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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03307980




Registration number
NCT03307980
Ethics application status
Date submitted
29/09/2017
Date registered
12/10/2017
Date last updated
13/08/2019

Titles & IDs
Public title
Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B
Scientific title
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
Secondary ID [1] 0 0
SPK-9001-LTFU-101
Secondary ID [2] 0 0
C0371003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia B 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - SPK-9001

Other interventions: SPK-9001
Observational long-term safety follow-up study of subjects previously treated in the C0371005 (formerly SPK-9001-101) protocol

Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
SPK-9001-related adverse advents - adverse events
Timepoint [1] 0 0
up to 5 years
Secondary outcome [1] 0 0
Coagulation Clotting Assay for FIX activity levels - Coagulation Clotting assays to assess FIX activity levels (percent of normal)
Timepoint [1] 0 0
up to 5 years
Secondary outcome [2] 0 0
Annualized (factor FIX) infusion rate - AIR (not including those for surgery)
Timepoint [2] 0 0
up to 5 years
Secondary outcome [3] 0 0
Total factor consumption (IU and volume) - total quantity of factor infused annually (not including those for surgery) as recorded on the infusion log
Timepoint [3] 0 0
up to 5 years
Secondary outcome [4] 0 0
Annualized bleeding rate - ABR (not including those for surgery)
Timepoint [4] 0 0
up to 5 years
Secondary outcome [5] 0 0
Total number of bleeding events - (spontaneous and traumatic)
Timepoint [5] 0 0
up to 5 years
Secondary outcome [6] 0 0
Haem-A-QoL - Quality-of-life (QoL) assessment
Timepoint [6] 0 0
up to 5 years
Secondary outcome [7] 0 0
EQ-5D-5L - Quality-of-life (QoL) assessment
Timepoint [7] 0 0
up to 5 years
Secondary outcome [8] 0 0
Brief Pain Inventory - Quality-of-life (QoL) assessment
Timepoint [8] 0 0
up to 5 years

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Gender
Males
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Mississippi
Country [3] 0 0
United States of America
State/province [3] 0 0
Pennsylvania

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Long-term safety follow-up for subjects with Hemophilia B and previously treated in the
C0371005 (formerly SPK-9001-101) protocol
Trial website
https://clinicaltrials.gov/show/NCT03307980
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Country 0 0
Phone 0 0
1-800-718-1021
Fax 0 0
Email 0 0
ClinicalTrials.gov_Inquiries@pfizer.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03307980