Clinical trial NCT06342466: Bortezomib, Pomalidomide, Dexamethasone for Systemic AL Amyloidosis
Official title: A Study of Bortezomib, Pomalidomide, Dexamethasone in Patients with Systemic AL Amyloidosis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Systemic Amyloidosis
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2024-04-02
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Bortezomib, pomalidomide, dexamethasone
Primary purpose: TREATMENT

Brief summary: This is an open-label, multicenter, Phase 2 study in subjects with newly diagnosed or previous treated systemic light chain (AL) amyloidosis. Approximately 40 subjects will receive therapy with bortezomib, pomalidomide, and dexamethasone. The primary outcome is hematologic very good partial response and complete response rate at 6 months.

Detailed description: Proteasome inhibitor and immunmodulators have synergistic effect for plasma cell dyscrasia. Due to the fact that more than 70-80% of patients with amyloidosis have renal involvement, the application of lenalidomide is limited. Thus, the investigators designed this open-label, multicenter, phase 2 study for newly diagnosed or previously treated systemic AL amyloidosis with bortezomib, pomalidomide and dexamethasone regimen.

Eligibility criteria: Inclusion Criteria:

1. Diagnosis of AL amyloidosis, confirmed by histology and typed with immunohistochemistry, immunoelectron microscopy or mass spectrometry.
2. Newly diagnosed or previous treated AL amyloidosis
3. Patients must be ≥ 18 years of age.
4. ECOG performance status 0, 1 or 2.
5. Measurable disease defined by at least one of the following:

   ① serum free light chain (FLC) ≥2.0 mg/dL (20 mg/L) with an abnormal kappa:lambda ratio or the difference between involved and uninvolved free light chains (dFLC) ≥2mg/dL (20 mg/L).

   ②. presence of a monoclonal spike that is ≥5 g/l.
6. Symptomatic organ involvement (heart, kidney, liver/GI tract, peripheral nervous system).
7. Absolute neutrophil count (ANC) ≥1.0 X 10\^9/L, Hemoglobin ≥70 g/L, Platelets ≥50 X 10\^9/L
8. eGFR ≥20 mL/min/ 1.73 m\^2
9. Written informed consent in accordance with local and institutional guidelines.
10. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

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: Hematologic VGPR + CR rate at 6 months
timeFrame: 6 months
Secondary outcomes:
measure: Overall Hematologic response rate at 6 months
timeFrame: 6 months
measure: At least one organ response at 6 months
timeFrame: 6 months
measure: TTNT at 2 years
timeFrame: 2 years
measure: Estimated PFS at 2 years
timeFrame: 2 years
measure: Estimated OS at 2 years
timeFrame: 2 years
measure: TRAE
timeFrame: 6 months
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: