Clinical trial NCT06376214: Daratumumab for Patients With Light Chain Amyloidosis
Official title: Daratumumab and Dexamethasone Combined With Pomalidomide (DPD) or ASCT in the Treatment of Newly Diagnosed Systemic Light Chain Amyloidosis (AL Amyloidosis): a Prospective, Single Center Clinical Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Light Chain (AL) Amyloidosis
Keywords: light chain amyloidosis
daratumumab
stem cell transplantation
Lead sponsor: Nanjing University School of Medicine
Sponsor class: OTHER
First posted: 2024-04-19
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen, and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen, and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment. The purpose of this study is to observe the efficacy and safety of Daratumumab, in the treatment of newly diagnosed systemic AL amyloidosis.

Detailed description: This is a prospective, single-center study exploratory clinical trial, aim to exploring the efficacy and safety of daratumumab in patients with AL amyloidosis, patients were divided into three groups: one group received long-term treatment with daratumumab based regimen (group A) and the other group received autologous stem cell transplantation after two standard treatment courses with daratumumab based regimen (group B) , and the third group consists of newly diagnosed stage IIIb AL amyloidosis patients who plan to receive DPD treatment (group C). Participants with AL Amyloidosis will receive the drug daratumumab by IV infusion once weekly for two months, then every 2 weeks for four months, then once each month. Study treatment may continue until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression. Group C were given Daratumumab (16mg/kg), dexamethasone (40mg) and pomalidomide. The usage of Daratumumab and dexamethasone is the same as group A and B. Patients received oral pomalidomide on days 1-21 of a 28-days cycle from cycle 1 to 6. The dose of pomalidomide is adjusted based on the patient's renal function and ECOG score.

Eligibility criteria: Inclusion Criteria:

1. Participants must be aged ≥18 and ≤75 years;
2. Newly diagnosed AL amyloidosis ((confirmed by pathological examination with at least one major organ involvement (heart, kidney, or liver));
3. In group A and B, according to the Mayo 2004 staging system, the disease is classified as stage Ⅰ-ⅢA; In group C, according to the Mayo 2004 staging system, the disease is classified as stage IIIB: NT⁃proBNP\>8500ng/L and cTnT\>0.035μg/L or cTnI\>0.01g/L;
4. Participants must personally sign an informed consent form approved by the Ethics Committee before the start of the study;
5. Expected survival ≥ 12 weeks;
6. ECOG performance status≤ 2;
7. Female participants of childbearing potential must agree to use effective contraception from the day of signing the informed consent until 365 days after the infusion. Effective contraception is defined as abstinence or the use of a contraceptive method with a failure rate of \<1% per year.

Exclusion Criteria:

1. eGFR\< 30ml/min/1.73m2;
2. Combined multiple myeloma;
3. Acute or chronic infection requiring treatment within 30 days prior to baseline;
4. Pregnant or breastfeeding women.
5. Participants known to have life-threatening allergic reactions, hypersensitivity, or intolerance to Monoclonal antibodies or immune modulators.
6. Other conditions deemed by the researcher as unsuitable for enrollment.

Primary outcomes:
measure: Hematological complete response rate
timeFrame: 6 months
measure: Organ response rate
timeFrame: 1 year
Secondary outcomes:
measure: Time to Next Treatment
timeFrame: 3 years
measure: Progression-free survival
timeFrame: 3 years
measure: Overall survival
timeFrame: 5 years
Other outcomes:
Locations:
city: Nanjing
country: China
facility: National Clinical Research Center for Kidney Diseases, Jinling Hospital
state: Jiangsu
Study officials:
affiliation: Jinling Hospital, China
name: Xianghua Huang, MD
role: PRINCIPAL_INVESTIGATOR
References: