Autologous Stem Cell Transplantation for Patients With AL Amyloidosis

NCT04210791 | Enrolling By Invitation DMC

• 1 other identifier: NJCT-1901
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

AL Amyloidosis; Plasma Cell Dyscrasia

Keywords

AL amyloidosis; autologous stem cell transplantation; induction therapy; bortezomib; lenalidomide; ixazomib; daratumumab; melphalan

Outcome Measures

Primary Outcomes (4)

• overall survival

  the 5 years overall survival after autologous stem cell transplantation.

  5 years

• progression-free survival

  the 5 years progression-free survival after autologous stem cell transplantation.

  5 years

• hematological complete response rate

  the 1 year hematologic complete response rate after autologous stem cell transplantation.

  1 year

• organ response rate

  the 1 years organ response rate after autologous stem cell transplantation.

  1 year

Secondary Outcomes (4)

• hematological overall response rate

  1 year

• the relapse rate of complete remission participants

  5 years

• the rate of Minimal Residual Disease-negative participants

  5 years

• the median time form stem cell transplantation to next treatment

  5 years

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Nanjing University School of Medicine: lead

Study Sites (1)

National Clinical Research Center of Kidney Diseases, Jinling Hospital

Nanjing, Jiangsu, 210016, China

Location

Related Publications (1)

• Zhang Y, Guo J, Chen W, Zhao L, Huang X. Risk factors, treatments and outcomes of patients with light chain amyloidosis who relapse after autologous stem cell transplantation. Bone Marrow Transplant. 2024 Mar;59(3):350-358. doi: 10.1038/s41409-023-02185-z. Epub 2023 Dec 26.

  PMID: 38148411 DERIVED

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis; Paraproteinemias

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zhihong Liu, MD

  Nanjing University School of Medicine

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 14, 2019
• First Posted: December 26, 2019
• Study Start: July 1, 2010
• Primary Completion: December 31, 2025
• Study Completion (Estimated): December 31, 2030
• Last Updated: February 26, 2024

Record last verified: 2024-02

Locations

CN (1)