|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
18 July 2023 |
|
Main ID: |
NCT03876301 |
|
Date of registration:
|
11/03/2019 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
|
|
Scientific title:
|
A Multi-Center, Observational Study in Males With Hemophilia A |
|
Date of first enrolment:
|
January 21, 2019 |
|
Target sample size:
|
25 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/ct2/show/NCT03876301 |
|
Study type:
|
Observational |
|
Study design:
|
|
|
Phase:
|
|
|
|
Countries of recruitment
|
|
Australia
|
Canada
|
Thailand
|
United States
| | | | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
1. Able and willing to provide written informed consent.
2. Males =18 years of age.
3. Clinically severe hemophilia A
4. Previous exposure to FVIII therapy
5. No prior history of hypersensitivity or anaphylaxis associated with an FVIII or
intravenous immunoglobulin administration.
6. No measurable inhibitor against FVIII
7. Willing to participate and receive treatment in a future Spark hemophilia A gene
therapy study.
Exclusion Criteria:
1. Documented active hepatitis B or C within the past 12 months of Screening
2. Currently on antiviral therapy to treat hepatitis B or C;
3. Documented significant liver disease within the past 6 months of Screening
4. Have serological evidence of HIV-1 or HIV-2
5. Anti-AAV-Spark 200 neutralizing titers =1:1
6. Previously received SPK-8011;
7. Previously dosed with any investigational or approved gene therapy product at any time
or treated with an investigational drug within the last 12 weeks;
8. Planned surgical procedure in the next 12 months requiring FVIII prophylactic
treatment.
9. Any history of chronic infection or other chronic disease, concurrent clinically
significant major disease (such as liver abnormalities or type I diabetes) including
active malignancy, except for non-melanoma skin cancer, any other condition or any
other unspecified reasons that, in opinion of the Investigator or Sponsor, makes the
participant unsuitable for participation and dosing in a future clinical study for
Spark's hemophilia A gene therapy.
10. Unable or unwilling to comply with the schedule of visits and/or study assessments
described in the protocol.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
Male
|
|
Health Condition(s) or Problem(s) studied
|
|
Coagulation Protein Disorders
|
|
Hematologic Diseases
|
|
Hemophilia A
|
|
Factor VIII Deficiency
|
|
Hemorrhagic Disorders
|
|
Blood Coagulation Disorder
|
|
Genetic Diseases, Inborn
|
|
Blood Coagulation Disorders, Inherited
|
|
Genetic Diseases, X-Linked
|
|
Intervention(s)
|
|
Drug: Standard of Care FVIII Replacement therapy
|
|
Primary Outcome(s)
|
|
Number of bleeding events, annualized
[Time Frame: 12 months]
|
|
Secondary Outcome(s)
|
|
Dose and total FVIIII consumption
[Time Frame: 12 months]
|
|
Annualized number of infusions (AIR)
[Time Frame: 12 months]
|
|
Secondary ID(s)
|
|
SPK-8011-301
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|