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A Trial to Learn if Linvoseltamab is Safe and Works in Adults With Relapsed or Refractory Systemic Light Chain Amyloidosis (AL Amyloidosis)

Phase 1
Recruiting
Conditions
Relapsed/Refractory Systemic Light Chain Amyloidosis
Interventions
Registration Number
NCT06292780
Lead Sponsor
Regeneron Pharmaceuticals
Brief Summary

This study is researching an experimental drug called linvoseltamab ("study drug").

This study is focused on patients who have AL amyloidosis that has returned or have failed other therapies and need to be treated again.

The study consists of 2 phases (Phase 1 and Phase 2):

* In Phase 1, linvoseltamab will be given to a small number of participants to study the side effects of the study drug and to determine the recommended doses of the study drug to be given to participants in Phase 2.

* In Phase 2, linvoseltamab will be given to more participants to continue to assess the side effects of the study drug and to evaluate the ability of linvoseltamab to treat AL amyloidosis.

The study is looking at several other research questions, including:

* How many participants treated with linvoseltamab have improvement in the abnormal proteins that cause organ problems and for how long

* How many participants treated with linvoseltamab have improvement in the heart or kidney and for how long

* What the right dosing regimen is for linvoseltamab

* What side effects may happen from taking linvoseltamab

* How much linvoseltamab is in the blood at different times

* Whether the body makes antibodies against linvoseltamab (which could make the drug less effective or could lead to side effects)

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
220
Inclusion Criteria
  1. Confirmed diagnosis of AL amyloidosis, as described in the protocol
  2. Measurable disease as defined by serum difference between involved and uninvolved free light chains (dFLC) concentration, as described in the protocol
  3. Previously treated after at least 1 prior therapy and requiring further treatment as assessed by the Investigator
  4. N-terminal pro b-type natriuretic peptide (NT-proBNP) ≤8500 ng/L during screening
  5. Adequate hepatic, hematologic, renal, and cardiac function, as described in the protocol
  6. Eastern Cooperative Oncology Group (ECOG) performance score ≤2 at screening

Key

Exclusion Criteria
  1. History of other non-AL amyloidosis
  2. Greater than 60% plasmacytosis on a bone marrow biopsy and/or aspirate during screening
  3. Presence of lytic bone lesion(s) or extramedullary plasmacytoma on imaging during screening
  4. Myocardial infarction within the past 6 months prior to the first screening visit
  5. Known active infection requiring hospitalization or treatment with IV anti-infectives within 28 days of first administration of study drug

NOTE: Other protocol defined inclusion/exclusion criteria apply

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Phase 1: Cohort 2: High DoseLinvoseltamabDose Escalation: Non-Randomized
Phase 2: Low DoseLinvoseltamabDose Expansion: Participants will be randomized in a 1:1 ratio
Phase 1: Cohort 1: Low DoseLinvoseltamabDose Escalation: Non-Randomized
Phase 2: High DoseLinvoseltamabDose Expansion: Participants will be randomized in a 1:1 ratio
Primary Outcome Measures
NameTimeMethod
Incidence of dose-limiting toxicity (DLTs)Up to 28 Days

Phase 1

Achievement of hematologic complete response (CR) as determined by the Independent Review Committee (IRC)Up to 3 years

Phase 2

Secondary Outcome Measures
NameTimeMethod
Time to first renal response in participants with renal involvement at baselineUp to 7 years
Severity of TEAEsUp to 39 months
Time from treatment initiation to kidney deterioration as determined by the IRCUp to 7 years
Time to initial hematologic responseUp to 3 years
Hematologic progression-free survival (PFS)Up to 7 years
Incidence of serious adverse events (SAEs)Up to 39 months
Severity of AESIsUp to 39 months
Time from treatment initiation to hematologic disease progression as determined by the IRCUp to 7 years
Time from treatment initiation to death as determined by the IRCUp to 7 years
Achievement of cardiac response in participants with cardiac involvement at baseline, as determined by IRCUp to 7 years
Time to best hematologic responseUp to 3 years
Incidence of treatment-emergent adverse events (TEAEs)Up to 39 months
Achievement of overall hematologic response (PR or better), as determined by the IRC in dose regimen 1 vs 2Up to 39 months

Phase 2

Achievement of renal response in participants with renal involvement at baseline, as determined by IRCUp to 7 years
Linvoseltamab concentration in serum over timeUp to 39 months
Incidence of anti-drug antibodies (ADAs) to linvoseltamab over timeUp to 39 months
Achievement of hematologic very good partial response (VGPR) or better response (CR + VGPR), as determined by the IRCUp to 3 years
Achievement of overall hematologic response (PR or better), as determined by the IRCUp to 3 years
Duration of hematologic response (ie, best response, VGPR or better, overall response), as determined by the IRCUp to 7 years
Severity of SAEsUp to 39 months
Incidence of TEAEs in dose regimen 1 vs 2Up to 39 months

Phase 2

Severity of TEAEs in dose regimen 1 vs 2Up to 39 months

Phase 2

Severity of SAEs in dose regimen 1 vs 2Up to 39 months

Phase 2

Incidence of AESIs in dose regimen 1 vs 2Up to 39 months

Phase 2

Titers of ADAs to linvoseltamab over timeUp to 39 months
Achievement of hematologic CR, as determined by the IRCUp to 3 years

Phase 1

Incidence of deathUp to 7 years
Incidence of adverse events of special interest (AESIs)Up to 39 months
Incidence of SAEs in dose regimen 1 vs 2Up to 39 months

Phase 2

Severity of AESIs in dose regimen 1 vs 2Up to 39 months

Phase 2

Time from treatment initiation to cardiac deterioration, as determined by the IRCUp to 7 years
Time from initiation of treatment to date of death from any causeUp to 7 years
Time to first cardiac response in participants with cardiac involvement at baselineUp to 7 years

Trial Locations

Locations (16)

Colorado Blood Cancer Institute/SCRI

🇺🇸

Denver, Colorado, United States

City of Hope

🇺🇸

Duarte, California, United States

Karmanos Cancer Institute

🇺🇸

Detroit, Michigan, United States

Roswell Park Comprehensive Cancer Center

🇺🇸

Buffalo, New York, United States

MD Anderson Cancer Center

🇺🇸

Houston, Texas, United States

Seoul National University Hospital

🇰🇷

Seoul, Korea, Republic of

Asan Medical Center

🇰🇷

Seoul, Korea, Republic of

Samsung Medical Center

🇰🇷

Seoul, Korea, Republic of

Seoul St. Mary's Hospital - The Catholic University of Korea

🇰🇷

Seoul, Korea, Republic of

Hospital de Cabuenes

🇪🇸

Gijon, Asturias, Spain

Hospital Universitari Son Espases

🇪🇸

Palma, Balearic Islands, Spain

Clinica Universidad de Navarra

🇪🇸

Pamplona, Navarra, Spain

Universität de Barcelona- Institut d' Investigacions Biomediques August Pi i Sunyer (IDIBAPS)

🇪🇸

Barcelona, Spain

University Hospital La Fe

🇪🇸

Valencia, Spain

University College London Hospitals

🇬🇧

London, United Kingdom

The Christie NHS Foundation Trust

🇬🇧

Manchester, United Kingdom

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