ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
- 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
- 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
NEED INFO?
Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

TRY A NEW SEARCH
Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.
Based on your search, you may also be interested in
- Chamberry, Cedex
- Le Chesnay, Cedex
- Caen Cedex 9,
- Clermont Ferrand,
- Dijon Cedex,
- leKremlin-Bicetre,
- Lyon,
- Marseille Cedex 05,
- Montmorency,
- Montpellier Cedex 5,
- Nantes cedex 1,
- Paris,
- Paris,
- Rouen,
- Saint Priest en Jarez,
- Tours,
- Vandoeuvre Les Nancy Cedex,
- Phoenix, Arizona
- Chicago, Illinois
- Chicago, Illinois
- New Brunswick, New Jersey
- Houston, Texas
- Edmonton, Alberta
- Edmonton, Alberta
- Ottawa, Ontario
- Zagreb,
- Budapest,
- Castelfranco Veneto (TV),
- Coppito (AQ),
- Bucuresti,
- Moscow,
- Saint Petersburg,
- Belgrade,
- Nis,
- Madrid,
- Sevilla,
- Los Angeles, California
- Aurora, Colorado
- Detroit, Michigan
- New Brunswick, New Jersey
- Chapel Hill, North Carolina
- Dayton, Ohio
- Houston, Texas
- Shinjuku-ku, Tokyo
Descriptive Information | |||||||
---|---|---|---|---|---|---|---|
Brief Title ICMJE | A Gene Therapy Study for Hemophilia B | ||||||
Official Title ICMJE | Gene Therapy, Open-label, Dose-escalation Study of PF-06838435 (SPK-9001) [Adeno-associated Viral Vector With Human Factor IX Gene] in Subjects With Hemophilia B | ||||||
Brief Summary | A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B. | ||||||
Detailed Description | 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). | ||||||
Study Type ICMJE | Interventional | ||||||
Study Phase ICMJE | Phase 2 | ||||||
Study Design ICMJE | Intervention Model: Sequential Assignment Masking: None (Open Label) Primary Purpose: Treatment | ||||||
Condition ICMJE | Hemophilia B | ||||||
Intervention ICMJE | Biological: SPK-9001
A novel, bioengineered adeno-associated viral vector carrying human factor IX variant | ||||||
Study Arms ICMJE | 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 | ||||||
Publications * | 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. | |||||||
Recruitment Information | |||||||
Recruitment Status ICMJE | Completed | ||||||
Actual Enrollment ICMJE | 15 | ||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||
Actual Study Completion Date ICMJE | April 8, 2019 | ||||||
Actual Primary Completion Date | April 8, 2019 (Final data collection date for primary outcome measure) | ||||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
| ||||||
Sex/Gender ICMJE |
| ||||||
Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||||
Accepts Healthy Volunteers ICMJE | No | ||||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||||
Listed Location Countries ICMJE | Australia, United States | ||||||
Removed Location Countries | Canada | ||||||
Administrative Information | |||||||
NCT Number ICMJE | NCT02484092 | ||||||
Other Study ID Numbers ICMJE | C0371005 SPK-9001-101 ( Other Identifier: Alias Study Number ) | ||||||
Has Data Monitoring Committee | Yes | ||||||
U.S. FDA-regulated Product |
| ||||||
IPD Sharing Statement ICMJE |
| ||||||
Responsible Party | Pfizer | ||||||
Study Sponsor ICMJE | Pfizer | ||||||
Collaborators ICMJE | Not Provided | ||||||
Investigators ICMJE |
| ||||||
PRS Account | Pfizer | ||||||
Verification Date | June 2020 | ||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |