Hemophilia APhase 1/2NCT04676048
ASC618 Gene Therapy in Hemophilia A Patients
A Phase 1/2 study testing ASC618 in people with Hemophilia A, run by ASC Therapeutics at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ASC618
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Aug 2022to Dec 2026
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests.
- Current inhibitors, or history of high titer FVIII inhibitors
- Presence of > Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology
- History of chronic renal disease
- Active infection or any immunosuppressive disorder
- History of cardiac surgery and need anticoagulant therapy
Show all 10Show fewer
- Any cardiovascular / genetic risk factors for thromboembolic disorders
- Evidence of active Hepatitis B, Hepatitis C, Human Immunodeficiency Virus (HIV)-1/2 or syphilis infection.
- Receipt of any vector or gene transfer agent
- Current antiviral therapy for hepatitis B or C
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
About this study
Official title: Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
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
- Conditions
- Hemophilia A
- Being tested
- ASC618
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ASC618Biological
ASC618 will be given as a single IV infusion
| Study group | Role | What they receive |
|---|---|---|
| ASC618 | Experimental | Experimental Arm Receives: Biological: ASC618 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse events, and serious AEs
Measured: 12 months post-infusion
Secondary outcomes (3)
Changes in FVIII activity levels from baseline
Measured: 12 months post-infusion
Annualized FVIII consumption
Measured: 12 months post-infusion
Annualized bleeding rate (ABR)
Measured: 12 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Dec 2020
- Study start3 Aug 2022
- Primary completionDec 2023 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- ASC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04676048
- Sponsor's protocol ID
- ASC-HA-001
- Status last verified
- Aug 2022
- First submitted
- 15 Dec 2020
- Last updated
- 1 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Safety and Preliminary Efficacy of ASC618 in Subjects With Severe and Moderately Severe Hemophilia A
- ClinicalTrials.gov ID
- NCT04676048
- Acronym / sponsor ID
- ASC-HA-001
- Conditions
- Hemophilia A
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- ASC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 3 Aug 2022 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 19 Dec 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ASC618 | Experimental | Biological: ASC618Experimental Arm |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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)
Key exclusion criteria (10 listed)
- Pre-existing immunity to AAV8 vector as defined by AAV8 total antibodies and neutralizing antibodies qualified tests.
- Current inhibitors, or history of high titer FVIII inhibitors
- Presence of > Grade 2 liver fibrosis on elastography/Fibroscan or comparable imaging methodology
- History of chronic renal disease
- Active infection or any immunosuppressive disorder
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ASC618ASC618 will be given as a single IV infusion | Biological | ASC618 |
Primary outcome
Number of adverse events, and serious AEs
Time frame: 12 months post-infusion
3 secondary outcomes
Changes in FVIII activity levels from baseline
Time frame: 12 months post-infusion
Annualized FVIII consumption
Time frame: 12 months post-infusion
Annualized bleeding rate (ABR)
Time frame: 12 months post-infusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Feb 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ASC618 in people with Hemophilia A, run by ASC Therapeutics at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Clinical Trial Manager, PhD ((408) 495-3891, gil.gonen@asctherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse events, and serious AEs (time frame: 12 months post-infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Feb 2023.