Clinical Trial: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Long-Term Study to Assess the Ongoing Safety and Efficacy of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency

Brief Summary:

The primary objective of this study is to obtain data regarding the safety of olipudase alfa in patients with acid sphingomyelinase deficiency (ASMD) who are exposed to long term treatment with olipudase alfa.

The secondary objectives of this study are to obtain data regarding the efficacy of olipudase alfa and to characterize olipudase alfa pharmacodynamics (PD) and pharmacokinetics (PK) following long-term administration.


Detailed Summary: 5 years: This study is an extension study for patients who have completed a previous study with olipudase alfa (DFI13803 for pediatric patients, and DFI13412 for adult patients).
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome:

  • Adverse events and treatment emergent adverse events including infusion-associated reactions [ Time Frame: Baseline to 5 years ]
    Number of patients experiencing Adverse Events
  • Physical examinations including neurologic examinations [ Time Frame: Baseline to 5 years ]
  • Clinical laboratory tests [ Time Frame: Baseline to 5 years ]
  • Safety biomarkers [ Time Frame: Baseline to 5 years ]
  • Immune response assessments [ Time Frame: Baseline to 5 years ]
  • Vital signs, echocardiogram and electrocardiogram [ Time Frame: Baseline to 5 years ]
  • Liver biopsy [ Time Frame: Baseline to 5 years ]
    Patients previously enrolled in DFI13412
  • Liver ultrasound/Doppler [ Time Frame: Baseline to 5 years ]
    Patients previously enrolled in DFI13803


Original Primary Outcome:

  • All Adverse events and Serious Adverse Events [ Time Frame: Baseline to Week 272 ]
    Number of patients experience AE
  • Physical examinations [ Time Frame: Baseline to Week 272 ]
  • Clinical laboratory tests [ Time Frame: Baseline to Week 272 ]
  • Safety biomarkers [ Time Frame: Baseline to Week 272 ]
  • Immune response assessments [ Time Frame: Baseline to Week 272 ]
  • Vital signs, echocardiogram and electrocardiogram [ Time Frame: Baseline to Week 272 ]
  • Continuous heart rate monitoring by telemetry (in-patients visits) [ Time Frame: Baseline to Week 272 ]


Current Secondary Outcome:

  • Spleen and Liver Volume [ Time Frame: Baseline to 5 years ]
    Abdominal magnetic resonance imaging (MRI) to evaluate improvements in spleen and liver volume
  • Pulmonary imaging tests [ Time Frame: Baseline to 5 years ]
  • Pulmonary function tests [ Time Frame: Baseline to 5 years ]
  • Hematology [ Time Frame: Baseline to 5 years ]
    Hemoglobin and platelet count
  • Lipid profile [ Time Frame: Baseline to 5 years ]
  • Health Outcomes Questionnaires [ Time Frame: Baseline to 5 years ]
  • Hand X-ray for bone age and bone maturation [ Time Frame: Baseline to 5 years ]
    Pediatric patients
  • Tanner staging [ Time Frame: Baseline to 5 years ]
    Pediatric patients
  • Linear patient growth by height Z-score [ Time Frame: Baseline to 5 years ]
    Pediatric patients


Original Secondary Outcome:

  • Spleen and Liver Volume [ Time Frame: Baseline to Week 272 ]
  • Pulmonary imaging and function tests [ Time Frame: Baseline to Week 272 ]
  • Exercise capacity by cycle ergometry [ Time Frame: Baseline to Week 272 ]
  • Hematology [ Time Frame: Baseline to Week 272 ]


Information By: Sanofi

Dates:
Date Received: November 26, 2013
Date Started: December 2013
Date Completion: October 2021
Last Updated: March 3, 2017
Last Verified: March 2017