ClinicalTrials.gov record NCT07250269
Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 1b, open-label, non-randomized study tests GC012F, a dual-target CAR‑T therapy, in adults with relapsed or refractory AL amyloidosis. It evaluates safety and tolerability across different doses and looks for early signs of hematologic response. Results from dose-escalation will be used to select a recommended phase 2 dose.
What it is studying. The study is testing the safety, tolerability, and early anti-disease activity of GC012F, an autologous CAR‑T product that targets CD19 and BCMA. It also measures how the drug behaves in the blood over time and records treatment-emergent adverse events and dose-limiting toxicities to guide dose selection.
What the researchers hope to learn. To evaluate safety and tolerability of GC012F; to identify dose-limiting toxicities; to measure levels of GC012F in blood over time; and to assess early hematologic response rates (complete response, very good partial response, partial response by modified criteria) to inform further development.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- GC012F Injection
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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.
Exclusion criteria
- 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
- 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
- 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.
- 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.
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will receive the investigational GC012F CAR‑T treatment (single‑arm; the study includes dose‑escalation to select a recommended dose). They will attend regular clinic visits with safety monitoring and blood samples to measure the drug and hematologic response, and investigators will record adverse events and any dose‑limiting toxicities. Follow-up for safety and response is planned through study completion (about 2 years on average). Women will have a pregnancy test before treatment and people of childbearing potential must use effective contraception during the study.
How the study is structured. Phase 1b, open-label, single‑arm (non-randomized) study with dose-escalation and dose-extension parts. Participants receive the investigational GC012F CAR‑T treatment and are followed for safety, laboratory measures, and disease response over the study period (primary outcomes tracked through about two years).
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
- The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on AL amyloidosis
Important dates
- Study start
- Oct 29, 2025
- Primary completion
- Nov 24, 2028 (estimated)
- Study completion
- Nov 24, 2028 (estimated)
- Target enrollment
- 9 participants (estimated)
- Last updated on registry
- Jul 23, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Gracell Biotechnologies (Shanghai) Co., Ltd.
- Collaborators
- Suzhou Gracell Biotechnologies Co., Ltd.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- AstraZeneca Clinical Study Information Center 📞 1-877-240-9479 ✉ information.center@astrazeneca.com
Participating sites (9)
- Research SiteRecruiting
Beijing, 100034 · China
- Research SiteRecruiting
Beijing, 100070 · China
- Research SiteRecruiting
Beijing, CN-100730 · China
- Research SiteRecruiting
Changchun, 130021 · China
- Research SiteRecruiting
Guangzhou, 510100 · China
- Research SiteNot Yet Recruiting
Hangzhou, 310003 · China
- Research SiteRecruiting
Suzhou, 215006 · China
- Research SiteRecruiting
Wenzhou, 325000 · China
- Research SiteNot Yet Recruiting
Wuhan, 430022 · China
Terms used on this page · explanations by MedicalUpdates.ai
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- CAR T
- A treatment in which a person's own immune cells (T cells) are collected, modified in a laboratory to recognise a target, and returned to the body.
- relapsed/refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- dFLC
- The difference between the abnormal ('involved') and normal ('uninvolved') free light chain levels in the blood. Used in some conditions to decide whether disease is measurable and to track response.
- Relapsed
- A condition that improved with treatment and has since returned or worsened.
- ECOG performance status
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- dose escalation
- Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
- Prior treatment
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- bispecific
- An engineered antibody that attaches to two targets at once, often linking an immune cell to a diseased cell so the immune cell can attack it.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- RP2D
- The dose chosen at the end of a phase 1 study for testing in later studies, based on safety and other measurements.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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