Clinical trial NCT06192979: Optimize First-line Treatment for AL Amyloidosis With t (11; 14)
Official title: Optimize First-line Treatment for Systemic Light Chain Amyloidosis With t (11; 14)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Amyloidosis; Systemic
AL Amyloidosis
Keywords: t(11;14)
Rapid Response
AL amyloidosis
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2024-01-05
Enrollment: 41
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Achievement of complete hematologic response (CHR) is vital for systemic AL amyloidosis. Currently, the CHR rate of daratumumab, bortezomib, and dexamethasone (DBD) is close to 60%. Considering that Bcl-2 inhibitor is effective for AL amyloidosis with t(11; 14) and the median hematologic onset time of DBD is 7 days. We design a a prospective study on AL amyloidosis with t(11; 14). All patients receive DBD at the beginning. Patient will receive DBD for at least 6 cycles if achieve rapid hematologic response at day 7, while other patients will receive daratumumab, venetoclax and dexamethasone.

Detailed description: The goal of this clinical trial is to optimize the first line treatment for systemic AL amyloidosis with t(11;14). The aim of this study is to pursue early complete hematologic response. The primary endpoint is overall complete hematologic response (CHR) rate at 6 months. Participants will be treated according to the hematologic response after 7 days. If the patient get rapid response after 7 days, he/she will receive daratumumab, venetoclax and dexamethasone (DBD) for at least 6 cycles. If the patient do not get rapid response, he/she will receive daratumumab, venetoclax and dexamethasone.

Eligibility criteria: Inclusion Criteria:

1. Diagnosis of systemic AL amyloidosis;
2. Daratumumab, bortezomib, dexamethasone used in 1st line treatment;
3. Life expectancy greater than 12 weeks;
4. HGB ≥70g/L;
5. Blood oxygen saturation \>90%;
6. Total bilirubin (TBil) ≤3×upper limit of normal (ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3.0×ULN;
7. Informed consent explained to, understood by and signed by the patient.

Exclusion Criteria:

1. Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma.
2. Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis;
3. Severe or persistent infection that cannot be effectively controlled;
4. Presence of severe autoimmune diseases or immunodeficiency disease;
5. Patients with active hepatitis B or hepatitis C (\[HBVDNA+\] or \[HCVRNA+\]);
6. Patients with HIV infection or syphilis infection;
7. Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.

Primary outcomes:
measure: Overall CHR rate at 6 months
timeFrame: Overall CHR rate at 6 months
Secondary outcomes:
measure: Cardiac response at 6 months
timeFrame: Cardiac response at 6 months
measure: Renal response at 6 months
timeFrame: Renal response at 6 months
measure: Hepatic response at 6 months
timeFrame: Hepatic response at 6 months
measure: Estimated 2-year PFS
timeFrame: Estimated 2-year PFS
measure: Estimated 2-year OS
timeFrame: Estimated 2-year overall survival
measure: MRD status at 6 months
timeFrame: MRD status at 6 months
measure: TRAEs
timeFrame: TRAEs
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital
name: Jin Lu
role: PRINCIPAL_INVESTIGATOR
References: