A Gene Therapy Study for Hemophilia B

Completed · Phase 2

Conditions studied: Hemophilia B

In brief

A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.

Key facts

Study ID

NCT02484092

Run by

Pfizer

People needed

15

Starts

2015-11-18

Expected to finish

2019-04-08

Last updated by the study team

2020-06-16

Who can join

Age: 18 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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)
• Neutralizing antibodies reactive with AAV-Spark100 above and/or below a defined titre
• 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

Where it is running

• UC Davis Comprehensive Cancer Center — Sacramento, California, United States
• UC Davis CTSC Clinical Research Center — Sacramento, California, United States
• UC Davis Ellison Ambulatory Care Clinic — Sacramento, California, United States
• UC Davis Investigational Pharmacy — Sacramento, California, United States
• UC Davis Medical Center — Sacramento, California, United States
• University of Mississippi Medical Center — Jackson, Mississippi, United States
• Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
• Weill Cornell Medicine - New York Presbyterian Hospital — New York, New York, United States
• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Royal Prince Alfred Hospital — Camperdown/Sydney, New South Wales, Australia

Full record on ClinicalTrials.gov

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