Acute Graft-versus-Host DiseasePhase 1NCT07070674
A Phase Ib Clinical Study of AC-003 Capsules in Subjects With aGVHD.
A Phase 1 study testing AC-003 in people with Acute Graft-versus-Host Disease, run by Accro Bioscience (Suzhou) Limited at 1 site in China.
Trial summary
The study was an open-label, single-arm, dose-escalation, Phase Ib, multi-center study to investigate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of AC-003 in patients with grade II-IV SR-aGVHD. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Graft-versus-Host Disease
- What is being tested?
- AC-003
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteChina
- Study dates
- 25 Jul 2024to 28 Sep 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female patients age ≥ 18 years.
- Patients who have undergone allogeneic hematopoietic stem cell transplantation (HSCT) from any donor source (including matched unrelated donor, haplo-identical).
- Diagnosis of grade II-IV aGVHD as per MAGIC criteria within 100 days after HSCT.
- Diagnosis of steroid-refractory or steroid-dependent aGVHD in at least one of the following criteria:
- Progressive Disease, defined as any target organ worsening, after 3 days of initial treatment with methylprednisolone 1~2 mg/kg/day (or equivalent corticosteroid)
- Not partial response after 7 days of initial treatment with methylprednisolone 1~2 mg/kg/day (or equivalent corticosteroid)
- Not complete response after 14 days of initial treatment with methylprednisolone 1~2 mg/kg/day (or equivalent corticosteroid)
- Relapse of aGVHD when corticosteroid tapering.
Who cannot join
- Evidence of aGVHD relapsed.
- Evidence of chronic GVHD or overlap syndrome
- Receipt of more than one allogeneic HSCT
- Receipt of more than one systemic treatment for aGVHD in addition to corticosteroid
- Any corticosteroid therapy for indications other than aGvHD at doses >= 1 mg/kg/day methylprednisolone (or equivalent corticosteroid) within 7 days prior to enrolment
- Severe organ dysfunction unrelated to aGVHD
- Uncontrolled active infection (i.e., bacterial, fungal, or viral)
Trial locations and contacts
1 site in 1 country.
China1 site
Hematology Hospital of Chinese Academy of Medical Sciences (Institute of Hematology, Chinese Academy of Medical Sciences)
Tianjin, Tianjin Municipality
About this study
Official title: A Phase Ib Clinical Trial to Investigate the Safety, Tolerability, Preliminary Efficacy, and Pharmacokinetic Profile of AC-003 Capsules in Patients With Acute GraftVersus-Host Disease (aGVHD)
The study was an open-label, single-arm, dose-escalation, Phase Ib, multi-center study to investigate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of AC-003 in patients with grade II-IV SR-aGVHD.
Read the detailed description
The study will enroll approximately 24 subjects in 2 parts:
Part A is a dose-escalation stage to investigate 4 cohorts of AC-003 in adult patients with grade II-IV SR-aGVHD who have undergone allogeneic hematopoietic stem cell transplantation.
The dose escalation will start with cohort 1, in which patients will receive specified dose of AC-003 capsules orally for 28 days and escalate to cohort 2, 3 and 4 sequentially when the previous cohort is deemed to be safe by Safety Review Committee (SRC). Safety follow-up will occur on Day 35. Decision of recommended dose will be made depend on the safety, PK and PD assessments across cohorts.
Part B is a dose-expansion stage to investigate the preliminary efficacy of recommended dose. Patient will receive recommended dose of AC-003 capsules orally for 28 days. Safety follow-up will occur on Day 35.
- Conditions
- Acute Graft-versus-Host Disease
- Being tested
- AC-003
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AC-003Drug
AC-003 capsules will be administered orally.
| Study group | Role | What they receive |
|---|---|---|
| AC-003 cohort 1 | Experimental | Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. Receives: Drug: AC-003 |
| AC-003 cohort 2 | Experimental | Specified dose of AC-003 will be administered orally once daily (QD) for 28 days. Receives: Drug: AC-003 |
| AC-003 cohort 3 | Experimental | Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. Receives: Drug: AC-003 |
| AC-003 cohort 4 | Experimental | Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. Receives: Drug: AC-003 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of Adverse Events
An Adverse Event (AE) is defined as any new untoward medical condition or worsening of a pre-existing medical condition following or during exposure to an investigation product, whether or not considered causally related to the product.
Measured: Up to Day 35
Secondary outcomes (2)
Overall Response Rate (ORR)
ORR is the percentage of patients with complete response (CR) or partial response (PR) as determined by investigator.
CR is defined as all target organs evaluated as Mount Sinai Acute GVHD International Consortium (MAGIC) Stage 0 [Skin - no active GVHD rash; Liver - bilirubin <2 mg/dL; Upper gastrointestinal (GI) tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: < 500 mL/day or <3 episodes/day (adults)].
PR is defined as improvement in one or more target organs (skin, liver, upper GI tract, lower GI tract) involved with aGVHD symptoms without worsening in others.
Measured: Day 14 and Day 28
Complete Response Rate
Complete Response Rate is the percentage of patients with complete response (CR).
CR is defined as all target organs evaluated as Mount Sinai Acute GVHD International Consortium (MAGIC) Stage 0 [Skin - no active GVHD rash; Liver - bilirubin <2 mg/dL; Upper gastrointestinal (GI) tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: < 500 mL/day or <3 episodes/day (adults)].
Measured: Day 14 and Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jul 2025
- Study start25 Jul 2024
- Primary completion28 Sep 2026
- Study completion28 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Graft-versus-Host DiseaseStudies 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Accro Bioscience (Suzhou) Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07070674
- Sponsor's protocol ID
- AC-003 -CN-PhIb
- Status last verified
- Oct 2026
- First submitted
- 30 Jun 2025
- Last updated
- 28 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib Clinical Trial to Investigate the Safety, Tolerability, Preliminary Efficacy, and Pharmacokinetic Profile of AC-003 Capsules in Patients With Acute GraftVersus-Host Disease (aGVHD)
- ClinicalTrials.gov ID
- NCT07070674
- Acronym / sponsor ID
- AC-003 -CN-PhIb
- Conditions
- Acute Graft-versus-Host Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Accro Bioscience (Suzhou) LimitedIndustry (pharma, biotech, devices)
- Study start
- 25 Jul 2024 (actual)
- Primary completion
- 28 Sep 2026 (actual)
- Study completion
- 28 Sep 2026 (actual)
- First posted
- 17 Jul 2025 (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.
The study was an open-label, single-arm, dose-escalation, Phase Ib, multi-center study to investigate safety, pharmacokinetics, pharmacodynamics and preliminary efficacy of AC-003 in patients with grade II-IV SR-aGVHD.
Detailed description
The study will enroll approximately 24 subjects in 2 parts:
Part A is a dose-escalation stage to investigate 4 cohorts of AC-003 in adult patients with grade II-IV SR-aGVHD who have undergone allogeneic hematopoietic stem cell transplantation.
The dose escalation will start with cohort 1, in which patients will receive specified dose of AC-003 capsules orally for 28 days and escalate to cohort 2, 3 and 4 sequentially when the previous cohort is deemed to be safe by Safety Review Committee (SRC). Safety follow-up will occur on Day 35. Decision of recommended dose will be made depend on the safety, PK and PD assessments across cohorts.
Part B is a dose-expansion stage to investigate the preliminary efficacy of recommended dose. Patient will receive recommended dose of AC-003 capsules orally for 28 days. Safety follow-up will occur on Day 35.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| AC-003 cohort 1 | Experimental | Drug: AC-003Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. |
| AC-003 cohort 2 | Experimental | Drug: AC-003Specified dose of AC-003 will be administered orally once daily (QD) for 28 days. |
| AC-003 cohort 3 | Experimental | Drug: AC-003Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. |
| AC-003 cohort 4 | Experimental | Drug: AC-003Specified dose of AC-003 will be administered orally twice daily (BID) for 28 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female patients age ≥ 18 years.
- Patients who have undergone allogeneic hematopoietic stem cell transplantation (HSCT) from any donor source (including matched unrelated donor, haplo-identical).
- Diagnosis of grade II-IV aGVHD as per MAGIC criteria within 100 days after HSCT.
- Diagnosis of steroid-refractory or steroid-dependent aGVHD in at least one of the following criteria:
Key exclusion criteria (7 listed)
- Evidence of aGVHD relapsed.
- Evidence of chronic GVHD or overlap syndrome
- Receipt of more than one allogeneic HSCT
- Receipt of more than one systemic treatment for aGVHD in addition to corticosteroid
- Any corticosteroid therapy for indications other than aGvHD at doses >= 1 mg/kg/day methylprednisolone (or equivalent corticosteroid) within 7 days prior to enrolment
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 |
|---|---|---|
| AC-003AC-003 capsules will be administered orally. | Drug | AC-003 cohort 1, AC-003 cohort 2, AC-003 cohort 3, AC-003 cohort 4 |
Primary outcome
Incidence and severity of Adverse Events
Time frame: Up to Day 35
An Adverse Event (AE) is defined as any new untoward medical condition or worsening of a pre-existing medical condition following or during exposure to an investigation product, whether or not considered causally related to the product.
2 secondary outcomes
Overall Response Rate (ORR)
Time frame: Day 14 and Day 28
Complete Response Rate
Time frame: Day 14 and Day 28
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Oct 2026.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AC-003 in people with Acute Graft-versus-Host Disease, run by Accro Bioscience (Suzhou) Limited at 1 site in China.
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 7 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: finished, as of the sponsor's last update on 28 Oct 2026. 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 China. 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 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of Adverse Events (time frame: Up to Day 35). 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 28 Oct 2026.