BCD With or Without Doxycycline in Mayo Stage II-III Light Chain Amyloidosis Patients

Summary

Survival of intermediate and high-risk primary light chain amyloidosis (pAL) remains poor due to high mortality within 3-6 months of diagnosis. Rapidly effective regimens such as bortezomib, cyclophosphamide and dexamethasone (BCD) still failed to overcome the poor prognosis in very advanced pAL amyloidosis patients. Recently, doxycycline was demonstrated to induce disruption of fibril formation and reduce the number of intact fibrils in transgenic mouse model of pAL amyloidosis. Furthermore, case-control study suggested that adjuvant oral doxycycline could improve response and survival in cardiac pAL amyloidosis, which necessities further confirmation through a randomized trial. Therefore, we designed a multi-center randomized open-label controlled study to investigate the efficacy and safety of co-administration of oral doxycycline with BCD regimen in treatment-naïve patients with Mayo stage II-III pAL amyloidosis. The primary outcome progression-free survival, and secondary endpoints including overall survival, hematologic response, organ response and toxicity of doxycycline will be evaluated.

Conditions

• Amyloidosis; Systemic

Interventions

DRUG

Doxycycline

Oral doxycycline 100mg twice daily

DRUG

Bortezomib

1.3mg/m2 of bortezomib on days 1, 8, 15 and 22 of a 35-day cycle

DRUG

Cyclophosphamide

300mg/m2 cyclophosphamide on days 1, 8, 15 and 22 of a 35-day cycle

DRUG

Dexamethasone

40mg of dexamethasone on days 1, 8, 15 and 22 of a 35-day cycle

Sponsors & Collaborators

• Peking University First Hospital

  collaborator OTHER

• Beijing Anzhen Hospital

  collaborator OTHER

• Beijing Chao Yang Hospital

  collaborator OTHER

• West China Hospital

  collaborator OTHER

• Tongji Hospital

  collaborator OTHER

• Union Hospital Affiliated with Tongji Medical College of HUST

  collaborator UNKNOWN

• Shanghai Changzheng Hospital

  collaborator OTHER

• Nanfang Hospital, Southern Medical University

  collaborator OTHER

• Jian Li

  lead OTHER

Principal Investigators

• Jian Li, MD · Peking Union Medical College Hospital

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2018-04-21

Primary Completion

2020-12-31

Completion

2020-12-31

Countries

• China

Study Locations