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A Gene Therapy Study for Hemophilia B

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NCT02484092

Last updated on August 22, 2018
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FOR MORE INFORMATION
Study Location
UC Davis Comprehensive Cancer Center
Sacramento, California, 95817 United States
See other locations
Contact
1-800-718-1021
[email protected]
Related Links
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

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

- Males ≥18 y.o. with confirmed diagnosis of hemophilia B (≤2 IU/dL or ≤2% endogenous
factor IX)

- Received ≥50 exposure days to factor IX products

- A minimum average of 4 bleeding events per year requiring episodic treatment of factor
IX infusions or prophylactic factor IX infusions

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

- Agree to use reliable barrier contraception until 3 consecutive samples are negative
for vector sequences

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

- Evidence of active hepatitis B or C

- Currently on antiviral therapy for hepatitis B or C

- Have significant underlying liver disease

- Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ?200/mm3 (* subjects who
are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible
to enroll)

- Have detectable antibodies reactive with AAV-Spark100

- Participated in a gene transfer trial within the last 52 weeks or in a clinical trial
with an investigational drug within the last 12 weeks

- Unable or unwilling to comply with study assessments

NCT02484092
Pfizer
Active, not recruiting
A Gene Therapy Study for Hemophilia B

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Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

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A Gene Therapy Study for Hemophilia B
Gene Therapy, Open-label, Dose-escalation Study Of Spk-9001 [Adeno-associated Viral Vector With Human Factor Ix Gene] In Subjects With Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

Hemophilia B, or Christmas disease, is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes leads to death due to the inability for blood to clot efficiently. This chronic joint disease can have significant physical, psychosocial, and quality-of-life effects, including financial burden. The current treatment is intravenous infusion of FIX protein products, either prophylactically or in response to bleeding.

The approach being tested in this study uses a novel recombinant adeno-associated virus (AAV), which in nature causes no disease, to deliver the human factor IX (hFIX) gene to the liver cells where FIX is normally made. Recent data of a gene therapy study showed preliminary encouraging results with the approach of using an AAV vector carrying the factor IX gene. This study will seek to determine the safety and kinetics of a single IV infusion of SPK-9001 (a novel AAV vector carrying a high specific activity factor IX variant).
Interventional
Phase 2
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Hemophilia B
Biological: SPK-9001
A novel, bioengineered adeno-associated viral vector carrying human factor IX variant
Experimental: SPK-9001
Single intravenous (i.v.) infusion of SPK-9001 [an adeno-associated viral (AAV) vector with human factor IX gene] Intervention: Gene Therapy / Gene Transfer
Intervention: Biological: SPK-9001
George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
15
Same as current
April 30, 2019
April 30, 2019   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Able to provide informed consent and comply with requirements of the study
  • Males ?18 y.o. with confirmed diagnosis of hemophilia B (?2 IU/dL or ?2% endogenous factor IX)
  • Received ?50 exposure days to factor IX products
  • A minimum average of 4 bleeding events per year requiring episodic treatment of factor IX infusions or prophylactic factor IX infusions
  • No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein
  • Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences

Exclusion Criteria:

  • Evidence of active hepatitis B or C
  • Currently on antiviral therapy for hepatitis B or C
  • Have significant underlying liver disease
  • Have serological evidence* of HIV-1 or HIV-2 with CD4 counts ?200/mm3 (* subjects who are HIV+ and stable with CD4 count >200/mm3 and undetectable viral load are eligible to enroll)
  • Have detectable antibodies reactive with AAV-Spark100
  • Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 12 weeks
  • Unable or unwilling to comply with study assessments
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Gender Eligibility Description: Genetic Males
18 Years and older   (Adult, Older Adult)
No
Contact information is only displayed when the study is recruiting subjects
Australia,   Canada,   United States
 
 
NCT02484092
C0371005
SPK-9001-101 ( Other Identifier: Sparks Therapeutics )
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
August 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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