Relapsed AL Amyloidosis, Refractory AL Amyloidosis, Light Chain Amyloidosis, Amyloidosis
Conditions
Keywords
AZD0120, Amyloidosis, AL Amyloidosis, CAR-T, Cell Therapy
Brief summary
Open-label Phase 1b/2 study with primary objective of this study is to evaluate the safety, tolerability and efficacy of AZD0120 in participants with light chain (AL) amyloidosis.
Interventions
DRUGAZD0120
Participants will receive AZD0120 via intravenous (IV) infusion.
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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 * NYHA class III or IV * Extensive GI involvement with evidence of active GI bleeding/risk of bleeding as determined by Investigator * Prior therapies: 1. CAR T cell therapy directed at any target 2. Prior BCMA-targeting therapy 3. Prior treatment with any FDA 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 * Symptomatic multiple myeloma (defined as clonal bone marrow plasma cells ≥10% plus at least one myeloma-defining event per IMWG 2014)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Phase 1b: Number of Participants With incidence and severity of Treatment-emergent Adverse Events | Through study completion, a minimum of 6 months |
| Phase 2: Proportion of Participants Experiencing a Complete Response | Through study completion, a minimum of 6 months |
Secondary
| Measure | Time frame |
|---|---|
| Phase 1b: Levels of AZD0120 in blood over time in participants with AL amyloidosis | Through study completion, a minimum of 6 months |
| Phase 2: Percentage of participants achieving hematologic response | Through study completion, a minimum of 6 months |
Countries
Canada, United Kingdom, United States
Contacts
CONTACTAlexion Pharmaceuticals, Inc. (Sponsor)
Outcome results
None listed