A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01650779
First received: July 24, 2012
Last updated: June 9, 2014
Last verified: June 2014

July 24, 2012
June 9, 2014
April 2012
March 2013   (final data collection date for primary outcome measure)
Percent Change From Baseline in Plasma Deacylated Globotriaosylceramide (Lyso-GL-3) at Month 2, 4 and 6 [ Time Frame: Baseline, Month 2, 4, 6 ] [ Designated as safety issue: No ]
Percent change from baseline = ([post-baseline value minus baseline value] divided by [baseline value]) multiplied by 100. For levels reported as below quantitative limit (BQL), the lower limit of quantitation (LLOQ) value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma lyso-GL-3 was 5.0 nanogram per milliliter (ng/mL). This study is exploratory because little is known about the dose-response of these biomarkers to enzyme replacement therapy (ERT) or about the clinical significance of the biomarkers.
  • Percent change from baseline at Months 2, 4, and 6 in plasma lyso-GL-3 (globotriaosylceramide) [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in plasma GL-3 [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in urine GL-3 [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
  • Percent change from baseline at Months 2, 4, and 6 in gastrointestinal (GI) symptoms (abdominal pain, abdominal distention, and bowel irregularities) [ Time Frame: Months 2, 4, 6 ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01650779 on ClinicalTrials.gov Archive Site
  • Percent Change From Baseline in Plasma Globotriaosylceramide (GL-3) at Month 2, 4 and 6 [ Time Frame: Baseline, Month 2, 4, 6 ] [ Designated as safety issue: No ]
    Percent change from baseline = ([post-baseline value minus baseline value] divided by [baseline value]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for plasma GL-3 was 2.0 microgram per milliliter (mcg/mL). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.
  • Percent Change From Baseline in Urine GL-3 at Month 2, 4, and 6 [ Time Frame: Baseline, Month 2, 4, 6 ] [ Designated as safety issue: No ]
    Percent change from baseline = ([post-baseline value minus baseline value] divided by [baseline value]) multiplied by 100. For levels reported as BQL, the LLOQ value was divided by 2 and used in the calculation to estimate values in samples that were BQL. The LLOQ for urine GL-3 was 0.2 mcg/mL. The absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing GL-3 (mcg/mL) by creatinine (mg/mL) and multiplying by 113.13 (mg/mmol), the molecular weight of creatinine. For levels reported BQL, the absolute values were calculated in microgram per millimole (mcg/mmol) of creatinine by dividing 0.1 (mcg/mL) by creatinine (mg/mL) and multiplying by 133.13 (mg/mmol). This study is exploratory because little is known about the dose-response of these biomarkers to ERT or about the clinical significance of the biomarkers.
  • Percent Change From Baseline in Gastrointestinal (GI) Symptoms (Abdominal Pain, Abdominal Distention, and Bowel Irregularities) at Month 2, 4, and 6 [ Time Frame: Baseline, Month 2, 4, 6 ] [ Designated as safety issue: No ]
    Gastrointestinal symptoms (abdominal pain, abdominal distention, and irregular bowel movements) were to be assessed by a modified version of the Irritable Bowel Syndrome (IBS) Severity Scoring System. The modified IBS Severity Scoring System is a 7-item questionnaire. The severity score calculated by summing the scores of 5 of the 7 questions. Each of the 5 questions were scored on a scale of 0 to 100, leading to a total possible score range of 0 to 500, where higher scores indicate more severe gastrointestinal symptoms. The data for this outcome measure was exploratory and to be collected in individual participant listing only.
Not Provided
Not Provided
Not Provided
 
A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta
Evaluation of Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta (The INFORM Study)

This is an exploratory study to evaluate changes in glycosphingolipid levels and other (exploratory) Fabry disease parameters in male Fabry disease participants who were previously treated with agalsidase alfa (Replagal®) 0.2 milligram per kilogram (mg/kg) every two weeks (q2w) and who are being switched to agalsidase beta (Fabrazyme®) 1.0 mg/kg q2w.

Not Provided
Interventional
Phase 4
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Biological: Agalsidase beta
Commercially available agalsidase beta 1.0 mg/kg administered as an intravenous infusion q2w up to Month 6.
Other Name: Fabrazyme®
Experimental: Agalsidase beta
Intervention: Biological: Agalsidase beta
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
15
March 2013
March 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • The participant and/or his parent/legal guardian is willing and able to provide signed informed consent, and the participant, if less than (<) 18 years of age, is willing to provide assent if deemed able to do so
  • Participant is male and has been treated with agalsidase alfa at 0.2 mg/kg q2w for the 12 months prior to switching to agalsidase beta
  • The participant has a confirmed diagnosis of Fabry disease by alfa-galactosidase A (alfa-GAL) activity and/or genotyping per local standards
  • The participant when switched to agalsidase beta receives the labeled dose, that is, 0.9 to 1.1 mg/kg (1 mg/kg) q2w, and must be willing to maintain the labeled dose for the duration of the study

Exclusion Criteria:

  • The participant is on dialysis or is post renal transplantation
  • The participant is in end-stage cardiac failure
  • The participant and/or his parent or legal guardian, in the opinion of the investigator, is unable to adhere to the requirements of the study
  • The participant has been switched from agalsidase alfa to agalsidase beta and does not have historical blood and urine samples
Male
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01650779
AGAL19412
No
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
June 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP