Relapsed/Refractory AL AmyloidosisPhase 1NCT07250269
Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis
A Phase 1 study testing GC012F Injection in people with Relapsed/Refractory AL Amyloidosis, run by Gracell Biotechnologies (Shanghai) Co., Ltd. at 9 sites in China.
Trial summary
This is a Phase 1b open-label, multicenter, non-randomized study of GC012F, a CD19/BCMA dual CAR T cell therapy, in adult participants with relapsed/refractory AL amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Relapsed/Refractory AL Amyloidosis
- What is being tested?
- GC012F Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 9 sitesChina
- Study dates
- 29 Oct 2025to 24 Nov 2028
- Phase
- Phase 1
- 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
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed histopathological diagnosis of AL amyloidosis
- One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
- Measurable hematologic disease: dFLC > 20 mg/L or serum M-protein > 5g/L
- Relapsed disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
- ECOG performance status of 0 to 1
- Must be able and willing to adhere to the study visit schedule and other protocol requirements
- Women of child-bearing potential (WCBP) must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study.
Who cannot join
- Have any other form of amyloidosis other than AL amyloidosis
- Mayo Stage IIIb AL amyloidosis
- Oxygen saturation < 95% on room air
- Systolic blood pressure <100mmHg
- Cardiac exclusion criteria:
- Mayo Stage IIIb AL amyloidosis as defined by
Show all 21Show fewer
- NT-proBNP>8500ng/L, and
- high sensitivity cardiac troponin T ≥ 50 ng/L cardiac Troponin T ≥ 0.035 ng/mL or cardiac Troponin I ≥ 0.1 ng/mL
- NT-proBNP levels as follows:
- NT-proBNP ≥ 2000 ng/L (for dose escalation portion)
- NT-proBNP < 2000 and > 8500 ng/L (for dose extension portion)
- NYHA class III or IV
- 5. Extensive GI involvement with evidence of active GI bleeding/risk of bleeding as determined by Investigator 6. Prior therapies:
- CAR T cell therapy directed at any target
- Prior BCMA-targeting therapy
- Prior treatment with any approved or investigational T cell engaging therapies (including T cell-directed bispecific or trispecific therapies) at any target within the last 6 months.
- 7. Toxicity from previous anti-cancer or anti-PC-directed therapy did not resolve to baseline levels or to Grade 1 or less except for alopecia or peripheral neuropathy.
- 8. Active plasma cell leukemia at the time of screening 9. Multiple myeloma defined as clonal bone marrow PCs ≥10% and any one or more of the following myeloma defining events (deemed as attributable to multiple myeloma by Investigator) 10. Seropositive for HIV 11. Serologic status reflecting active hepatitis B or C:
- Positive HBsAg, or
- Patients with positive core antibody (anti-HBc) and HBV-DNA positive.
- Patients with positive hepatitis C antibody and HCV RNA positive.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
9 sites in 1 country
China9 sites, 9 recruiting
- Recruiting
Research Site
Beijing, 100034
- Recruiting
Research Site
Beijing, 100070
- Recruiting
Research Site
Beijing, CN-100730
- Recruiting
Research Site
Changchun, 130021
- Recruiting
Research Site
Guangzhou, 510100
- Not yet recruiting
Research Site
Hangzhou, 310003
- Recruiting
Research Site
Suzhou, 215006
- Recruiting
Research Site
Wenzhou, 325000
- Not yet recruiting
Research Site
Wuhan, 430022
About this study
Official title: A Phase 1b Study of GC012F, a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B-cell Maturation Antigen in Chinese Participants With Relapsed or Refractory AL Amyloidosis
This is a Phase 1b open-label, multicenter, non-randomized study of GC012F, a CD19/BCMA dual CAR T cell therapy, in adult participants with relapsed/refractory AL amyloidosis.
Read the detailed description
This is a Phase 1b, open-label, multicenter, non-randomized study to evaluate the safety, tolerability, and efficacy of GC012F in adult participants with relapsed/refractory AL amyloidosis. A single-arm design was chosen for the study due to the absence of approved therapies for use as a concurrent control for this patient population. In the study, the safety of different doses of GC012F will be evaluated and the RP2D will be selected based on the totality of clinical safety, preliminary efficacy, CK and PD data.
- Conditions
- Relapsed/Refractory AL Amyloidosis
- Being tested
- GC012F Injection
- Keywords
- AZD0120, GC012F, Amyloidosis, AL Amyloidosis, CAR-T, Cell Therapy
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis, Amyloidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GC012F InjectionDrug
Also called AZD0120
The investigational agent, GC012F, is an autologous BCMA/CD19 dual directed CAR product under investigation for the treatment of patients with RRMM, ELMM, SLE, and B NHL.
| Study group | Role | What they receive |
|---|---|---|
| GC012F | Experimental | GC012F Injection Receives: Drug: GC012F Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With incidence and severity of Treatment-emergent Adverse Events
Measured: Through study completion, an average of 2 years
Number of participants with dose-limiting toxicities during dose escalation phase
Measured: Through study completion, an average of 2 years
Secondary outcomes (3)
Levels of GC012F in blood over time in participants with AL amyloidosis
Measured: Through study completion, an average of 2 years
Percentage of participants who achieve a hematologic response based on modified response criteria (CR, VGPR, or low dFLC PR)
Measured: Through study completion, an average of 2 years
Percentage of participants who achieve a complete response based on hematologic response criteria for AL amyloidosis
Measured: Through study completion, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2025
- Study start29 Oct 2025
- Primary completion24 Nov 2028 (estimated)
- Study completion24 Nov 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Relapsed/Refractory AL AmyloidosisStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gracell Biotechnologies (Shanghai) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Suzhou Gracell Biotechnologies Co., Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07250269
- Sponsor's protocol ID
- D831AC00002
- Other IDs
- AZD0120-AL-202
- Status last verified
- Jul 2026
- First submitted
- 21 Oct 2025
- Last updated
- 23 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
Study at a glance
From the registry record, last updated by the sponsor on 23 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b Study of GC012F, a Chimeric Antigen Receptor T Cell Therapy Targeting CD19 and B-cell Maturation Antigen in Chinese Participants With Relapsed or Refractory AL Amyloidosis
- ClinicalTrials.gov ID
- NCT07250269
- Acronym / sponsor ID
- D831AC00002
- Conditions
- Relapsed/Refractory AL Amyloidosis
- Keywords
- AZD0120, GC012F, Amyloidosis, AL Amyloidosis, CAR-T, Cell Therapy
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- Gracell Biotechnologies (Shanghai) Co., Ltd.Industry (pharma, biotech, devices)
- Collaborators
- Suzhou Gracell Biotechnologies Co., Ltd.
- Study start
- 29 Oct 2025 (actual)
- Primary completion
- 24 Nov 2028 (estimated)
- Study completion
- 24 Nov 2028 (estimated)
- First posted
- 26 Nov 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.
This is a Phase 1b open-label, multicenter, non-randomized study of GC012F, a CD19/BCMA dual CAR T cell therapy, in adult participants with relapsed/refractory AL amyloidosis.
Detailed description
This is a Phase 1b, open-label, multicenter, non-randomized study to evaluate the safety, tolerability, and efficacy of GC012F in adult participants with relapsed/refractory AL amyloidosis. A single-arm design was chosen for the study due to the absence of approved therapies for use as a concurrent control for this patient population. In the study, the safety of different doses of GC012F will be evaluated and the RP2D will be selected based on the totality of clinical safety, preliminary efficacy, CK and PD data.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| GC012F | Experimental | Drug: GC012F InjectionGC012F Injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Confirmed histopathological diagnosis of AL amyloidosis
- One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines
- Measurable hematologic disease: dFLC > 20 mg/L or serum M-protein > 5g/L
- Relapsed disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
- ECOG performance status of 0 to 1
Key exclusion criteria (21 listed)
- Have any other form of amyloidosis other than AL amyloidosis
- Mayo Stage IIIb AL amyloidosis
- Oxygen saturation < 95% on room air
- Systolic blood pressure <100mmHg
- Cardiac exclusion criteria:
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 |
|---|---|---|
| GC012F InjectionAlso: AZD0120The investigational agent, GC012F, is an autologous BCMA/CD19 dual directed CAR product under investigation for the treatment of patients with RRMM, ELMM, SLE, and B NHL. | Drug | GC012F |
Primary outcome
Number of Participants With incidence and severity of Treatment-emergent Adverse Events
Time frame: Through study completion, an average of 2 years
Number of participants with dose-limiting toxicities during dose escalation phase
Time frame: Through study completion, an average of 2 years
3 secondary outcomes
Levels of GC012F in blood over time in participants with AL amyloidosis
Time frame: Through study completion, an average of 2 years
Percentage of participants who achieve a hematologic response based on modified response criteria (CR, VGPR, or low dFLC PR)
Time frame: Through study completion, an average of 2 years
Percentage of participants who achieve a complete response based on hematologic response criteria for AL amyloidosis
Time frame: Through study completion, an average of 2 years
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 23 Jul 2026.
- Individual participant dataSharing planned: Yes. Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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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.
AstraZeneca Clinical Study Information Center contact
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing GC012F Injection in people with Relapsed/Refractory AL Amyloidosis, run by Gracell Biotechnologies (Shanghai) Co., Ltd. at 9 sites 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 7 inclusion and 21 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 23 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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?
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 AstraZeneca Clinical Study Information Center (1-877-240-9479, information.center@astrazeneca.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With incidence and severity of Treatment-emergent Adverse Events (time frame: Through study completion, an average of 2 years). The study lists 2 primary outcomes in all. 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 23 Jul 2026.