ASC618 Gene Therapy in Hemophilia A Patients

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
ASC Therapeutics
Study ID
NCT04676048
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Hemophilia A

Eligibility Criteria

Sex
MALE
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • ASC618 — BIOLOGICAL
    ASC618 will be given as a single IV infusion

Study Details

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

Key Dates

Start date
Aug 3, 2022
Status verified
Aug 2022
Primary completion
Dec 31, 2023
Completion
Dec 31, 2026

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: ASC618
    Experimental Arm

Primary Outcome Measure

Number of adverse events, and serious AEs [ Time Frame: 12 months post-infusion ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202-

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By research site
Arkansas Children's Hospital· Little Rock, AR

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