Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B



Status:Recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:18 - Any
Updated:2/14/2019
Start Date:June 22, 2017
End Date:December 29, 2023
Contact:Pfizer CT.gov Call Center
Email:ClinicalTrials.gov_Inquiries@pfizer.com
Phone:1-800-718-1021

Use our guide to learn which trials are right for you!

FIX LONG STUDY: A FACTOR IX (FIX) GENE TRANSFER, MULTI CENTER EVALUATION OF THE LONG TERM SAFETY AND EFFICACY STUDY OF SPK 9001 (RAAV SPARK100 HFIX PADUA) IN INDIVIDUALS WITH HEMOPHILIA B

Long-term safety follow-up for subjects with Hemophilia B and previously treated in the
C0371005 (formerly SPK-9001-101) protocol

Evaluation of the long term level of persistence and potential late or delayed adverse events
associated with SPK-9001, assessment of the durability of the transgene expression, and
determination of the effects of SPK-9001 on clinical outcomes in individuals who have
previously received a single administration of SPK-9001

Inclusion Criteria:

1. Evidence of a personally signed and dated informed consent document indicating that
the subject has been informed of all pertinent aspects of the study.

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

3. Subjects who met the C0371005 eligibility criteria, have received a single
administration of SPK-9001, and completed the required assessments in the C0371005
clinical study.

Exclusion Criteria:

1. Investigator site staff members directly involved in the conduct of the study and
their family members, site staff members otherwise supervised by the investigator, or
subjects who are Pfizer employees, including their family members, directly involved
in the conduct of the study.

2. Participation in other studies involving investigational drug(s) within the last 12
weeks prior to study entry, excluding participation in C0371005, and/or during study
participation.

3. Other acute or chronic medical or psychiatric condition including recent (within the
past year) or active suicidal ideation or behavior or laboratory abnormality that may
increase the risk associated with study participation or investigational product
administration or may interfere with the interpretation of study results and, in the
judgment of the investigator, would make the subject inappropriate for entry into this
study.

4. Fertile male subjects who are unwilling or unable to use a condom as outlined in this
protocol until 3 consecutive semen samples are negative in the vector shedding
analysis after the last dose of investigational product.

5. Subjects who will not consent for up to 5 years of safety follow-up.

6. Subjects who are unable or unwilling to comply with the study visits and requirements.

7. Subjects with any clinically significant medical condition that the Investigator
believes would pose as a safety risk or are not capable of performing the endpoints of
this study.
We found this trial at
8
sites
Sacramento, California 95817
?
mi
from
Sacramento, CA
Click here to add this to my saved trials
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
?
mi
from
Philadelphia, PA
Click here to add this to my saved trials
Camperdown, New South Wales 2050
?
mi
from
Camperdown,
Click here to add this to my saved trials
Madison, Mississippi 39110
?
mi
from
Madison, MS
Click here to add this to my saved trials
Sacramento, California 95816
?
mi
from
Sacramento, CA
Click here to add this to my saved trials
4501 X St
Sacramento, California 95817
(916) 734-5800
UC Davis Comprehensive Cancer Center When faced with cancer, you want the best hope for...
?
mi
from
Sacramento, CA
Click here to add this to my saved trials
Sacramento, California 95817
?
mi
from
Sacramento, CA
Click here to add this to my saved trials
2315 Stockton Blvd
Sacramento, California 95817
?
mi
from
Sacramento, CA
Click here to add this to my saved trials