ClinicalTrials.gov record NCT04210791

Autologous Stem Cell Transplantation for Patients With AL Amyloidosis

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.The registry record was last updated on Feb 26, 2024, about 2 years ago, so the status shown may be out of date. The study team or the registry can confirm.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

Age18 years old to 75 years old

SexAll

LocationChina

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study looks at outcomes after autologous stem cell transplantation (ASCT) for AL amyloidosis. It has a retrospective part analyzing patients treated with ASCT from July 2010–December 2019 and a prospective part that will observe ASCT treatment protocols. The prospective work includes induction with bortezomib plus daratumumab before ASCT and lenalidomide as maintenance afterward. The study aims to examine safety, response, long-term outcomes, and factors linked to prognosis.

What it is studying. The study is testing how patients with AL amyloidosis do after autologous stem cell transplantation: overall survival and progression-free survival, blood and organ response rates, relapse and minimal residual disease outcomes, and which induction or maintenance approaches and patient factors relate to better or worse outcomes. The prospective portion will observe specific protocols (bortezomib + daratumumab induction; lenalidomide maintenance) and options used if disease returns.

What the researchers hope to learn. To describe the safety and long-term outcomes after ASCT for AL amyloidosis; to compare outcomes by different induction and maintenance regimens; to identify prognostic factors (including plasma cell test results and degree of organ involvement); and to inform which ASCT protocols warrant further study.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This study mainly evaluated the efficacy and safety of autologous stem cell transplantation for the treatment of AL amyloidosis, the role of induction and maintenance therapy in autologous stem cell transplantation, and the long-term efficacy and prognosis risk factors of autologous stem cell transplantation for the treatment of AL amyloidosis.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

Male or female;
aged 18-75 years;
Patients with newly diagnosed AL;
Appropriate for autologous hematopoietic stem cell transplantation;
Abnormal M protein or free light chain detected in serum and/or urine
ECOG score 0-2 points;
Subjects (or their legal representatives) must sign an informed consent document indicating understanding the purpose of and procedures required for the study and willingness to participate in the study.

Exclusion criteria

Pregnant and breastfeeding women;
Subjects suffering from multiple myeloma;
hypersensitivity to any treatment drugs;
Subjects have severe cardiovascular disease;
Subjects have a serious physical disease and mental illnesses;
Other conditions that researchers consider are not suitable for transplantation.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

This study is divided into two parts. In the first part, the investigators retrospectively analyze the data of patients with AL amyloidosis who treated with autologous stem cell transplantation from July 2010 to December 2019. All patients had a biopsy-proven disease by positive Congo red stain with a concomitant demonstration of plasma cell dyscrasia. Organ involvement was established according to the criteria established at the 10th International Symposium on Amyloid and Amyloidosis. The protocol of ASCT included mobilization with colony-stimulating factor alone and conditioning with high-dose melphalan 140 or 200 mg/m2. In addition to analyzing the efficacy and safety of all the patients, the investigators also analyzed the difference in efficacy between patients in different subgroups. For example, subgroups divided according to different induction treatment regimens; subgroups divided according to different plasma cell FISH data and FCM data; subgroups divided according to the degree of organ involvement, and subgroups divided according to different maintenance treatment regimens. In the second part of the study, the investigators will conduct a prospective study to explore the best autologous stem cell transplantation treatment protocol for AL amyloidosis. The protocol includes the induction therapy with bortezomib and daratumumab before ASCT, the maintenance treatment with lenalidomide after ASCT, and the treatment options for recurrence after transplantation.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

The study includes a retrospective part (review of past medical records) and a prospective part that involves treatment and follow-up. Prospective participants may receive induction therapy with bortezomib and daratumumab, stem‑cell mobilization with colony‑stimulating factor, conditioning with high‑dose melphalan (140 or 200 mg/m2), and autologous stem cell transplantation, followed by possible lenalidomide maintenance. Participants will have periodic assessments and follow-up for outcomes such as overall survival, progression‑free survival, hematologic response, organ response, and relapse over up to five years.

How the study is structured. An observational cohort study in two parts. First, a retrospective review of people with biopsy‑proven AL amyloidosis who received ASCT between July 2010 and December 2019. Second, a prospective observational component that follows patients treated with specified induction and maintenance approaches after ASCT. The study collects clinical outcomes and compares subgroups (for example by induction or maintenance regimen and by plasma cell test results). Target follow-up is five years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The registry says participants are enrolled by invitation. Who decides who is invited, and how?
The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on AL amyloidosis

Clinical trials69 trials listed for AL amyloidosisEvery trial the registry lists for AL amyloidosis, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in AL amyloidosis researchLatest monthly update: September 2026. Includes: Daratumumab alone. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Jul 1, 2010
Primary completion: Dec 31, 2025 (estimated)
Study completion: Dec 31, 2030 (estimated)
Target enrollment: 500 participants (estimated)
Last updated on registry: Feb 26, 2024
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: Nanjing University School of Medicine
Principal investigator: Zhi-Hong Liu, MD, Professor
Nanjing University School of Medicine

Participating sites (1)

National Clinical Research Center of Kidney Diseases, Jinling Hospital
Nanjing, Jiangsu, 210016 · China

Terms used on this page · explanations by MedicalUpdates.ai

free light chain: Small pieces of antibody protein made by plasma cells and measured in a blood test. In some conditions, such as AL amyloidosis, abnormal free light chains are the source of the disease.
ECOG score: A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
biopsy-proven: Removal of a small piece of tissue so it can be examined under a microscope. 'Biopsy-proven' or 'histologically confirmed' means the diagnosis was confirmed by examining tissue under a microscope.
ASCT: A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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