Change From Baseline to Week 24 in Total Body Fat
Time frame:
Baseline to Week 24
Reported as:
Mean · percent change
Percent Change From Baseline to Week 24 in Total Body Fat
percent changeStandard Dose GroupHigh Dose Group
Percent Change From Baseline to Week 24 in Total Body Fat-4.62 ± 9.96-9.69 ± 10.54
Statistical analysis
  • Standard Dose Group · ANOVA · p = 0.653
  • High Dose Group · ANOVA · p = 0.002
SecondaryPercent Change From Baseline to Week 24 in Limb Fat
Time frame:
Baseline to Week 24
Reported as:
Mean · percent change
Percent Change From Baseline to Week 24 in Limb Fat
percent changeStandard Dose GroupHigh Dose Group
Percent Change From Baseline to Week 24 in Limb Fat-3.77 ± 8.85-11.09 ± 8.97
Statistical analysis
  • Standard Dose Group · ANOVA · p = 0.755
  • High Dose Group · ANOVA · p = <0.001
SecondaryPercent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio
Time frame:
Baseline to Week 24
Reported as:
Mean · percent change
Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio
percent changeStandard Dose GroupHigh Dose Group
Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio-2.86 ± 6.772.92 ± 16.16
Statistical analysis
  • Standard Dose Group · ANOVA · p = 0.041
  • High Dose Group · ANOVA · p = 0.044

Adverse events

Collected over Baseline to Week 28(+/-5 days). Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Standard Dose Group—0/15 (0%)13/15 (86.7%)
High Dose Group—1/16 (6.3%)14/16 (87.5%)
Most frequent serious events
Most frequent serious events
EventStandard Dose GroupHigh Dose Group
AsthmaRespiratory, thoracic and mediastinal disorders0/151/16
Most frequent other events
Showing 10 of 50
Most frequent other events
EventStandard Dose GroupHigh Dose Group
HeadacheNervous system disorders6/156/16
Upper respiratory tract infectionInfections and infestations4/153/16
ArthralgiaMusculoskeletal and connective tissue disorders3/150/16
NasopharyngitisInfections and infestations1/153/16
SinusitisInfections and infestations0/153/16
DysmenorrhoeaReproductive system and breast disorders2/151/16
Injection site bruisingGeneral disorders2/151/16
Back painMusculoskeletal and connective tissue disorders0/152/16
DiarrhoeaGastrointestinal disorders0/152/16
Abdominal pain upperGastrointestinal disorders1/151/16

Baseline characteristics

Age Continuous
Age Continuous(years)Standard Dose GroupHigh Dose GroupTotal
Mean17.4 ± 1.818.1 ± 1.917.8 ± 1.9
Sex: Female, Male
Sex: Female, Male(Participants)Standard Dose GroupHigh Dose GroupTotal
Female5611
Male101020
Region of Enrollment
Region of Enrollment(participants)Standard Dose GroupHigh Dose GroupTotal
United States151631
Lean body mass
Lean body mass(kg)Standard Dose GroupHigh Dose GroupTotal
Mean44.03 ± 11.0944.38 ± 12.9844.21 ± 11.90
Limb fat
Limb fat(kg)Standard Dose GroupHigh Dose GroupTotal
Mean11.47 ± 5.4912.92 ± 9.2812.22 ± 7.59
Total body fat
Total body fat(kg)Standard Dose GroupHigh Dose GroupTotal
Mean24.02 ± 10.8125.11 ± 18.2924.58 ± 14.90
Trunk fat
Trunk fat(kg)Standard Dose GroupHigh Dose GroupTotal
Mean11.73 ± 5.5811.40 ± 9.0911.56 ± 7.47
Trunk to limb fat ratio
Trunk to limb fat ratio(ratio)Standard Dose GroupHigh Dose GroupTotal
Mean1.05 ± 0.290.87 ± 0.170.96 ± 0.25
08

Study locations

9 sites
  • Children's Hospital of Orange County
    Orange, California 92868, United States
  • Nemours Children's Clinic
    Jacksonville, Florida 32226, United States
  • Nemours Children's Clinic
    Orlando, Florida 32806, United States
  • Pediatric Endocrinology Children's Clinic
    Tallahassee, Florida 32308, United States
  • Pediatric Endocrine Associates
    Atlanta, Georgia 30342, United States
  • Women's and Children's Hospital of Buffalo
    Buffalo, New York 14222, United States
  • Columbia University
    New York, New York 10032, United States
  • Children's Hospital of Pittsburgh
    Pittsburgh, Pennsylvania 15213, United States
  • Children's Hospital of Wisconsin
    Milwaukee, Wisconsin 53226, United States
09

References and documents

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 7, 2013, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT00109733
Lead sponsor
EMD Serono
Responsible party
Sponsor
First posted
May 3, 2005
Start date
Jan 2005
Primary completion
Jun 2006
Completion
Jul 2006
Results posted
Jun 22, 2010
Last update
Aug 7, 2013

Study contacts

Sanja Dragnic, MD
study director · EMD Serono
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2013. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Human Growth Hormone

Other studies in this condition

Dwarfism, Pituitary →

Other studies from this sponsor

EMD Serono →

Start the discussion