Relapsed AL AmyloidosisRefractory AL AmyloidosisLight Chain AmyloidosisAmyloidosis
Investigational drug(s) / intervention(s)
AZD0120
AZD0120: Participants will receive AZD0120 via intravenous (IV) infusion.
Study 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
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 or refractory disease with a need for additional therapy after at least 1 line of anti-plasma cell-directed therapy.
* ECOG performance status of 0 to 2
* 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 and/or urine pregnancy test result prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use highly 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)
Primary outcome measure(s)
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 Achieving Complete Response — Through study completion, a minimum of 6 months
Trial sites (18)
Facility
City
Region
Status
Research Site
Phoenix
Arizona
Recruiting
Research Site
San Francisco
California
Recruiting
Research Site
Tampa
Florida
Recruiting
Research Site
Boston
Massachusetts
Recruiting
Research Site
Detroit
Michigan
Recruiting
Research Site
Rochester
Minnesota
Recruiting
Research Site
St Louis
Missouri
Recruiting
Research Site
New York
New York
Recruiting
Research Site
New York
New York
Recruiting
Research Site
New York
New York
Recruiting
Research Site
Cleveland
Ohio
Recruiting
Research Site
Nashville
Tennessee
Recruiting
Research Site
Calgary
Alberta
Withdrawn
Research Site
Calgary
Alberta
Recruiting
Research Site
Toronto
Ontario
Withdrawn
Research Site
Toronto
Ontario
Recruiting
Research Site
London
United Kingdom
Withdrawn
Research Site
London
United Kingdom
Recruiting
More Alexion Pharmaceuticals, Inc. trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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