Engineered immune cells take on rare protein disease in major trial

NCT ID NCT07709715

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 16, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This phase 3 trial compares an experimental CAR-T cell therapy, NXC-201, with a standard drug combination (daratumumab plus CyBorD) in people newly diagnosed with AL amyloidosis, a rare condition where abnormal proteins damage organs. Participants receive either a single infusion of their own genetically modified immune cells or a course of standard medications. The study measures how well each approach controls the disease and prevents organ deterioration.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
a CAR-T cell therapy called NXC-201, made from the patient's own immune cells
What this could lead to
If successful, NXC-201 could offer a more effective, one-time treatment option for people newly diagnosed with AL amyloidosis, potentially improving organ function and survival.
What could go wrong
This is an early-stage comparison against an existing therapy, and CAR-T therapies carry risks like cytokine release syndrome and neurological side effects. Long-term outcomes are not yet known.

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

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 260 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jan 2027

An estimate. Start dates often move.

Expected to finish

Dec 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: 1. Eastern Cooperative Oncology Group performance status score ≤2 2. Newly diagnosed AL Amyloidosis per consensus criteria as defined in the protocol 3. Measurable disease as defined in the protocol 4. One or more organs impacted by AL amyloidosis Key Exclusion Criteria: 1. Prior therapy for AL amyloidosis or multiple myeloma 2. Diagnosis of multiple myeloma 3. Cardiac stage IIIb patients 4. History of malignancy (other than AL amyloidosis) within 3 years 5. Stroke or seizure within 6 months of signing ICF NOTE: Other protocol defined inclusion/exclusion criteria apply

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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.

How to take part

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  1. The official record

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