Venetoclax for Systemic Light Chain Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
University of California, San Francisco, San Francisco, CA
Systemic Light Chain Disease+4 More
Venetoclax - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This phase I/II trial tests the safety, side effects, and best dose of venetoclax, daratumumab, and dexamethasone for the treatment of systemic light-chain amyloidosis in patients with a deoxyribonucleic acid (DNA) abnormality called a translocation involving chromosomes 11 and 14, or "t(11;14)". Venetoclax works by attaching to a protein called Bcl-2, in order to kill cancer cells. Daratumumab works by binding to a target on the surface of cancer cells called CD38. When daratumumab binds to CD38, it enables the immune system to find the cancer cell and kill it. Dexamethasone is a type of drug called a corticosteroid. A corticosteroid is a drug made of artificial steroid hormones, that are used to treat symptoms such as inflammation (swelling and irritation to a part of the body). The combination of these medications may more effectively treat patients with systemic light-chain amyloidosis and t(11;14).

Eligible Conditions

  • Systemic Light Chain Disease
  • Light Chain (AL) Amyloidosis
  • AL Amyloidosis

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Systemic Light Chain Disease

Study Objectives

5 Primary · 14 Secondary · Reporting Duration: Up to 2 years

Day 28
Maximum tolerated dose (MTD) (Phase 1a)
Proportion of participants with reported dose limiting toxicities (Phase 1a)
Recommended phase 2 dose (RP2D) (Phase1a)
Up to 2 years
Median Duration of response (DOR) (Phase 1b/2)
Median Major Organ Deterioration Progression-Free Survival (MOD-PFS) (Phase 1b/2)
Median Overall Survival (OS) (Phase 1b/2)
Median Progression-Free Survival (PFS) (Phase 1b/2)
Median Time To Next Treatment (TTNT) (Phase 1b/2)
Number of treatment-related mortalities (Phase 2)
Organ response rate (orRR) (Phase 1b/2)
Overall hematologic response rate (ORR) (Phase 1b/2)
Overall number of participant deaths (Phase 2)
Percentage of participants with a cardiac event (Phase 2)
Percentage of participants with treatment-emergent adverse events attributable to study treatment (Phase 1a)
Percentage of participants with treatment-emergent adverse events attributable to study treatment (Phase 1b/2)
Proportion of participants who achieve a complete hematologic response (CHR) (Phase 2)
Proportion of participants with complete hematologic response (CHR) (Phase 1b)
Proportion of participants with infection (Phase 2)
Time to complete response (TTCR) (Phase 1b/2)

Trial Safety

Safety Progress

1 of 3

Other trials for Systemic Light Chain Disease

Trial Design

7 Treatment Groups

Phase 2: Arm A (venetoclax, dexamethasone, daratumumab)
1 of 7
Phase 1a: Dose Escalation Level 4 (venetoclax, dexamethasone, daratumumab)
1 of 7
Phase 1a: Dose Escalation Level 2 (venetoclax)
1 of 7
Phase 1a: Dose Escalation Level 3 (venetoclax, dexamethasone)
1 of 7
Phase 1a: Dose Escalation Level 1 (venetoclax)
1 of 7
Phase 1b: Dose Expansion (venetoclax, dexamethasone, daratumumab)
1 of 7
Phase 2: Arm B (dexamethasone, daratumumab)
1 of 7
Experimental Treatment

78 Total Participants · 7 Treatment Groups

Primary Treatment: Venetoclax · No Placebo Group · Phase 1 & 2

Phase 2: Arm A (venetoclax, dexamethasone, daratumumab)Experimental Group · 3 Interventions: Dexamethasone, Daratumumab, Venetoclax · Intervention Types: Drug, Drug, Drug
Phase 1a: Dose Escalation Level 4 (venetoclax, dexamethasone, daratumumab)Experimental Group · 3 Interventions: Dexamethasone, Daratumumab, Venetoclax · Intervention Types: Drug, Drug, Drug
Phase 1a: Dose Escalation Level 2 (venetoclax)
Drug
Experimental Group · 1 Intervention: Venetoclax · Intervention Types: Drug
Phase 1a: Dose Escalation Level 3 (venetoclax, dexamethasone)Experimental Group · 2 Interventions: Dexamethasone, Venetoclax · Intervention Types: Drug, Drug
Phase 1a: Dose Escalation Level 1 (venetoclax)
Drug
Experimental Group · 1 Intervention: Venetoclax · Intervention Types: Drug
Phase 1b: Dose Expansion (venetoclax, dexamethasone, daratumumab)Experimental Group · 3 Interventions: Dexamethasone, Daratumumab, Venetoclax · Intervention Types: Drug, Drug, Drug
Phase 2: Arm B (dexamethasone, daratumumab)Experimental Group · 2 Interventions: Dexamethasone, Daratumumab · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Dexamethasone
2007
Completed Phase 4
~2420
Daratumumab
2016
Completed Phase 4
~1400
Venetoclax
2019
Completed Phase 3
~1670

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years
Closest Location: University of California, San Francisco · San Francisco, CA
Photo of San Francisco  1Photo of San Francisco  2Photo of San Francisco  3
2008First Recorded Clinical Trial
1 TrialsResearching Systemic Light Chain Disease
688 CompletedClinical Trials

Eligibility Criteria

Age 18+ · All Participants · 8 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
Presence of t(11;14) on BMPC by FISH, performed at the University of California San Francisco (UCSF) cytogenetics laboratory.
The patient has received daratumumab and relapsed.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.