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Gene Therapy
Gene Therapy for Hemophilia A
Phase 1 & 2
Recruiting
Research Sponsored by ASC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Male ≥18 years of age
Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated days (exposure days)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months post-infusion
Awards & highlights
Study Summary
This trial will test a new gene therapy for hemophilia A, which if effective could provide a much more convenient treatment option than current methods.
Who is the study for?
This trial is for male adults over 18 with severe or moderately severe hemophilia A, experiencing at least 12 bleeding episodes a year if on-demand therapy. They must have used replacement therapy for over 150 days and have a BMI of ≤30. Participants need to commit to using double-barrier contraception post-treatment until semen tests are negative.Check my eligibility
What is being tested?
The study is testing ASC618, an advanced gene therapy designed to deliver a corrected form of the factor VIII gene to patients with hemophilia A. The goal is to reduce the frequency of bleeding episodes and treatment injections by enabling sustained production of factor VIII in the body.See study design
What are the potential side effects?
Potential side effects may include immune reactions against the vector (AAV8), liver complications due to gene transfer, injection site reactions, and other unforeseen issues related to altering genes within the body.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am a man aged 18 or older.
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I have received FVIII therapy for at least 150 days.
Select...
I have severe or moderately severe hemophilia A.
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I've had 12 or more bleeding episodes in the last year while on on-demand therapy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months post-infusion
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months post-infusion
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of adverse events, and serious AEs
Secondary outcome measures
Annualized FVIII consumption
Annualized bleeding rate (ABR)
Changes in FVIII activity levels from baseline
Trial Design
1Treatment groups
Experimental Treatment
Group I: ASC618Experimental Treatment1 Intervention
Experimental Arm
Find a Location
Who is running the clinical trial?
ASC TherapeuticsLead Sponsor
1 Previous Clinical Trials
60 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am currently on medication for hepatitis B or C.I have or had high levels of Factor VIII inhibitors.My liver has significant scarring detected by a special scan.I do not have immunity to the AAV8 vector as tested by specific antibody tests.I have heart or genetic conditions that increase my risk for blood clots.I am a man aged 18 or older.I have received FVIII therapy for at least 150 days.I have had heart surgery and need blood thinners.I have received gene therapy.I have severe or moderately severe hemophilia A.I have a long-term kidney disease.I've had 12 or more bleeding episodes in the last year while on on-demand therapy.I do not have an active infection or a condition that weakens my immune system.
Research Study Groups:
This trial has the following groups:- Group 1: ASC618
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are we able to enroll new patients in this research project at this time?
"The clinicaltrials.gov website indicates that this study is still looking for participants. This research was originally posted on August 1st, 2022 and updated 2 weeks ago on the 17th of August. So far, only 12 people have been recruited from a single location."
Answered by AI
How many people will be included in this clinical research?
"That is correct, the online clinicaltrial.gov registry shows that this study is actively looking for participants. The trial was first announced on August 1st, 2022 and updated two weeks ago. So far, only 12 patients have been recruited from a single site."
Answered by AI
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