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Treatment for Hemophilia (HOT Trial)

N/A
Waitlist Available
Led By Margaret Ragni, MD, MPH
Research Sponsored by American Thrombosis and Hemostasis Network
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1year
Awards & highlights

Summary

This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.

Eligible Conditions
  • Hemophilia
  • Liver Transplant

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Quality of Life (HOT)
Quality of life (Haem-A-QoL)
Quality of life (PROMIS-29)
Secondary study objectives
Blood Coagulation Factor

Find a Location

Who is running the clinical trial?

BioMarin PharmaceuticalIndustry Sponsor
158 Previous Clinical Trials
189,916 Total Patients Enrolled
1 Trials studying Hemophilia
165 Patients Enrolled for Hemophilia
American Thrombosis and Hemostasis NetworkLead Sponsor
9 Previous Clinical Trials
4,407 Total Patients Enrolled
3 Trials studying Hemophilia
3,547 Patients Enrolled for Hemophilia
Margaret Ragni, MD, MPHPrincipal InvestigatorUniversity of Pittsburgh Medical Center
1 Previous Clinical Trials
7 Total Patients Enrolled
~16 spots leftby Sep 2025