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Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%)

Last updated on August 9, 2018

FOR MORE INFORMATION
Study Location
Contact
1-800-718-1021
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
18-64 years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- Males ≥ 18 and documented FIX activity (≤2%) within the last 12 months prior to baseline visit.

- Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan,
laboratory tests and other study procedures.

- Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein
product such as recombinant, plasma-derived or extended half-life FIX product)

- Subjects as per usual care setting on FIX prophylaxis replacement therapy
(recombinant, plasma-derived or extended half-life FIX product) must have the
intention to remain on FIX prophylaxis replacement therapy for the duration of the
study

- No known hypersensitivity to FIX replacement product

- No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a
titer ≥ to 0.6 BU/mL

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

- Anti-AAV-Spark100 neutralizing antibodies titer (above the lowest detectable titer)
performed by a central laboratory during screening.

- Lack of patient compliance with documentation of bleeds and/or FIX prophylaxis
replacement therapy administration.

- A subject is not eligible if either HbsAg is positive or HBV-DNA is
positive/detectable.

- A subject who is currently undergoing anti-viral therapy for hepatitis B is not
eligible

- A subject who is currently undergoing anti-viral therapy for chronic hepatitis C is
not eligible.

- A subject is not eligible if his HCV-RNA load assay result is positive/detectable.

- Currently on antiviral therapy for hepatitis B or C.

- A subject is not eligible if any of the following pre-existing diagnoses, which are
indicative of significant underlying liver disease, are present in the medical record:

- Portal hypertension; or

- Splenomegaly; or

- Hepatic encephalopathy

- A subject is not eligible if the serum albumin level is below the testing laboratory's
lower limit of normal; and

- At least one of the following diagnostic tests for liver fibrosis indicating ?
stage 3. The following results are indicative of fibrosis ? stage 3 and exclude
the subject from participation:

- FibroScan, with a score >8.3 kPa units;

- FibroTest/FibroSURE with a result >0.48; or

- AST-to-Platelet Ratio Index (APRI) >1.

- Documented serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with
Cluster of Differentiation 4 positive (CD4+) cell count ?200 mm3 within the last 12
months prior to screening.

- History of chronic infection or other chronic disease that the investigator deems an
unacceptable risk. Any patient with a history of thrombotic events including but not
limited to stroke or myocardial infarction. Any concurrent clinically significant
major disease or condition that the investigator deems unsuitable for participation.

- Participation in other studies involving investigational drug(s) within the last 3
months prior to study entry and/or during study participation or in a previous gene
therapy clinical study within the last 12 months prior to screening.

- Any subject with a planned surgical procedure requiring FIX surgical prophylactic
factor treatment in the next 24 months.

NCT03587116
Pfizer
Not yet recruiting
Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%)

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Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C?2%)
An Open-label, Non-investigational Product, Multi-center, Lead-in Study To Evaluate At Least 6 Months Of Prospective Efficacy And Selected Safety Data Of Factor Ix (Fix) Prophylaxis Replacement Therapy In The Usual Care Setting Of Moderately Severe To Severe Adult Hemophilia B Subjects (Fix:c?2%) Who Are Negative For Neutralizing Antibodies (Nab) To Adeno-associated Virus Vector (Aav)-spark100
To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for NAb to AAV-Spark100, prior to the Phase 3 gene therapy study.
Not Provided
Interventional
Phase 3
Intervention Model: Single Group Assignment
Intervention Model Description:
The data obtained from this 6 month lead-in study will serve as the control group for the subsequent Phase 3 gene therapy study.

Masking: None (Open Label)
Primary Purpose: Other

Hemophilia B
Other: Standard of Care FIX Replacement therapy
There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy.
Standarad of Care FIX replacement therapy
Intervention: Other: Standard of Care FIX Replacement therapy
Not Provided


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Not yet recruiting
110
Same as current
November 16, 2020
November 16, 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Males ? 18 and < 65 years of age with moderate severe to severe hemophilia B and documented FIX activity (?2%) within the last 12 months prior to baseline visit.
  • Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan, laboratory tests and other study procedures.
  • Previous experience with FIX therapy (?50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product)
  • Subjects as per usual care setting on FIX prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FIX product) must have the intention to remain on FIX prophylaxis replacement therapy for the duration of the study
  • No known hypersensitivity to FIX replacement product
  • No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a titer ? to 0.6 BU/mL

Exclusion Criteria:

  • Anti-AAV-Spark100 neutralizing antibodies titer (above the lowest detectable titer) performed by a central laboratory during screening.
  • Lack of patient compliance with documentation of bleeds and/or FIX prophylaxis replacement therapy administration.
  • A subject is not eligible if either HbsAg is positive or HBV-DNA is positive/detectable.
  • A subject who is currently undergoing anti-viral therapy for hepatitis B is not eligible
  • A subject who is currently undergoing anti-viral therapy for chronic hepatitis C is not eligible.
  • A subject is not eligible if his HCV-RNA load assay result is positive/detectable.
  • Currently on antiviral therapy for hepatitis B or C.
  • A subject is not eligible if any of the following pre-existing diagnoses, which are indicative of significant underlying liver disease, are present in the medical record:

    • Portal hypertension; or
    • Splenomegaly; or
    • Hepatic encephalopathy
  • A subject is not eligible if the serum albumin level is below the testing laboratory's lower limit of normal; and

    • At least one of the following diagnostic tests for liver fibrosis indicating ? stage 3. The following results are indicative of fibrosis ? stage 3 and exclude the subject from participation:
    • FibroScan, with a score >8.3 kPa units;
    • FibroTest/FibroSURE with a result >0.48; or
    • AST-to-Platelet Ratio Index (APRI) >1.
  • Documented serological evidence of human immunodeficiency virus HIV-1 or HIV-2 with Cluster of Differentiation 4 positive (CD4+) cell count ?200 mm3 within the last 12 months prior to screening.
  • History of chronic infection or other chronic disease that the investigator deems an unacceptable risk. Any patient with a history of thrombotic events including but not limited to stroke or myocardial infarction. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation.
  • Participation in other studies involving investigational drug(s) within the last 3 months prior to study entry and/or during study participation or in a previous gene therapy clinical study within the last 12 months prior to screening.
  • Any subject with a planned surgical procedure requiring FIX surgical prophylactic factor treatment in the next 24 months.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
18 Years to 64 Years   (Adult)
No

Contact: Pfizer CT.gov Call Center 1-800-718-1021 [email protected]
Not Provided
 
 
NCT03587116
C0371004
2017-001271-23 ( EudraCT Number )
NAB PROTOCOL ( Other Identifier: Alias Study Number )
Not Provided
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
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/da...
URL: http://
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
July 2018

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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1-800-718-1021

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[email protected]



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