HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Completed · Phase 3 · NCT03569891
Conditions: Hemophilia B
At a glance
- Research payments reported, 2025
- $220,438
- U.S. investigators named
- 14
- Sites paid
- 13
- Planned or actual enrollment
- 67
About the trial
- Sponsor
- CSL Behring
- Tested
- AAV5-hFIXco-Padua (genetic), Factor IX (FIX) (biological)
- Dates
- Started 2018-06-27; completion 2025-03-19
- Results posted
- Yes
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 10\^13 gc/kg.
From ClinicalTrials.gov, last updated 2026-03-27. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Csl Behring reported $220,438 in research payments for this study in 2025 (65 payments).
Products named: Hemgenix
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Shelley E. Crary, MD Pediatric Hematology & Oncology · Little Rock, AR
- Shanna L. White, M.D. Pediatric Hematology & Oncology · Phoenix, AZ
- Annette Von Drygalski, MD Hematology & Oncology · San Diego, CA
- Guy A. Young, M.D. Pediatric Hematology & Oncology · Los Angeles, CA
- Tyler W. Buckner, M.D. Hematology · Aurora, CO
- Bradley S. Fletcher, MD Hematology · Tampa, FL
- Nathan I. Visweshwar, M.D. Hematology & Oncology · Tampa, FL
- Steven W. Pipe, MD Pediatric Hematology & Oncology · Ann Arbor, MI
- Nigel S. Key, MD Hematology & Oncology · Chapel Hill, NC
- Kristina M. Haley, DO Pediatric Hematology & Oncology · Portland, OR
- Shannon C. Walker, M.D. Pathology · Nashville, TN
- Miguel A. Escobar, M.D. Hematology · Houston, TX
- Richard S. Lemons Pediatric Hematology & Oncology · Salt Lake City, UT
- Barbara A. Konkle, MD Hematology · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of Utah Hospital and Clinics, UT $36,309
- University of Michigan Health System, MI $31,911
- Regents of the University of Colorado dba Exsera Biolabs $27,437
- University of North Carolina at Chapel Hill $21,682
- Ohsu Hospital and Clinics, OR $20,702
- Washington Institute for Coagulation $18,489
- Vanderbilt University Medical Center, TN $17,910
- Regents of the University of California San Diego $17,482
- University of South Florida $13,799
- Phoenix Children's Hospital, AZ $5,400
- Children's Hosp of Los Angeles, CA $5,395
- The University of Texas Health Science Center at Houston $3,612
- Arkansas Children's Research Institute $310
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.