Promising drug combo targets Hard-to-Treat amyloidosis
NCT ID NCT05451771
First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time
Summary
This study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find the safest dose and see how well the drugs work at reducing abnormal proteins and improving organ function.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Venetoclax (Venclexta) and dexamethasone
- What this could lead to
- If successful, this could offer a new treatment option for people with a specific genetic type of AL amyloidosis that has not responded to prior therapy.
- What could go wrong
- This is an early-phase trial with only 53 participants, so results may not apply broadly. Side effects like low blood counts and tumor lysis syndrome are possible.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 53 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Oct 2022
- Expected to finish
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Sep 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * 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 Exclusion Criteria: * 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 * 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%
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Boston Medical Center
Boston, Massachusetts, 02118, United States
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Froedtert Hospital & the Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States
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New York Presbyterian Hospital/Columbia University Irving Medical Center
New York, New York, 10032, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a radioactive peptide spot hidden amyloid in the heart?
- Real-world data on multiple myeloma immunotherapies could reshape monitoring and improve survival
- Nationwide registry aims to crack the code of a rare protein-clogging disease
- New scan spots hidden organ damage before symptoms start
- Could a simple scan unlock better heart treatment?