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A Study to Evaluate Nutropin AQ for the Treatment of Growth Restriction in Children With Cystic Fibrosis

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
Nutropin AQ [somatropin (DNA origin) injection]
Sponsored by
Genentech, Inc.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Growth restriction in children with cystic fibrosis

Eligibility Criteria

5 Years - 13 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Ability of parent or legal guardian to provide written informed consent and, if applicable, pediatric assent and compliance with study assessments for the full duration of the study Diagnosis of CF by sweat or genetic testing Between the ages of 5 and 12 years for girls and 5 and 13 years for boys Ability to perform pulmonary function tests in a reproducible manner, per American Thoracic Society guidelines for spirometry Height <= 10th percentile for age and sex Prepubertal, Tanner Stage 1 Bone age of the non-dominant hand and wrist obtained no more than 6 months prior to study entry (bone age must be <= 10 years for girls and <= 11 years for boys as read using the method of Greulich and Pyle) Adequate caloric intake (following the CFF guidelines is encouraged; caloric intake must be documented at screening using a 24-hour food diary) Normal thyroid function Exclusion Criteria: Prior or current rhGH use History of short stature due to GHD History within the 12 months prior to screening of glucose intolerance (impaired glucose tolerance) or CF-related diabetes (CFRD) as defined by at least one of the following: fasting serum glucose of >= 126 mg/dL on two or more occasions; fasting serum glucose of >= 126 mg/dL plus any casual (previously called random) glucose level >= 200 mg/dL; casual (previously called random) glucose of >= 200 mg/dL on two or more occasions; fasting serum glucose of <= 126 mg/dL but 2-hour post oral glucose load of 140-199 mg/dL (impaired glucose tolerance) on two or more occasions; if a subject meets the criteria for impaired glucose tolerance or CFRD in the screening glucose tolerance test, even if there is no history of impaired glucose tolerance, the subject will not be eligible for the study. Infection with Burkholderia cepacia Qualitative change in antibiotic treatment (e.g., for exacerbation of lung infection) within 14 days of study entry Hospitalization or treatment with systemic corticosteroids during the 30 days prior to study entry Inability to adhere to previously documented adequate nutrition Active neoplasia Participation in any other investigational study (including investigational drug studies) within 30 days of enrollment or during the study if in the treatment arm, except for participation in observational and questionnaire studies (untreated) Subjects who require, as part of the their medical care, scheduled elective hospitalizations for IV antibiotic therapy

Sites / Locations

    Outcomes

    Primary Outcome Measures

    To determine the safety and efficacy of Nutropin AQ in treating growth restriction and increasing lean body mass (LBM) in children with cystic fibrosis (CF) and growth restriction.

    Secondary Outcome Measures

    To evaluate the effects of Nutropin AQ treatment on pulmonary function, disease-related exacerbations, and exercise tolerance in children with CF and growth restriction.

    Full Information

    First Posted
    March 11, 2004
    Last Updated
    June 5, 2008
    Sponsor
    Genentech, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00079742
    Brief Title
    A Study to Evaluate Nutropin AQ for the Treatment of Growth Restriction in Children With Cystic Fibrosis
    Official Title
    A Phase II, Multicenter, Randomized, Controlled, Open-Label Study of the Safety and Efficacy of Nutropin AQ [Somatropin (DNA Origin) Injection] for the Treatment of Growth Restriction in Children With Cystic Fibrosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    June 2008
    Overall Recruitment Status
    Completed
    Study Start Date
    September 2003 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    May 2007 (Actual)

    3. Sponsor/Collaborators

    Name of the Sponsor
    Genentech, Inc.

    4. Oversight

    5. Study Description

    Brief Summary
    This is a Phase II, multicenter, randomized, controlled, open-label trial of the safety and efficacy of Nutropin AQ administered subcutaneously (SC) daily in prepubertal children with CF and growth restriction.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystic Fibrosis
    Keywords
    Growth restriction in children with cystic fibrosis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 2
    Enrollment
    68 (Actual)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Nutropin AQ [somatropin (DNA origin) injection]
    Primary Outcome Measure Information:
    Title
    To determine the safety and efficacy of Nutropin AQ in treating growth restriction and increasing lean body mass (LBM) in children with cystic fibrosis (CF) and growth restriction.
    Secondary Outcome Measure Information:
    Title
    To evaluate the effects of Nutropin AQ treatment on pulmonary function, disease-related exacerbations, and exercise tolerance in children with CF and growth restriction.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    5 Years
    Maximum Age & Unit of Time
    13 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Ability of parent or legal guardian to provide written informed consent and, if applicable, pediatric assent and compliance with study assessments for the full duration of the study Diagnosis of CF by sweat or genetic testing Between the ages of 5 and 12 years for girls and 5 and 13 years for boys Ability to perform pulmonary function tests in a reproducible manner, per American Thoracic Society guidelines for spirometry Height <= 10th percentile for age and sex Prepubertal, Tanner Stage 1 Bone age of the non-dominant hand and wrist obtained no more than 6 months prior to study entry (bone age must be <= 10 years for girls and <= 11 years for boys as read using the method of Greulich and Pyle) Adequate caloric intake (following the CFF guidelines is encouraged; caloric intake must be documented at screening using a 24-hour food diary) Normal thyroid function Exclusion Criteria: Prior or current rhGH use History of short stature due to GHD History within the 12 months prior to screening of glucose intolerance (impaired glucose tolerance) or CF-related diabetes (CFRD) as defined by at least one of the following: fasting serum glucose of >= 126 mg/dL on two or more occasions; fasting serum glucose of >= 126 mg/dL plus any casual (previously called random) glucose level >= 200 mg/dL; casual (previously called random) glucose of >= 200 mg/dL on two or more occasions; fasting serum glucose of <= 126 mg/dL but 2-hour post oral glucose load of 140-199 mg/dL (impaired glucose tolerance) on two or more occasions; if a subject meets the criteria for impaired glucose tolerance or CFRD in the screening glucose tolerance test, even if there is no history of impaired glucose tolerance, the subject will not be eligible for the study. Infection with Burkholderia cepacia Qualitative change in antibiotic treatment (e.g., for exacerbation of lung infection) within 14 days of study entry Hospitalization or treatment with systemic corticosteroids during the 30 days prior to study entry Inability to adhere to previously documented adequate nutrition Active neoplasia Participation in any other investigational study (including investigational drug studies) within 30 days of enrollment or during the study if in the treatment arm, except for participation in observational and questionnaire studies (untreated) Subjects who require, as part of the their medical care, scheduled elective hospitalizations for IV antibiotic therapy
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Barbara Lippe, M.D.
    Organizational Affiliation
    Genentech, Inc.
    Official's Role
    Study Director

    12. IPD Sharing Statement

    Learn more about this trial

    A Study to Evaluate Nutropin AQ for the Treatment of Growth Restriction in Children With Cystic Fibrosis

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