Clinical trial NCT04210791: Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
Official title: Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: AL Amyloidosis
Plasma Cell Dyscrasia
Keywords: AL amyloidosis
autologous stem cell transplantation
induction therapy
bortezomib
lenalidomide
ixazomib
daratumumab
melphalan
Lead sponsor: Nanjing University School of Medicine
Sponsor class: OTHER
First posted: 2019-12-26
Enrollment: 500
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: overall survival
timeFrame: 5 years
measure: progression-free survival
timeFrame: 5 years
measure: hematological complete response rate
timeFrame: 1 year
measure: organ response rate
timeFrame: 1 year
Secondary outcomes:
measure: hematological overall response rate
timeFrame: 1 year
measure: the relapse rate of complete remission participants
timeFrame: 5 years
measure: the rate of Minimal Residual Disease-negative participants
timeFrame: 5 years
measure: the median time form stem cell transplantation to next treatment
timeFrame: 5 years
Other outcomes:
Locations:
city: Nanjing
country: China
facility: National Clinical Research Center of Kidney Diseases, Jinling Hospital
state: Jiangsu
Study officials:
affiliation: Nanjing University School of Medicine
name: Zhihong Liu, MD
role: STUDY_DIRECTOR
References: