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

NCT00109733 | Completed Phase 3 Results

• 1 other identifier: 25253
• Study Type: interventional
• Target: 31
• Locations: 1 country
• Sites: 9

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.

Conditions

Childhood-onset Growth Hormone Deficiency; Pituitary Dwarfism

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline to Week 24 in Trunk Fat

  Baseline to Week 24

Secondary Outcomes (4)

• Percent Change From Baseline to Week 24 in Lean Body Mass

  Baseline to Week 24

• Percent Change From Baseline to Week 24 in Total Body Fat

  Baseline to Week 24

• Percent Change From Baseline to Week 24 in Limb Fat

  Baseline to Week 24

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

  Baseline to Week 24

Study Arms (2)

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.

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.

Biological: recombinant human growth hormone

Interventions

recombinant human growth hormone BIOLOGICAL

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

Standard dose group

Eligibility Criteria

• Age: 13 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• EMD Serono: lead

Study Sites (9)

Children's Hospital of Orange County

Orange, California, 92868, United States

Location

Nemours Children's Clinic

Jacksonville, Florida, 32226, United States

Location

Nemours Children's Clinic

Orlando, Florida, 32806, United States

Location

Pediatric Endocrinology Children's Clinic

Tallahassee, Florida, 32308, United States

Location

Pediatric Endocrine Associates

Atlanta, Georgia, 30342, United States

Location

Women's and Children's Hospital of Buffalo

Buffalo, New York, 14222, United States

Location

Columbia University

New York, New York, 10032, United States

Location

Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

Location

Children's Hospital of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Links

• Full FDA approved prescribing information can be found here

MeSH Terms

Conditions

Dwarfism, Pituitary

Interventions

Growth Hormone; Human Growth Hormone

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Hypopituitarism; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Sanja Dragnic/Medical Responsible
• Organization: EMD Serono

sanja.dragnic@emdserono.com

+7816814017

Study Officials

• Sanja Dragnic, MD

  EMD Serono

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 2, 2005
• First Posted: May 3, 2005
• Study Start: January 1, 2005
• Primary Completion: June 1, 2006
• Study Completion: July 1, 2006
• Last Updated: August 7, 2013
• Results First Posted: June 22, 2010

Record last verified: 2013-08

Locations

US (9)