Relapsed/Refractory AL Amyloidosis
Conditions
Keywords
AZD0120, GC012F, Amyloidosis, AL Amyloidosis, CAR-T, Cell Therapy
Brief 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.
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.
Interventions
DRUGGC012F Injection
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.
Sponsors
Gracell Biotechnologies (Shanghai) Co., Ltd.
Suzhou Gracell Biotechnologies Co., Ltd.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Confirmed histopathological diagnosis of AL amyloidosis 2. One or more organs currently or historically impacted by AL amyloidosis according to consensus guidelines 3. Measurable hematologic disease: dFLC \> 20 mg/L or serum M-protein \> 5g/L 4. Relapsed disease or refractory disease defined as a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy. 5. ECOG performance status of 0 to 1 6. Must be able and willing to adhere to the study visit schedule and other protocol requirements 7. 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
1. Have any other form of amyloidosis other than AL amyloidosis 2. Mayo Stage IIIb AL amyloidosis 3. Oxygen saturation \< 95% on room air 4. Systolic blood pressure \<100mmHg 5. Cardiac
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With incidence and severity of Treatment-emergent Adverse Events | Through study completion, an average of 2 years |
| Number of participants with dose-limiting toxicities during dose escalation phase | Through study completion, an average of 2 years |
Secondary
| Measure | Time frame |
|---|---|
| Levels of GC012F in blood over time in participants with AL amyloidosis | 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) | Through study completion, an average of 2 years |
| Percentage of participants who achieve a complete response based on hematologic response criteria for AL amyloidosis | Through study completion, an average of 2 years |
Countries
China
Contacts
CONTACTAstraZeneca Clinical Study Information Center
Outcome results
None listed