Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

NCT03307980

Last updated date
Study Location
Weill Cornell Medicine - New York Presbyterian Hospital
New York, New York, 10065, United States
Contact
1-800-718-1021

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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
18-65 years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

1. Able to provide informed consent and comply with requirements of the study

2. Males age 18 to 65 years with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous factor IX)

3. Received ≥50 exposure days to factor IX products

4. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein

5. Agree to refrain from donating sperm and either abstain from intercourse or use reliable barrier contraception until 3 consecutive semen samples are negative for vector sequences

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


1. Evidence of active hepatitis B or C


2. Currently on antiviral therapy for hepatitis B or C


3. Have significant underlying liver disease


4. Serological evidence* of HIV-1 or HIV-2 with CD4 counts ≤200/mm3 (* participants who
are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible
to enroll)


5. Neutralizing antibody titers to the capsid portion of PF-06838435 above the
established threshold


6. Sensitivity to heparin or heparin induced thrombocytopenia; sensitivity to any of the
study interventions, or components thereof, or drug or other allergy


7. Previously dosed in a gene therapy research trial at any time or in an interventional
clinical study within 3 months of screening visit


8. Any concurrent clinically significant major disease or condition


9. Unable or unwilling to comply with the study procedures

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Advanced Information
Descriptive Information
Brief Title  ICMJE Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Official Title  ICMJE A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF PF 06838435 AND A DOSE ESCALATION SUBSTUDY IN INDIVIDUALS WITH HEMOPHILIA B
Brief Summary Long-term safety and efficacy follow-up for participants with Hemophilia B who were previously treated in the C0371005 (formerly SPK-9001-101) study, and a dose-escalation substudy evaluating safety, tolerability, and kinetics of a higher dose with long-term safety and efficacy follow-up.
Detailed Description Evaluation of the long-term level of persistence and potential late or delayed adverse events associated with PF-06838435 (formerly SPK-9001), assessment of the durability of the transgene expression, and determination of the effects of PF-06838435 on clinical outcomes in individuals who have previously received a single administration of PF-06838435 in the C0371005 study. Amendment 2 of this study incorporates a dose-escalation substudy to evaluate the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher dose than that used in the C0371005 study. The dose-escalation participants will also be followed for long-term safety and efficacy.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Sequential Assignment
Intervention Model Description:

This study was originally designed as a long-term follow up study for individuals dosed in Study C0371005 to evaluate the overall long-term safety, durability of transgene expression, and effect on clinical outcomes of PF-06838435 mediated gene transfer. For these individuals this study will last for 5 years providing a minimum of 6 years of follow up post vector administration.

Amendment 2 of this study introduces a dose-escalation substudy to evaluate the safety, tolerability, and kinetics of a single IV infusion of PF-06838435 at a higher dose(s) than that used in the C0371005 study. For these participants this study will last for a total of 6 years post vector administration.

Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hemophilia B
Intervention  ICMJE Biological: PF-06838435 (formerly SPK-9001)
Gene Therapy: A novel, bioengineered adeno-associated viral vector carrying human factor IX variant
Study Arms  ICMJE Experimental: PF-06838435 Dose-Escalation
Single intravaneous infusion of PF-06838435. After 2 participants receive initial dose, data will be evaluated and a decision will be made to escalate or reduce the dose being evaluated, increase the number of participants receiving the dose, or stop dosing. Multiple iterations may be undertaken.
Intervention: Biological: PF-06838435 (formerly SPK-9001)
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: January 6, 2021)
55
Original Estimated Enrollment  ICMJE
 (submitted: October 6, 2017)
11
Estimated Study Completion Date  ICMJE November 10, 2027
Estimated Primary Completion Date November 10, 2027   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

This study is currently only enrolling into the dose-escalation substudy with subsequent long-term follow-up. The Eligibility Criteria for entry into the dose-escalation substudy is presented below:

Inclusion Criteria:

  1. Able to provide informed consent and comply with requirements of the study
  2. Males age 18 to 65 years with confirmed diagnosis of hemophilia B (?2 IU/dL or ?2% endogenous factor IX)
  3. Received ?50 exposure days to factor IX products
  4. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
  5. Agree to refrain from donating sperm and either abstain from intercourse or use reliable barrier contraception until 3 consecutive semen samples are negative for vector sequences

Exclusion Criteria:

  1. Evidence of active hepatitis B or C
  2. Currently on antiviral therapy for hepatitis B or C
  3. Have significant underlying liver disease
  4. Serological evidence* of HIV-1 or HIV-2 with CD4 counts ?200/mm3 (* participants who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
  5. Neutralizing antibody titers to the capsid portion of PF-06838435 above the established threshold
  6. Sensitivity to heparin or heparin induced thrombocytopenia; sensitivity to any of the study interventions, or components thereof, or drug or other allergy
  7. Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within 3 months of screening visit
  8. Any concurrent clinically significant major disease or condition
  9. Unable or unwilling to comply with the study procedures
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Gender Eligibility Description:Genetic males
Ages  ICMJE 18 Years to 65 Years   (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 January 2021

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