Childhood-onset Growth Hormone DeficiencyPhase 3NCT00109733
Cool.Click™ Adolescent Transition Study: Study of Saizen® in Subjects With Childhood-onset Growth Hormone Deficiency
A Phase 3 study testing recombinant human growth hormone in people with Childhood-onset Growth Hormone Deficiency and Pituitary Dwarfism, run by EMD Serono at 9 sites in United States.
Trial 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 more about this study
- Who is it for?
- Ages 13 to 25 years
- Conditions the study is for
- Childhood-onset Growth Hormone Deficiency, Pituitary Dwarfism
- What is being tested?
- recombinant human growth hormone
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 9 sitesUnited States
- Study dates
- Jan 2005to Jul 2006
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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).
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- 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.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Pediatric Endocrine Associates
Atlanta, Georgia, 30342
Women's and Children's Hospital of Buffalo
Buffalo, New York, 14222
Nemours Children's Clinic
Jacksonville, Florida, 32226
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Columbia University
New York, New York, 10032
Children's Hospital of Orange County
Orange, California, 92868
Nemours Children's Clinic
Orlando, Florida, 32806
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15213
Pediatric Endocrinology Children's Clinic
Tallahassee, Florida, 32308
About this study
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)
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.
- Being tested
- recombinant human growth hormone
- Keywords
- Childhood-onset growth hormone deficiency (COGHD), Saizen®
- Medical subject headings
- Dwarfism, Pituitary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
recombinant human growth hormoneBiological
Also called Saizen, r-hGH, cool click
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.
recombinant human growth hormoneBiological
Also called Saizen, r-hGH, cool click
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.
| Study group | Role | What they receive |
|---|---|---|
| Standard dose group | Experimental | 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. Receives: Biological: recombinant human growth hormone |
| High dose group | Experimental | 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. Receives: Biological: recombinant human growth hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent Change From Baseline to Week 24 in Trunk Fat
Measured: Baseline to Week 24
Secondary outcomes (4)
Percent Change From Baseline to Week 24 in Lean Body Mass
Measured: Baseline to Week 24
Percent Change From Baseline to Week 24 in Total Body Fat
Measured: Baseline to Week 24
Percent Change From Baseline to Week 24 in Limb Fat
Measured: Baseline to Week 24
Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio
Measured: Baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2005 (estimated)
- Study startJan 2005
- Primary completionJun 2006
- Study completionJul 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Childhood-onset Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- EMD Serono
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sanja Dragnic, MD, Study director, EMD Serono
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00109733
- Sponsor's protocol ID
- 25253
- Status last verified
- Aug 2013
- First submitted
- 2 May 2005
- Last updated
- 7 Aug 2013
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT00109733
- Acronym / sponsor ID
- 25253
- Keywords
- Childhood-onset growth hormone deficiency (COGHD), Saizen®
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- EMD SeronoIndustry (pharma, biotech, devices)
- Study start
- Jan 2005
- Primary completion
- Jun 2006 (actual)
- Study completion
- Jul 2006 (actual)
- First posted
- 3 May 2005 (estimated)
- Results
- Posted 22 Jun 2010
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard dose group | Experimental | Biological: recombinant human growth hormone0.005 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. |
| High dose group | Experimental | Biological: recombinant human growth hormone0.010 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. |
Who may qualify
- Ages
- 13 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (12 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| recombinant human growth hormoneAlso: Saizen, r-hGH, cool click0.005 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. | Biological | Standard dose group |
| recombinant human growth hormoneAlso: Saizen, r-hGH, cool click0.010 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. | Biological | High dose group |
Primary outcome
Percent Change From Baseline to Week 24 in Trunk Fat
Time frame: Baseline to Week 24
4 secondary outcomes
Percent Change From Baseline to Week 24 in Lean Body Mass
Time frame: Baseline to Week 24
Percent Change From Baseline to Week 24 in Total Body Fat
Time frame: Baseline to Week 24
Percent Change From Baseline to Week 24 in Limb Fat
Time frame: Baseline to Week 24
Percent Change From Baseline to Week 24 in Trunk to Limb Fat Ratio
Time frame: Baseline to Week 24
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Full FDA approved prescribing information can be found hereLink provided by the sponsor.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sanja Dragnic, MD study director
EMD Serono
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing recombinant human growth hormone in people with Childhood-onset Growth Hormone Deficiency and Pituitary Dwarfism, run by EMD Serono at 9 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 Aug 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Jun 2010). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percent Change From Baseline to Week 24 in Trunk Fat (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Aug 2013.