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Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

NCT03876301 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 25

Last updated 2023-07-10

No results posted yet for this study

Summary

The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.

Conditions

  • Blood Coagulation Disorder
  • Blood Coagulation Disorders, Inherited
  • Coagulation Protein Disorders
  • Hemophilia A
  • Genetic Diseases, Inborn
  • Genetic Diseases, X-Linked
  • Hematologic Diseases
  • Hemorrhagic Disorders
  • Factor VIII Deficiency

Interventions

DRUG

Standard of Care FVIII Replacement therapy

There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Sponsors & Collaborators

  • Spark Therapeutics, Inc.

    lead INDUSTRY

Eligibility

Min Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-21
Primary Completion
2023-05-02
Completion
2023-05-02
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Thailand

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03876301 on ClinicalTrials.gov