Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B

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NCT03307980

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Study Location
Weill Cornell Medicine - New York Presbyterian Hospital
New York, New York, 10065, United States
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Contact
1-800-718-1021
[email protected]
Related Links

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting the study website. Please see the references below:

By phone

Pfizer Clinical Trials Contact Center

1-800-718-1021

By email

Contact

[email protected]

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Hemophilia B
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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.

Exclusion Criteria
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


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.

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Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

Pfizer Clinical Trials Contact Center

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[email protected]

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Advanced Information
Descriptive Information
Brief Title  ICMJE Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B
Official Title  ICMJE 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
Brief Summary Long-term safety follow-up for subjects with Hemophilia B and previously treated in the C0371005 (formerly SPK-9001-101) protocol
Detailed Description 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
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:
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.
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia B
Intervention  ICMJE Biological: SPK-9001
Observational long-term safety follow-up study of subjects previously treated in the C0371005 (formerly SPK-9001-101) protocol
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 9, 2018)		20
Original Estimated Enrollment  ICMJE
 (submitted: October 6, 2017)		11
Estimated Study Completion Date  ICMJE October 6, 2026
Estimated Primary Completion Date October 6, 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  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.

Exclusion Criteria:

  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.
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Gender Eligibility Description:Genetic males
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Australia,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03307980
Other Study ID Numbers  ICMJE C0371003
SPK-9001-LTFU-101 ( Other Identifier: Alias Study Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description: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/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date December 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP