Autologous Stem Cell Transplantation for Patients With AL Amyloidosis

February 22, 2024 updated by: Zhi-Hong Liu, MD, Nanjing University School of Medicine
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.

Study Overview

Status

Enrolling by invitation

Conditions

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All patients had an amyloid disease, which was confirmed by biopsy and documented plasma cell dyscrasia; the AL amyloidosis diagnosis and the assessment of organ involvement were based on previous consensus criteria. All patients had received autologous stem cell transplantation.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall survival
Time Frame: 5 years
the 5 years overall survival after autologous stem cell transplantation.
5 years
progression-free survival
Time Frame: 5 years
the 5 years progression-free survival after autologous stem cell transplantation.
5 years
hematological complete response rate
Time Frame: 1 year
the 1 year hematologic complete response rate after autologous stem cell transplantation.
1 year
organ response rate
Time Frame: 1 year
the 1 years organ response rate after autologous stem cell transplantation.
1 year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
hematological overall response rate
Time Frame: 1 year
the 1 years hematologic overall response rate after autologous stem cell transplantation.
1 year
the relapse rate of complete remission participants
Time Frame: 5 years
the relapse rate of complete remission participants after autologous stem cell transplantation.
5 years
the rate of Minimal Residual Disease-negative participants
Time Frame: 5 years
the rate of Minimal Residual Disease-negative participants after autologous stem cell transplantation.
5 years
the median time form stem cell transplantation to next treatment
Time Frame: 5 years
the median time form stem cell transplantation to next chemotherapy or antibody treatment.
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nanjing University School of Medicine

Investigators

  • Study Director: Zhihong Liu, MD, Nanjing University School of Medicine

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2010

Primary Completion (Estimated)

December 31, 2025

Study Completion (Estimated)

December 31, 2030

Study Registration Dates

First Submitted

December 14, 2019

First Submitted That Met QC Criteria

December 22, 2019

First Posted (Actual)

December 26, 2019

Study Record Updates

Last Update Posted (Estimated)

February 26, 2024

Last Update Submitted That Met QC Criteria

February 22, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NJCT-1901

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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