RECRUITING

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)

Conditions

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2024-01-30

Study Completion:2028-10

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06003387

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Has congenital hemophilia B with known severe or moderately severe FIX deficiency (≤ 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis * Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results) * Has \> 150 previous exposure days to FIX replacement therapy * Has been on stable FIX prophylaxis for at least 2 months before Screening * Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator * Acceptance to barrier contraception protection for 1 year starting the day of CSL222 treatment * Able to provide informed consent after receipt of verbal and written information about the study * Investigator believes that the participant (or the participant's legally acceptable representative\[s\]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.	* History of FIX inhibitors or positive FIX inhibitor test at Screening or Visit L (lead-in period)-Final (based on central laboratory results) * Screening and Visit L-Final laboratory values that meet the definition of Severe Hepatic Impairment per Common Terminology Criteria for Adverse Events (CTCAE) (based on central laboratory results) * ALT \> 2 × the upper limit of normal (ULN) at Screening and Visit L-Final (based on central laboratory results) * Any condition other than hemophilia B resulting in an increased bleeding tendency * Any uncontrolled or untreated infection (human immunodeficiency virus \[HIV\], hepatitis C, etc.) or any other significant concurrent, uncontrolled medical condition evaluated by the investigator to interfere with adherence to the clinical study protocol or with the degree of tolerance to CSL222. * Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at Screening and Visit L-Final (based on central laboratory results) * Known history of allergy to corticosteroids or known medical condition that would require chronic administration of steroids. * Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients * Previous gene therapy treatment * Receipt of an experimental agent or device within 60 days before Screening until the end of the study. * Note: Other protocol pre-specified exclusion criteria may apply.

Inclusion Criteria

Exclusion Criteria

* Has congenital hemophilia B with known severe or moderately severe FIX deficiency (≤ 2% of normal circulating FIX) for which the participant is on continuous routine FIX prophylaxis
* Has 2 consecutive detectable AAV5 NAb titer results between Screening and Visit L-Final using a validated AAV5 NAb assay (based on central laboratory results)
* Has \> 150 previous exposure days to FIX replacement therapy
* Has been on stable FIX prophylaxis for at least 2 months before Screening
* Has demonstrated capability to independently, accurately, and in a timely manner complete the eDiary during the Lead-in Period, as judged by the investigator
* Acceptance to barrier contraception protection for 1 year starting the day of CSL222 treatment
* Able to provide informed consent after receipt of verbal and written information about the study
* Investigator believes that the participant (or the participant's legally acceptable representative\[s\]) understands the nature, scope, and possible consequences of the study and is able to adhere to the study procedures.

* History of FIX inhibitors or positive FIX inhibitor test at Screening or Visit L (lead-in period)-Final (based on central laboratory results)
* Screening and Visit L-Final laboratory values that meet the definition of Severe Hepatic Impairment per Common Terminology Criteria for Adverse Events (CTCAE) (based on central laboratory results)
* ALT \> 2 × the upper limit of normal (ULN) at Screening and Visit L-Final (based on central laboratory results)
* Any condition other than hemophilia B resulting in an increased bleeding tendency
* Any uncontrolled or untreated infection (human immunodeficiency virus \[HIV\], hepatitis C, etc.) or any other significant concurrent, uncontrolled medical condition evaluated by the investigator to interfere with adherence to the clinical study protocol or with the degree of tolerance to CSL222.
* Thrombocytopenia, defined as a platelet count below 50 × 10\^9/L, at Screening and Visit L-Final (based on central laboratory results)
* Known history of allergy to corticosteroids or known medical condition that would require chronic administration of steroids.
* Known uncontrolled allergic conditions or allergy / hypersensitivity to any component of the CSL222 excipients
* Previous gene therapy treatment
* Receipt of an experimental agent or device within 60 days before Screening until the end of the study.
* Note: Other protocol pre-specified exclusion criteria may apply.

Contacts and Locations

Study Contact

Trial Registration Coordinator

CONTACT

1-610-878-4000

clinicaltrials@cslbehring.com

Principal Investigator

Study Director

STUDY_DIRECTOR

CSL Behring LLC

Study Locations (Sites)

University of Michigan - 84000285

Ann Arbor, Michigan, 48109

United States

Collaborators and Investigators

Sponsor: CSL Behring

Study Director, STUDY_DIRECTOR, CSL Behring LLC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-01-30

Study Completion Date2028-10

Study Record Updates

Study Start Date2024-01-30

Study Completion Date2028-10

Terms related to this study

Additional Relevant MeSH Terms

Hemophilia B