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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 : June 4, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Brief Summary:
Long-term safety follow-up for subjects with Hemophilia B and previously treated in the C0371005 (formerly 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

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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 : May 16, 2026
Estimated Study Completion Date : May 16, 2026

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 C0371005 (formerly 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

  8. Brief Pain Inventory [ Time Frame: up to 5 years ]
    Quality-of-life (QoL) assessment



Information from the National Library of Medicine

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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. 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.

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

Locations
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United States, California
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
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Study Director: Pfizer CT.gov Call Center Pfizer

Additional Information:
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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: June 4, 2019
Last Verified: June 2019
Individual Participant Data (IPD) Sharing Statement:
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/data_requests.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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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:
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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