Rapid dFLC Response Predict CHR in AL Amyloidosis

NCT06627309 | Recruiting

• 1 other identifier: 2023PHB319-001-02
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 8

Brief Summary

Light chain amyloidosis (AL amyloidosis) is a rare plasma cell dyscrasia characterized by the deposition of insoluble amyloid fibrils in multiple organ systems. The treatment of amyloidosis primarily relies on anti-plasma cell therapy and supportive care. The application of anti-plasma cell therapy has significantly improved outcomes in patients with AL amyloidosis. Standard first-line therapy typically includes daratumumab, bortezomib, cyclophosphamide, and dexamethasone (Dara-BCD), achieving a complete hematologic response in nearly 60% of patients.The depth and speed of the hematologic response are strongly correlated with organ response and overall survival. An early achievement of a complete hematologic response is particularly crucial in cases of AL amyloidosis characterized by significant organ involvement. The median time to a hematologic response for the daratumumab based treatment is only 7-9 days. The retrospective data showed that the hematologic response in Day 7 in Cycle 1 (C1D7) may predict the complete hematologic response rate. In order to validate the conclusion, the investigator design this prospective study.

Conditions

Systemic AL Amyloidosis

Keywords

rapid dFLC response; daratumumab; systemic al amyloidosis

Outcome Measures

Primary Outcomes (1)

• Complete hematologic response

  6 months

Secondary Outcomes (6)

• Overall hematologic response

  6 months

• At least one organ response

  12 months

• Minimal residual disease

  6 months, 12 months

• TTNT

  12 months

• MOD-EFS

  12 months

Study Arms (1)

Group 1

All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment. All patients receive additional sFLC examination in C1D7, C1D14.

Drug: Dara IV; Drug: Bortezomib (drug); Drug: Dexamethasone; Drug: Cyclophosphamide (CTX); Drug: Dara SC

Interventions

Dexamethasone DRUG

Dexamethasone is administered intravenously at a dose of 40 mg weekly for each cycle, for a maximum of 6 cycles. For patients over 70 years of age or with severe edema, heart failure, or uncontrolled diabetes mellitus, dexamethasone can be administered at a dose of 10-20 mg per week.

Group 1

Cyclophosphamide (CTX) DRUG

Some patients may receive cyclophosphamide orally or intravenously at a dosage of 300 mg/m2 weekly.

Group 1

Dara IV DRUG

The treament follow the clinical routine practice, as daratumumab 16mg/kg iv qw OR dara SC 1800mg initially. The treatment schedule involve weekly administrations during cycles 1 to 2, following by biweekly administrations during cycles 3 to 6. Subsequently, monthly administrations are given as monotherapy.

Group 1

Bortezomib (drug) DRUG

Bortezomib is administered subcutaneously. The dosage range from 0.7 to 1.3 mg/m2 on days 1, 8, 15, and 22 of each cycle, for a maximum of 6 cycles

Group 1

Dara SC DRUG

The treament follow the clinical routine practice, as daratumumab 16mg/kg iv qw OR dara SC 1800mg initially. The treatment schedule involve weekly administrations during cycles 1 to 2, following by biweekly administrations during cycles 3 to 6. Subsequently, monthly administrations are given as monotherapy.

Group 1

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Systemic AL amyloidosis patients with evaluable hematologic parameter

You may qualify if:

• Diagnosis of systemic AL amyloidosis;
• Daratumumab, bortezomib, dexamethasone used in treatment;
• Informed consent explained to, understood by and signed by the patient;
• dFLC ≥ 50 mg/L;

You may not qualify if:

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

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (8)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

RECRUITING

Fuxing Hospital affiliated to Capital Medical University

Beijing, Beijing Municipality, 100045, China

RECRUITING

Beijing Anzhen Hospital

Beijing, Beijing Municipality, 101118, China

RECRUITING

Peking University First Hospital

Beijing, Beijing Municipality, China

RECRUITING

First Affiliated Hospital of Harbin Medical University

Harbin, Heilongjiang, China

RECRUITING

Chinese PLA Eastern Theater General Hospital

Nanjing, Jiangsu, China

RECRUITING

Shengjing Hospital of China Medical University

Shenyang, Liaoning, China

RECRUITING

The First Affiliated Hospital of Xi'an Jiao Tong University

Xi'an, China

RECRUITING

MeSH Terms

Conditions

Immunoglobulin Light-chain Amyloidosis

Interventions

Bortezomib; Pharmaceutical Preparations; Dexamethasone; Cyclophosphamide

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Paraproteinemias

Intervention Hierarchy (Ancestors)

Boronic Acids; Acids, Noncarboxylic; Acids; Inorganic Chemicals; Boron Compounds; Organic Chemicals; Pyrazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Phosphoramides; Organophosphorus Compounds

Central Study Contacts

Yang Liu, M.D.

CONTACT

+86-13716926210; pkuphliuyang@bjmu.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 1 Year
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal of Investigator

Study Record Dates

• First Submitted: September 29, 2024
• First Posted: October 4, 2024
• Study Start: October 26, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: March 9, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (8)