Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation i… (NCT03734588) | Clinical Trial Compass
CompletedPhase 1/2
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
United States4 participantsStarted 2019-01-30
Plain-language summary
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with severe hemophilia A and no measurable inhibitor against FVIII.
Who can participate
Age range
18 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Be male and ≥18 years of age;
. Have clinically severe hemophilia A, defined as:
. \<1% (\<1 IU/dL) endogenous FVIII activity levels as historically documented by a certified laboratory or screening data results; OR
. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and \> 10 bleeding events per year (in the last 52 weeks prior to screening); OR
. 1-2% (1-2 IU/dL) endogenous FVIII activity levels and on prophylaxis;
. Have had \>150 exposure days (EDs) to any recombinant and/or plasma-derived FVIII concentrates or cryoprecipitates
. Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Adverse Events (AEs)
Timeframe: Up to week 52
2
Number of Participants With Hepatic Transaminase Elevation Requiring Immunosuppression.
Timeframe: Up to week 52
3
Peak FVIII Activity Levels Assessed by Coagulation Clotting Assays
Timeframe: Up to week 52
4
Steady-state FVIII Activity Levels Assessed by Coagulation Clotting Assays
Timeframe: Up to week 52
5
Number of Bleeding Events (Spontaneous and Traumatic) Since 28 Day Post Vector Administration
Timeframe: From 28 days post vector administration up to week 52
6
Annualized Infusion Rate
Timeframe: From 28 days post vector administration up to week 52
. Have no measurable inhibitor against FVIII as assessed by central laboratory, have no confirmed history of clinically significant FVIII inhibitor, and no clinical signs or symptoms of decreased response to FVIII administration (Note: family history of inhibitors will not exclude study participation)
Exclusion criteria
. Have active hepatitis B or C
. Have significant underlying liver disease.
. Have serological evidence of HIV-1 or HIV-2 with CD4 counts ≤200/mm3. Participants who are HIV-positive and stable, with an adequate CD4 count (\>200/mm3) and undetectable viral load, and are on an antiretroviral drug regimen are eligible to enroll
. Have detectable antibodies reactive with AAV-Spark capsid
. Have history of chronic infection or other chronic disease
. Have been dosed in a previous gene therapy research trial within the last 52 weeks or with an investigational drug within the last 12 weeks
. Any concurrent clinically significant major disease (such as liver abnormalities or type I diabetes) or other condition that, in the opinion of the Investigator and/or Sponsor, makes the subject unsuitable for participation in the study;
. Unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol.