Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B
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Long-term safety follow-up for subjects with Hemophilia B and previously treated in the C0371005 (formerly SPK-9001-101) protocol
Condition or disease
Evaluation of the long term level of persistence and potential late or delayed adverse events associated with SPK-9001, assessment of the durability of the transgene expression, and determination of the effects of SPK-9001 on clinical outcomes in individuals who have previously received a single administration of SPK-9001
This is a long term follow up (LTFU) study designed to evaluate the overall long term safety, durability of transgene expression, and effect on clinical outcomes of SPK 9001 mediated gene transfer. While safety will be monitored in general, new onset of oncologic, hematologic, neurologic, or auto immune events will be of particular interest. This trial will last for 5 years providing a minimum of 6 years of follow up post vector administration.
None (Open Label)
FIX LONG STUDY: A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF SPK 9001 (RAAV SPARK100 HFIX PADUA) IN INDIVIDUALS WITH HEMOPHILIA B
Actual Study Start Date :
June 22, 2017
Estimated Primary Completion Date :
May 16, 2026
Estimated Study Completion Date :
May 16, 2026
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Gender Based Eligibility:
Gender Eligibility Description:
Accepts Healthy Volunteers:
Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
Subjects who met the C0371005 eligibility criteria, have received a single administration of SPK-9001, and completed the required assessments in the C0371005 clinical study.
Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or subjects who are Pfizer employees, including their family members, directly involved in the conduct of the study.
Participation in other studies involving investigational drug(s) within the last 12 weeks prior to study entry, excluding participation in C0371005, and/or during study participation.
Other acute or chronic medical or psychiatric condition including recent (within the past year) or active suicidal ideation or behavior or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.
Fertile male subjects who are unwilling or unable to use a condom as outlined in this protocol until 3 consecutive semen samples are negative in the vector shedding analysis after the last dose of investigational product.
Subjects who will not consent for up to 5 years of safety follow-up.
Subjects who are unable or unwilling to comply with the study visits and requirements.
Subjects with any clinically significant medical condition that the Investigator believes would pose as a safety risk or are not capable of performing the endpoints of this study.
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD:
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.