Summary

for males ages 18 years and up (full criteria)
at Sacramento, California and other locations
study started
estimated completion

Description

Summary

Long-term safety follow-up for subjects with Hemophilia B and previously treated in the C0371005 (formerly SPK-9001-101) protocol

Official Title

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

Details

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

Keywords

Hemophilia B gene therapy Hemophilia A SPK-9001

Eligibility

You can join if…

Open to males ages 18 years and up

  1. Evidence of a personally signed and dated informed consent document indicating that the subject has been informed of all pertinent aspects of the study.
  2. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
  3. 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.

You CAN'T join if...

  1. 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.
  2. 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.
  3. 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.
  4. 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.
  5. Subjects who will not consent for up to 5 years of safety follow-up.
  6. Subjects who are unable or unwilling to comply with the study visits and requirements.
  7. 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.

Locations

  • UC Davis CTSC Clinical Research Center accepting new patients
    Sacramento California 95817 United States
  • UC Davis Health accepting new patients
    Sacramento California 95817 United States
  • UC Davis Investigational Pharmacy accepting new patients
    Sacramento California 95817 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
Sign up for this study
ID
NCT03307980
Phase
Phase 2
Study Type
Interventional
Last Updated