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

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Primary Purpose

Status

Completed

Phase

Phase 3

Locations

United States

Study Type

Interventional

Intervention

recombinant human growth hormone

About this trial

This is an interventional treatment trial for Childhood-onset Growth Hormone Deficiency focused on measuring Childhood-onset growth hormone deficiency (COGHD), Saizen®

Eligibility Criteria

13 Years - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Standard dose group

High dose group

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Percent Change From Baseline to Week 24 in Trunk Fat

Secondary Outcome Measures

Percent Change From Baseline to Week 24 in Lean Body Mass

Percent Change From Baseline to Week 24 in Total Body Fat

Percent Change From Baseline to Week 24 in Limb Fat

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

Full Information

NCT ID

NCT00109733

First Posted

May 2, 2005

Last Updated

August 4, 2013

Sponsor

EMD Serono

1. Study Identification

Unique Protocol Identification Number

NCT00109733

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

August 2013

Overall Recruitment Status

Completed

Study Start Date

January 2005 (undefined)

Primary Completion Date

June 2006 (Actual)

Study Completion Date

July 2006 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

EMD Serono

4. Oversight

5. Study Description

Brief 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Childhood-onset Growth Hormone Deficiency, Pituitary Dwarfism

Keywords

Childhood-onset growth hormone deficiency (COGHD), Saizen®

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

31 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Standard dose group

Arm Type

Experimental

Arm Description

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.

Arm Title

High dose group

Arm Type

Experimental

Arm Description

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.

Intervention Type

Biological

Intervention Name(s)

recombinant human growth hormone

Other Intervention Name(s)

Saizen, r-hGH, cool click

Intervention Description

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.

Intervention Type

Biological

Intervention Name(s)

recombinant human growth hormone

Other Intervention Name(s)

Saizen, r-hGH, cool click

Intervention Description

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.

Primary Outcome Measure Information:

Title

Percent Change From Baseline to Week 24 in Trunk Fat

Time Frame

Baseline to Week 24

Secondary Outcome Measure Information:

Title

Percent Change From Baseline to Week 24 in Lean Body Mass

Time Frame

Baseline to Week 24

Title

Percent Change From Baseline to Week 24 in Total Body Fat

Time Frame

Baseline to Week 24

Title

Percent Change From Baseline to Week 24 in Limb Fat

Time Frame

Baseline to Week 24

Title

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

Time Frame

Baseline to Week 24

10. Eligibility

Sex

All

Minimum Age & Unit of Time

13 Years

Maximum Age & Unit of Time

25 Years

Accepts Healthy Volunteers

No

Eligibility 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Sanja Dragnic, MD

Organizational Affiliation

EMD Serono

Official's Role

Study Director

Facility Information:

Facility Name

Children's Hospital of Orange County

City

Orange

State/Province

California

ZIP/Postal Code

92868

Country

United States

Facility Name

Nemours Children's Clinic

City

Jacksonville

State/Province

Florida

ZIP/Postal Code

32226

Country

United States

Facility Name

Nemours Children's Clinic

City

Orlando

State/Province

Florida

ZIP/Postal Code

32806

Country

United States

Facility Name

Pediatric Endocrinology Children's Clinic

City

Tallahassee

State/Province

Florida

ZIP/Postal Code

32308

Country

United States

Facility Name

Pediatric Endocrine Associates

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30342

Country

United States

Facility Name

Women's and Children's Hospital of Buffalo

City

Buffalo

State/Province

New York

ZIP/Postal Code

14222

Country

United States

Facility Name

Columbia University

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

Children's Hospital of Pittsburgh

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15213

Country

United States

Facility Name

Children's Hospital of Wisconsin

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

12. IPD Sharing Statement

Links:

URL

http://www.saizenus.com

Description

Full FDA approved prescribing information can be found here

Childhood-onset Growth Hormone Deficiency, Pituitary Dwarfism

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial