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ClinicalTrials.gov public records Last synced Oct 8, 2026, 10:09 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–15 of 15 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Hemophilia B, Moderately Severe or Severe Clear all

Conditions: Hemophilia B
Interventions: CSL222 (AAV5-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2032
U.S. locations: 4
States / cities: San Diego, California • Ann Arbor, Michigan • Pittsburgh, Pennsylvania + 1 more

Conditions: Hemophilia A, Hemophilia B
Interventions: ALN-AT3SC, Sterile Normal Saline (0.9% NaCl); Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 65 Years · Male only
Enrollment: 51 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2017
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hemophilia B
Interventions: AAV5-hFIXco-Padua, Factor IX (FIX); Genetic · Biological
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 19
States / cities: Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more

Conditions: Hemophilia A
Interventions: SPK-8011; Genetic
Lead sponsor: Spark Therapeutics, Inc.; Industry
Eligibility: 18 Years and older · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2035
U.S. locations: 27
States / cities: Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more

Conditions: Hemophilia B
Interventions: AAV5-hFIXco-Padua (AMT-061); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2023
U.S. locations: 4
States / cities: Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more

Conditions: Hemophilia a, Hemophilia B
Interventions: SerpinPC; Drug
Lead sponsor: ApcinteX Ltd; Industry
Eligibility: 12 Years to 65 Years · Male only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 10
States / cities: Aurora, Colorado • Tampa, Florida • Indianapolis, Indiana + 7 more

Conditions: Hemophilia A, Hemophilia B
Interventions: PF-06741086; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 1 Year to 74 Years · Male only
Enrollment: 245 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2030
U.S. locations: 4
States / cities: Iowa City, Iowa • New Hyde Park, New York • Philadelphia, Pennsylvania + 1 more

Conditions: Hemophilia B
Interventions: CSL222 (Adeno-associated viral vector serotype 5 [AAV5]-hFIXco-Padua); Genetic
Lead sponsor: CSL Behring; Industry
Eligibility: 138 Months to 206 Months · Male only
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2033
U.S. locations: 5
States / cities: Orange, California • Gainesville, Florida • Atlanta, Georgia + 2 more

Conditions: Hemophilia A, Hemophilia B
Interventions: Fitusiran (SAR439774); Drug
Lead sponsor: Genzyme, a Sanofi Company; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 34 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2023
U.S. locations: 2
States / cities: Ann Arbor, Michigan • Pittsburgh, Pennsylvania

Conditions: Hemophilia A, Hemophilia B
Interventions: Not listed
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 1 Month and older · Female only
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2010
U.S. locations: 18
States / cities: Duarte, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more

Conditions: Hemophilia B, Hemophilia B, Moderately Severe or Severe
Interventions: BE-101; Drug
Lead sponsor: Be Biopharma; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 5
States / cities: Davis, California • Washington D.C., District of Columbia • Ann Arbor, Michigan + 2 more

Conditions: Hemophilia A, Hemophilia B
Interventions: marstacimab; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 1 Year to 17 Years · Male only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2028
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Hemophilia A, Hemophilia B
Interventions: Not listed
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: Female only
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 1
States / cities: New York, New York

Conditions: Hemophilia B, Hemophilia A
Interventions: Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 18 Years to 64 Years · Male only
Enrollment: 212 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 13
States / cities: Palo Alto, California • San Francisco, California • Stanford, California + 7 more

Conditions: Hemophilia A, Hemophilia B
Interventions: PF-06741086; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years to 74 Years · Male only
Enrollment: 188 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 4
States / cities: Tampa, Florida • Iowa City, Iowa • New Hyde Park, New York + 1 more

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Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy in Adults With Hemophilia B With Pretreatment Adeno-associated Virus Serotype 5 (AAV5) Neutralizing Antibodies (Nabs)

A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

Study of a Gene Therapy Treatment for Hemophilia A

Dose Confirmation Trial of AAV5-hFIXco-Padua

The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B

Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B