NCT04676048RecruitingPhase 1/2
ASC618 Gene Therapy in Hemophilia A Patients
Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
Source: ClinicalTrials.gov · Last updated 1 Feb 2023 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
Currently, hemophilia A patients are managed with prophylactic or on-demand replacement therapy with recombinant FVIII or alternative therapeutics. The major challenges of current treatment regimens, such as the short half-life of hemophilia therapeutics with the need for frequent IV injections, encourage the current efforts for gene transfer therapy. This study will evaluate the safety and preliminary efficacy of ASC618, an AAV vector encoding B-domain deleted codon-optimized human factor VIII under a synthetic liver-directed promoter
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 12 (estimated)
- First submitted
- 15 Dec 2020
- Start date
- 3 Aug 2022
- Primary completion date
- Dec 2023
- Lead sponsor
- ASC Therapeutics
- Diseases
- Haemophilia
- NCT number
- NCT04676048
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 12
- From 3 Aug 2022 to the primary completion date, Dec 2023
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests.
Other illness
- Current inhibitors, or history of high titer FVIII inhibitors
- History of chronic renal disease
- Active infection or any immunosuppressive disorder
- History of cardiac surgery and need anticoagulant therapy
- Any cardiovascular / genetic risk factors for thromboembolic disorders
- Evidence of active Hepatitis B, Hepatitis C, Human Immunodeficiency Virus (HIV)-1/2 or syphilis infection.
- Current antiviral therapy for hepatitis B or C
Other
- Presence of \> Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology
- Receipt of any vector or gene transfer agent
Who the study is looking for
- Male ≥18 years of age
- Severe or moderately severe hemophilia A (FVIII activity ≤ 2 IU/dL) as evidenced by
- medical history
- Received FVIII prophylactic or on-demand replacement therapy for ≥ 150 accumulated
- days (exposure days)
- ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
- BMI ≤ 30
- Agree to use double-barrier contraceptive until at least 3 consecutive semen samples are negative after ASC-618 infusion
Age in the registry: 18 years to - · Sex: Male · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Arkansas Children's Hospital Coordinator of this site? Confirm its status | Little Rock | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.