RecruitingPhase 3NCT06003387

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)

Phase 3b, Open-label, Multicenter, Single-dose Study Investigating Efficacy and Safety of CSL222 (Etranacogene Dezaparvovec) Gene Therapy Administered to Adult Subjects With Severe or Moderately Severe Hemophilia B With Detectable Pretreatment AAV5 Neutralizing Antibodies

Sponsor

CSL Behring

Enrollment

35 participants

Start Date

Jan 30, 2024

Study Type

INTERVENTIONAL

Conditions

Hemophilia B

Summary

The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying CSL222 (AAV5-hFIXco-Padua) for people with hemophilia b. The study is currently recruiting participants at 30 locations. People eligible for this study include aged 18 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

GENETICCSL222 (AAV5-hFIXco-Padua)

Administered as a single IV infusion.

Locations(32)

University of California, San Diego (UCSD)

San Diego, California, United States

University of Michigan

Ann Arbor, Michigan, United States

Hemophilia Center of Western Pennsylvania (HCWP)

Pittsburgh, Pennsylvania, United States

The University of Texas Health Science Center at Houston

Houston, Texas, United States

Royal Prince Alfred Hospital

Camperdown, New South Wales, Australia

Royal Brisbane Hospital

Herston, Queensland, Australia

Royal Adelaide Hospital

Adelaide, South Australia, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo

São Paulo, Brazil

UNICAMP Universidade Estadual de Campinas

São Paulo, Brazil

Specialized Hospital for Active Treatment of Hematological Diseases

Sofia, Bulgaria

McMaster University - Hamilton

Hamilton, Ontario, Canada

Queen Mary Hospital

Hong Kong, Hong Kong

Prince of Wales Hospital Chinese University of Hong Kong

Shatin, Hong Kong

Sheba Medical Center

Tel Litwinsky, Israel

Centro de Investigacion Clinica GRAMEL S.C.

Mexico City, Mexico City, Mexico

Klinika Zaburzen Hemostazy i Chorob Wewnetrznych

Warsaw, Poland

King Faisal Specialist Hospital and Research Center

Riyadh, Saudi Arabia

National University Hospital

Singapore, Singapore

Singapore General Hospital

Singapore, Singapore

Haemophilia Comprehensive Care Centre

Johannesburg, South Africa

Kyungpook National University Hospital

Daegu, South Korea

Kyung Hee University Hospital at Gangdong

Seoul, South Korea

Severance Hospital, Yonsei University Health System

Seoul, South Korea

Tri-Service General Hospital

Taipei, Neihu District, Taiwan

Kaohsiung Medical University Chung-Ho Memorial Hospital (KMUH)

Kaohsiung City, Sanmin District, Taiwan

Changhua Christian Hospital (CCH)

Chang-hua, Taiwan

Taichung Veterans General Hospital -

Taichung, Taiwan

National Taiwan University Hospital

Taipei, Taiwan

Ege University Medical Faculty

Bornova, Turkey (Türkiye)

Gaziantep University Sahinbey Research and Practice Hospital

Gaziantep, Turkey (Türkiye)

Özel Acibadem Adana Hastanesi

Seyhan, Turkey (Türkiye)

View Full Details on ClinicalTrials.gov

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NCT06003387

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