Treatment for systemic AL amyloidosis using bortezomib, pomalidomide, and dexamethasone

A Study of Bortezomib, Pomalidomide, Dexamethasone in Patients with Systemic AL Amyloidosis

This study is testing a new combination of medications to see if it can help people with systemic AL amyloidosis feel better and improve their blood health.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Ages: 18 Years to 90 Years
• Sex: All
• Sponsor: Peking University People's Hospital (Academic / other)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06342466 on ClinicalTrials.gov

What this trial studies

This open-label, multicenter Phase 2 study aims to evaluate the efficacy of a combination therapy involving bortezomib, pomalidomide, and dexamethasone in patients with newly diagnosed or previously treated systemic light chain (AL) amyloidosis. Approximately 40 subjects will be enrolled, and the primary outcome will focus on achieving hematologic very good partial response and complete response rates at the six-month mark. The study is designed to leverage the synergistic effects of proteasome inhibitors and immunomodulators to address the challenges posed by renal involvement in amyloidosis patients.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of AL amyloidosis and measurable disease.

Not a fit: Patients with severe renal impairment or those who do not meet the inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve hematologic responses in patients with systemic AL amyloidosis.

How similar studies have performed: While the combination of these agents has been explored in other contexts, this specific approach for systemic AL amyloidosis is relatively novel.

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.

Where this trial is running

Beijing, Beijing Municipality

• Peking University People's Hospital — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Jin Lu — Peking University People's Hospital
• Study coordinator: Yang Liu, Dr

Conditions Systemic Amyloidosis