AL AmyloidosisPhase 1/2NCT05451771
Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Amyloidosis
A Phase 1/2 study testing Venetoclax Oral Tablet, 200 mg, FISH assay and Venetoclax Oral Tablet, 400 mg in people with AL Amyloidosis, run by Columbia University at 4 sites in United States.
Trial summary
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- AL Amyloidosis
- What is being tested?
- Venetoclax Oral Tablet, 200 mg, FISH assay and Venetoclax Oral Tablet, 400 mg
- Type of treatment
- Pill or oral medicine, Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 4 sitesUnited States
- Study dates
- 26 Oct 2022to Sep 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Confirmed diagnosis of systemic AL amyloidosis by mass spectrometry or immunohistochemistry (IHC) on a tissue biopsy
- Has received ≥1 prior lines of therapy, including an anti-cluster of differentiation 38 (CD 38) monoclonal antibody
- Participants with a history of autologous hematopoietic cell transplantation must have recovered from any transplant-related toxicities
- Presence of t(11;14) on FISH at any time since diagnosis (Eligibility must confirmed by FISH testing at Columbia University Irving Medical Center (CUIMC)
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Who cannot join
- Known hypersensitivity to any of the study drugs
- History of other malignancy that could affect compliance with the protocol or interpretation of results (Patients with a history of curatively treated basal or squamous cell carcinoma of the skin, in situ carcinoma of the cervix, breast cancer, or Hodgkin's Lymphoma are generally eligible. Patients with a malignancy that has been treated, but not with curative intent, will be excluded, unless the malignancy has been in remission without treatment for ≥ 2 years prior to enrollment.)
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to, uncontrolled systemic infection (viral, bacterial, or fungal)
- Patients on renal replacement therapy
- Known GI disease or GI procedure that could interfere with oral absorption (including difficulty swallowing)
- New York Heart Association (NYHA) Class III or IV heart failure
Show all 10Show fewer
- Mayo stage three-B (IIIB) with N-terminal pro-hormone B-type natriuretic peptide (NT-Pro BNP) > 8500 pg/mL
- Prior exposure to anti-apoptotic protein B-cell lymphoma 2 (BCL-2) inhibitors
- Patients with human immunodeficiency virus (HIV) who are not on highly active antiretroviral therapy (HAART) or those with active hepatitis A, B, or C infection
- Patients meeting criteria for symptomatic multiple myeloma by one of the following:(a) Lytic lesions on imaging (b) Plasmacytoma, (c) Hypercalcemia without any alternate etiology, or (c) Bone marrow plasma cell infiltrate of greater than 60%
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Boston Medical Center
Boston, Massachusetts, 02118
Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
New York Presbyterian Hospital/Columbia University Irving Medical Center
New York, New York, 10032
Mayo Clinic Rochester
Rochester, Minnesota, 55905
About this study
Official title: An Open-label Phase I/II Trial of Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Systemic Light-Chain Amyloidosis
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis.
Read the detailed description
This study is a phase 1/2 study of venetoclax-dexamethasone combination therapy in relapsed/refractory t(11;14) systemic immunoglobulin light chain amyloidosis (AL) amyloidosis. The phase 1 is a dose escalation designed to determine the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D) of venetoclax in combination with low-dose weekly dexamethasone. There will be four candidate-dosing cohorts of venetoclax with or without dexamethasone in the Phase I dose-escalation. Dose escalation will be guided by the Bayesian optimal interval (BOIN) design with accelerated titration up to a total sample size of 15 participants.
The phase 2 portion is a randomized open-label study comparing the MTD or RP2D of venetoclax in combination with dexamethasone versus investigator's choice (daratumumab, pomalidomide, bendamustine, or ixazomib (with or without dexamethasone).
- Conditions
- AL Amyloidosis
- Being tested
- Venetoclax Oral Tablet, 200 mg, FISH assay, Venetoclax Oral Tablet, 400 mg, Dexamethasone Oral, 10 mg, Dexamethasone Oral, 20 mg
- Keywords
- Light Chain Amyloidosis, Protein Misfolding Disorder
- Medical subject headings
- Immunoglobulin Light-chain Amyloidosis, Proteostasis Deficiencies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Venetoclax Oral Tablet, 200 mgDrug
Also called Venclexta
200 mg oral tablet daily
FISH assayDevice
Also called t(11;14) FISH assay
Cytogenetic analysis is intended for evaluation of relapsed/refractory AL amyloidosis using fluorescence in situ hybridization (FISH) using known translocation probes. Bone marrow aspirate (BMA) samples are collected in lavender top (Ethylenediaminetetraacetic acid (EDTA)) or green top (Sodium heparin) tubes. Specimen tubes shall be transported at room temperature to the laboratory on the same day of collection.
Venetoclax Oral Tablet, 400 mgDrug
Also called Venclexta
400 mg oral tablet daily
Dexamethasone Oral, 10 mgDrug
Also called Decadron, Hemady
10 mg oral tablet weekly
Dexamethasone Oral, 20 mgDrug
Also called Decadron, Hemady
20 mg oral tablet weekly
Daratumumab InjectionDrug
Also called Darzalex
Daratumumab will be administered at a dose of 16 mg/kg by IV infusion once weekly for weeks 1 to 8, every 2 weeks for weeks 9 to 24, and every 4 weeks thereafter for a maximum of 6 months of therapy. If subcutaneous formulation is available, participants can also receive subcutaneous daratumumab (1800 mg in 15 ml) in the same schedule.
BendamustineDrug
Also called Treanda
Bendamustine will be given at an initial dose of 100 mg/m^2 intravenously on days 1 and 2 in each 28-day cycle.
PomalidomideDrug
Also called Pomalyst
Pomalidomide will be administered at an initial dose of 2 mg per days on days 1-21 every 28 days.
IxazomibDrug
Also called Ninlaro
Ixazomib will be administered at an initial dose of 4 mg per days on days 1, 8, and 15 every 28 days.
Venetoclax MTD with DexamethasoneDrug
Venetoclax MTD (200 mg or 400 mg) with Dexamethasone (10 mg or 20 mg) as determined by the phase I results
| Study group | Role | What they receive |
|---|---|---|
| Phase 1: Venetoclax 200 mg | Experimental | Cohort 1: Venetoclax 200 mg tablet, once daily for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) Receives: Drug: Venetoclax Oral Tablet, 200 mg; Device: FISH assay |
| Phase 1: Venetoclax 400mg | Experimental | Cohort 2: Venetoclax 400 mg tablet, once daily for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) Receives: Device: FISH assay; Drug: Venetoclax Oral Tablet, 400 mg |
| Phase 1: Venetoclax 400mg + Dexamethasone 10 mg | Experimental | Cohort 3: Venetoclax 400 mg tablet, once daily and Dexamethasone 10 mg tablet once weekly, for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) Receives: Device: FISH assay; Drug: Venetoclax Oral Tablet, 400 mg; Drug: Dexamethasone Oral, 10 mg |
| Phase 1: Venetoclax 400mg + Dexamethasone 20 mg | Experimental | Cohort 4: Venetoclax 400 mg tablet, once daily and Dexamethasone 20 mg tablet once weekly, for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) Receives: Device: FISH assay; Drug: Venetoclax Oral Tablet, 400 mg; Drug: Dexamethasone Oral, 20 mg |
| Phase 2: Venetoclax MTD with Dexamethasone | Experimental | Venetoclax MTD (200 mg or 400 mg) with Dexamethasone (10 mg or 20 mg) as determined by the phase I results Receives: Drug: Venetoclax MTD with Dexamethasone |
| Phase 2: Control Arm (Investigator's Choice) | Active comparator | Participants will receive one of the following as determined by the investigator:
Daratumumab, Pomalidomide, Bendamustine, or Ixazomib (+/- dexamethasone) Receives: Drug: Dexamethasone Oral, 20 mg; Drug: Daratumumab Injection; Drug: Bendamustine; Drug: Pomalidomide; Drug: Ixazomib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Dose Limiting Toxicities (DLT) (Phase 1)
The number of participants with dose limiting toxicities for each treatment dose will be used to determine the MTD. Dose limiting toxicity defined as grade 4 neutropenia lasting more than 5 days, any grade febrile neutropenia, grade 4 thrombocytopenia, grade 3 thrombocytopenia with bleeding, other therapy related non-hematologic toxicity of grade 2 or higher that requires discontinuation of therapy, clinical tumor lysis syndrome (TLS), laboratory TLS if the metabolic abnormalities are considered clinically significant by the investigator. All other grade 3 or higher adverse events (AEs) will be considered as DLTs with a few exceptions.
Measured: Up to 6 cycles (approximately 6 months)
Hematologic ≥ Very Good Partial Response (VGPR) Rate (Phase 2)
Hematologic ≥VGPR rate defined as proportion of participants achieving VGPR, low serum differential free light chain concentration (dFLC) partial response (PR), or a complete (CR).
VGPR is defined as the difference between involved and uninvolved free light chain (FLC) [dFLC] < 40 mg/L. Low dFLC PR is defined as achieving a dFLC<10 mg/L, low dFLC PR will be considered as a deep hematologic response and included in the ≥VGPR category). CR is defined as negative serum and urine immunofixation electrophoresis along with a serum free light chain ratio that lies within the normal range or skewed towards the non-amyloid forming light chain, as per institutional laboratory values
Measured: Up to 6 cycles (approximately 6 months)
Secondary outcomes (9)
Overall Organ Response Rate (ORR) (Phase 2)
ORR defined as proportion of evaluable participants achieving organ response in each involved organ
Measured: Up to 1 year
Progression Free Survival (PFS) (Phase 2)
PFS defined as time from the date of enrollment to hematologic progression or death, whichever is earlier
Measured: Up to 1 year
Overall Hematologic Response Rate (HRR) (Phase 2)
HRR defined as proportion of patients achieving partial response (PR) or better
Measured: Up to 1 year
Duration of Hematologic Response (DOHR) (Phase 2)
DOHR defined as time from the date of first documentation of hematologic response to the date of first documented hematologic disease progression
Measured: Up to 1 year
Time to hematologic ≥VGPR (Phase 2)
Time to hematologic ≥VGPR defined as time from the first dose of study treatment to achievement of deep hematologic response (VGPR, low dFLC PR, or a CR)
Measured: Up to 1 year
Time to next treatment (TTNT) (Phase 2)
TTNT defined as time from the date of enrollment to initiation of a subsequent line of therapy
Measured: Up to 1 year
Major Organ Deterioration-Progression Free Survival (MOD-PFS) (Phase 2)
MOD-PFS defined as time from the date of enrollment to one of the following events (whichever occurs first): death, clinical manifestation of cardiac/renal failure, or development of hematologic progression of disease
Measured: Up to 1 year
Overall Survival (OS) (Phase 2)
OS defined as the time from the date of enrollment to death from any cause
Measured: Up to 1 year
Patient-reported outcomes (PROs) (Phase 2)
PROs defined as longitudinal score of "Physical Functioning" domain of Patient-Reported Outcomes Measurement Information System (PROMIS)-29 Profile v2.0 The Physical Functioning domain contains four items with a 1 (unable to do) to 5 (without any difficulty) numeric rating.
Measured: Start of each cycle (Day 1 of each 28 day cycle) and Follow-up (every 8 weeks for up to 1 year)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2022
- Study start26 Oct 2022
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for AL AmyloidosisStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 53 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Principal investigator, Rajshekhar Chakraborty, MD, Columbia University
- Investigators
- Rajshekhar Chakraborty, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05451771
- Sponsor's protocol ID
- AAAT8639
- Status last verified
- Oct 2026
- First submitted
- 6 Jul 2022
- Last updated
- 8 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
Publications
Locke M, Nieto M. AL Amyloidosis: Current Treatment and Outcomes. Adv Hematol. 2025 Mar 3;2025:7280805. doi: 10.1155/ah/7280805. eCollection 2025.
PubMed 40226119
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Phase I/II Trial of Venetoclax-Dexamethasone in Relapsed and/or Refractory t(11;14) Systemic Light-Chain Amyloidosis
- ClinicalTrials.gov ID
- NCT05451771
- Acronym / sponsor ID
- AAAT8639
- Conditions
- AL Amyloidosis
- Keywords
- Light Chain Amyloidosis, Protein Misfolding Disorder
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 53 participants (estimated)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 26 Oct 2022 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 11 Jul 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is assess safety, safest dose, and effectiveness of venetoclax in combination with dexamethasone in participants with t(11;14) positive relapsed (comes back) or refractory (did not get better) light chain amyloidosis.
Detailed description
This study is a phase 1/2 study of venetoclax-dexamethasone combination therapy in relapsed/refractory t(11;14) systemic immunoglobulin light chain amyloidosis (AL) amyloidosis. The phase 1 is a dose escalation designed to determine the maximum tolerated dose (MTD) and the recommended phase 2 dose (RP2D) of venetoclax in combination with low-dose weekly dexamethasone. There will be four candidate-dosing cohorts of venetoclax with or without dexamethasone in the Phase I dose-escalation. Dose escalation will be guided by the Bayesian optimal interval (BOIN) design with accelerated titration up to a total sample size of 15 participants.
The phase 2 portion is a randomized open-label study comparing the MTD or RP2D of venetoclax in combination with dexamethasone versus investigator's choice (daratumumab, pomalidomide, bendamustine, or ixazomib (with or without dexamethasone).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
| Arm | Type | Receives |
|---|---|---|
| Phase 1: Venetoclax 200 mg | Experimental | Drug: Venetoclax Oral Tablet, 200 mg, Device: FISH assayCohort 1: Venetoclax 200 mg tablet, once daily for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) |
| Phase 1: Venetoclax 400mg | Experimental | Device: FISH assay, Drug: Venetoclax Oral Tablet, 400 mgCohort 2: Venetoclax 400 mg tablet, once daily for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) |
| Phase 1: Venetoclax 400mg + Dexamethasone 10 mg | Experimental | Device: FISH assay, Drug: Venetoclax Oral Tablet, 400 mg, Drug: Dexamethasone Oral, 10 mgCohort 3: Venetoclax 400 mg tablet, once daily and Dexamethasone 10 mg tablet once weekly, for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) |
| Phase 1: Venetoclax 400mg + Dexamethasone 20 mg | Experimental | Device: FISH assay, Drug: Venetoclax Oral Tablet, 400 mg, Drug: Dexamethasone Oral, 20 mgCohort 4: Venetoclax 400 mg tablet, once daily and Dexamethasone 20 mg tablet once weekly, for up to 2 cycles after achieving best response, for a maximum of 6 cycles (1 cycle = 28 days) |
| Phase 2: Venetoclax MTD with Dexamethasone | Experimental | Drug: Venetoclax MTD with DexamethasoneVenetoclax MTD (200 mg or 400 mg) with Dexamethasone (10 mg or 20 mg) as determined by the phase I results |
| Phase 2: Control Arm (Investigator's Choice) | Active comparator | Drug: Dexamethasone Oral, 20 mg, Drug: Daratumumab Injection, Drug: Bendamustine, Drug: Pomalidomide, Drug: IxazomibParticipants will receive one of the following as determined by the investigator: Daratumumab, Pomalidomide, Bendamustine, or Ixazomib (+/- dexamethasone) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age ≥ 18 years at time of signing Informed Consent Form
- Ability to comply with the study protocol, in the investigator's judgment
- Confirmed diagnosis of systemic AL amyloidosis by mass spectrometry or immunohistochemistry (IHC) on a tissue biopsy
- Has received ≥1 prior lines of therapy, including an anti-cluster of differentiation 38 (CD 38) monoclonal antibody
- Participants with a history of autologous hematopoietic cell transplantation must have recovered from any transplant-related toxicities
Key exclusion criteria (10 listed)
- Known hypersensitivity to any of the study drugs
- History of other malignancy that could affect compliance with the protocol or interpretation of results (Patients with a history of curatively treated basal or squamous cell carcinoma of the skin, in situ carcinoma of the cervix, breast cancer, or Hodgkin's Lymphoma are generally eligible. Patients with a malignancy that has been treated, but not with curative intent, will be excluded, unless the malignancy has been in remission without treatment for ≥ 2 years prior to enrollment.)
- Evidence of other clinically significant uncontrolled condition(s) including, but not limited to, uncontrolled systemic infection (viral, bacterial, or fungal)
- Patients on renal replacement therapy
- Known GI disease or GI procedure that could interfere with oral absorption (including difficulty swallowing)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Venetoclax Oral Tablet, 200 mgAlso: Venclexta200 mg oral tablet daily | Drug | Phase 1: Venetoclax 200 mg |
| FISH assayAlso: t(11;14) FISH assayCytogenetic analysis is intended for evaluation of relapsed/refractory AL amyloidosis using fluorescence in situ hybridization (FISH) using known translocation probes. Bone marrow aspirate (BMA) samples are collected in lavender top (Ethylenediaminetetraacetic acid (EDTA)) or green top (Sodium heparin) tubes. Specimen tubes shall be transported at room temperature to the laboratory on the same day of collection. | Device | Phase 1: Venetoclax 200 mg, Phase 1: Venetoclax 400mg, Phase 1: Venetoclax 400mg + Dexamethasone 10 mg, Phase 1: Venetoclax 400mg + Dexamethasone 20 mg |
| Venetoclax Oral Tablet, 400 mgAlso: Venclexta400 mg oral tablet daily | Drug | Phase 1: Venetoclax 400mg, Phase 1: Venetoclax 400mg + Dexamethasone 10 mg, Phase 1: Venetoclax 400mg + Dexamethasone 20 mg |
| Dexamethasone Oral, 10 mgAlso: Decadron, Hemady10 mg oral tablet weekly | Drug | Phase 1: Venetoclax 400mg + Dexamethasone 10 mg |
| Dexamethasone Oral, 20 mgAlso: Decadron, Hemady20 mg oral tablet weekly | Drug | Phase 1: Venetoclax 400mg + Dexamethasone 20 mg, Phase 2: Control Arm (Investigator's Choice) |
| Daratumumab InjectionAlso: DarzalexDaratumumab will be administered at a dose of 16 mg/kg by IV infusion once weekly for weeks 1 to 8, every 2 weeks for weeks 9 to 24, and every 4 weeks thereafter for a maximum of 6 months of therapy. If subcutaneous formulation is available, participants can also receive subcutaneous daratumumab (1800 mg in 15 ml) in the same schedule. | Drug | Phase 2: Control Arm (Investigator's Choice) |
| BendamustineAlso: TreandaBendamustine will be given at an initial dose of 100 mg/m^2 intravenously on days 1 and 2 in each 28-day cycle. | Drug | Phase 2: Control Arm (Investigator's Choice) |
| PomalidomideAlso: PomalystPomalidomide will be administered at an initial dose of 2 mg per days on days 1-21 every 28 days. | Drug | Phase 2: Control Arm (Investigator's Choice) |
| IxazomibAlso: NinlaroIxazomib will be administered at an initial dose of 4 mg per days on days 1, 8, and 15 every 28 days. | Drug | Phase 2: Control Arm (Investigator's Choice) |
| Venetoclax MTD with DexamethasoneVenetoclax MTD (200 mg or 400 mg) with Dexamethasone (10 mg or 20 mg) as determined by the phase I results | Drug | Phase 2: Venetoclax MTD with Dexamethasone |
Primary outcome
Number of Participants with Dose Limiting Toxicities (DLT) (Phase 1)
Time frame: Up to 6 cycles (approximately 6 months)
The number of participants with dose limiting toxicities for each treatment dose will be used to determine the MTD. Dose limiting toxicity defined as grade 4 neutropenia lasting more than 5 days, any grade febrile neutropenia, grade 4 thrombocytopenia, grade 3 thrombocytopenia with bleeding, other therapy related non-hematologic toxicity of grade 2 or higher that requires discontinuation of therapy, clinical tumor lysis syndrome (TLS), laboratory TLS if the metabolic abnormalities are considered clinically significant by the investigator. All other grade 3 or higher adverse events (AEs) will be considered as DLTs with a few exceptions.
Hematologic ≥ Very Good Partial Response (VGPR) Rate (Phase 2)
Time frame: Up to 6 cycles (approximately 6 months)
Hematologic ≥VGPR rate defined as proportion of participants achieving VGPR, low serum differential free light chain concentration (dFLC) partial response (PR), or a complete (CR). VGPR is defined as the difference between involved and uninvolved free light chain (FLC) [dFLC] < 40 mg/L. Low dFLC PR is defined as achieving a dFLC<10 mg/L, low dFLC PR will be considered as a deep hematologic response and included in the ≥VGPR category). CR is defined as negative serum and urine immunofixation electrophoresis along with a serum free light chain ratio that lies within the normal range or skewed towards the non-amyloid forming light chain, as per institutional laboratory values
9 secondary outcomes
Overall Organ Response Rate (ORR) (Phase 2)
Time frame: Up to 1 year
Progression Free Survival (PFS) (Phase 2)
Time frame: Up to 1 year
Overall Hematologic Response Rate (HRR) (Phase 2)
Time frame: Up to 1 year
Duration of Hematologic Response (DOHR) (Phase 2)
Time frame: Up to 1 year
Time to hematologic ≥VGPR (Phase 2)
Time frame: Up to 1 year
Time to next treatment (TTNT) (Phase 2)
Time frame: Up to 1 year
Major Organ Deterioration-Progression Free Survival (MOD-PFS) (Phase 2)
Time frame: Up to 1 year
Overall Survival (OS) (Phase 2)
Time frame: Up to 1 year
Patient-reported outcomes (PROs) (Phase 2)
Time frame: Start of each cycle (Day 1 of each 28 day cycle) and Follow-up (every 8 weeks for up to 1 year)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2026.
- Derived from this study: PubMed 40226119Locke M, Nieto M. AL Amyloidosis: Current Treatment and Outcomes. Adv Hematol. 2025 Mar 3;2025:7280805. doi: 10.1155/ah/7280805. eCollection 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rajshekhar Chakraborty, MD principal investigator
Columbia University
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Venetoclax Oral Tablet, 200 mg, FISH assay and Venetoclax Oral Tablet, 400 mg in people with AL Amyloidosis, run by Columbia University at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 8 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants with Dose Limiting Toxicities (DLT) (Phase 1) (time frame: Up to 6 cycles (approximately 6 months)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05451771, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.