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Trial record 1 of 2 for:    SPK-9001
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Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B

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ClinicalTrials.gov Identifier: NCT03307980
Recruitment Status : Recruiting
First Posted : October 12, 2017
Last Update Posted : December 7, 2018
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
Long-term safety follow-up for subjects with Hemophilia B and previously treated in the SPK-9001-101 protocol

Condition or disease Intervention/treatment Phase
Hemophilia B Biological: SPK-9001 Phase 2

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 : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
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
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]
Actual Study Start Date : June 22, 2017
Estimated Primary Completion Date : March 30, 2024
Estimated Study Completion Date : March 30, 2024

Resource links provided by the National Library of Medicine



Intervention Details:
  • Biological: SPK-9001
    Observational long-term safety follow-up study of subjects previously treated in the SPK-9001-101 protocol


Primary Outcome Measures :
  1. SPK-9001-related adverse advents [ Time Frame: up to 5 years ]
    adverse events


Secondary Outcome Measures :
  1. Coagulation Clotting Assay for FIX activity levels [ Time Frame: up to 5 years ]
    Coagulation Clotting assays to assess FIX activity levels (percent of normal)

  2. Annualized (factor FIX) infusion rate [ Time Frame: up to 5 years ]
    AIR (not including those for surgery)

  3. Total factor consumption (IU and volume) [ Time Frame: up to 5 years ]
    total quantity of factor infused annually (not including those for surgery) as recorded on the infusion log

  4. Annualized bleeding rate [ Time Frame: up to 5 years ]
    ABR (not including those for surgery)

  5. Total number of bleeding events [ Time Frame: up to 5 years ]
    (spontaneous and traumatic)

  6. Haem-A-QoL [ Time Frame: up to 5 years ]
    Quality-of-life (QoL) assessment

  7. EQ-5D-5L [ Time Frame: up to 5 years ]
    Quality-of-life (QoL) assessment



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Genetic males
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Ability to understand the purpose and risks of the study, as well as provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local study participant privacy regulations.
  2. Subjects who have received a single administration of SPK-9001, and completed the required assessments in the SPK-9001-101 clinical study.

Exclusion Criteria:

  1. Subjects who will not consent for up to 5 years of safety follow-up.
  2. Subjects who are unable or unwilling to comply with the study visits and requirements.
  3. Subjects who have planned to participate in another interventional investigational gene transfer study.
  4. Subjects with any clinically significant medical condition that the Investigator believes would pose as a safety risk or are not capable of the performing endpoints of this study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03307980


Contacts
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

Locations
United States, California
UC Davis Midtown Clinic Recruiting
Sacramento, California, United States, 95816
UC Davis Comprehensive Cancer Center Recruiting
Sacramento, California, United States, 95817
UC Davis CTSC Clinical Research Center Recruiting
Sacramento, California, United States, 95817
UC Davis Health Recruiting
Sacramento, California, United States, 95817
UC Davis Investigational Pharmacy Recruiting
Sacramento, California, United States, 95817
United States, Mississippi
Mississippi Center for Advanced Medicine Recruiting
Madison, Mississippi, United States, 39110
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Australia, New South Wales
Royal Prince Alfred Hospital Recruiting
Camperdown, New South Wales, Australia, 2050
Sponsors and Collaborators
Pfizer
Investigators
Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03307980     History of Changes
Other Study ID Numbers: C0371003
SPK-9001-LTFU-101 ( Other Identifier: Alias Study Number )
First Posted: October 12, 2017    Key Record Dates
Last Update Posted: December 7, 2018
Last Verified: December 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: http://www.pfizer.com/research/clinical_trials/trial_data_and_results/data_requests

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pfizer:
gene therapy

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked