Daratumumab for the Treatment of Patients With AL Amyloidosis
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02841033|
Recruitment Status : Completed
First Posted : July 21, 2016
Results First Posted : May 7, 2021
Last Update Posted : May 7, 2021
|Condition or disease||Intervention/treatment||Phase|
|AL Amyloidosis||Drug: daratumumab||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I-II Trial of Daratumumab for the Treatment of Patients With AL Amyloidosis|
|Actual Study Start Date :||March 20, 2017|
|Actual Primary Completion Date :||May 28, 2020|
|Actual Study Completion Date :||July 17, 2020|
Daratumumab, 16mg/kg body weight in 1000 mL for the first dose, followed by 500mL for subsequent doses, once weekly for two months, then every 2 weeks for four months, then once each month.
Daratumumab by IV infusion once weekly for two months, then every 2 weeks for four months, then once each month until progression or inability to tolerate.
Other Name: Darzalex
- Assess the Number of Patients Who Respond to Treatment [ Time Frame: 3 months ]
Number of participants with response and ability to tolerate study treatment in each of these categories: Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Stable Disease (SD), and Progressive Disease (PD).
All participants were able to tolerate study treatment.
Per protocol, overall response designations are a combination of hematologic response.
A CR is defined as negative serum and urine immunofixation electrophoresis with normal serum free light chain ratio; a VGPR is reduction in the dFLC* to <40 mg/L, a PR is dFLC reduction by >50%; SD is not meeting criteria for CR, VGPR, PR, or PD; and PD is an increase in FLC of 50% to >100 mg/L.
* "dFLC" is difference in involved and uninvolved serum Free Light-Chain levels.
- Time to Next Treatment [ Time Frame: Up to 3 years ]Number of months from study drug initiation to starting another treatment
- Assess Hematologic Response Based on Blood and Urine Testing Using Standard Criteria [ Time Frame: 3 months ]
Number of patients with hematologic complete response (CR), very good partial response (VGPR), or partial response (PR).
Per protocol, a hematologic CR is defined as negative serum and urine immunofixation electrophoresis with normal serum free light chain ratio; a VGPR is reduction in the dFLC* to <40 mg/L; and a PR is dFLC reduction by >50%.
* "dFLC" is difference in involved and uninvolved serum free light-chain levels
- Assess Organ Responses Based on Standard Criteria Included in Protocol [ Time Frame: 3 months ]
Number of patients with organ response based on standard criteria included in protocol.
Cardiac response is defined as: NT-proBNP response (>30% and >300 ng/L decrease in patients with a baseline NT-proBNP >650 ng/L; and/or NYHA class response (> two-class decrease if baseline NYHA class 3 or 4)
Renal response is defined as: Decrease in proteinuria by > 30% or below 0.5 g/24 h without renal progression. Serum creatinine and creatinine clearance must not worsen by 25% over baseline
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02841033
|United States, Massachusetts|
|Boston Medical Center|
|Boston, Massachusetts, United States, 02118|
|Principal Investigator:||Vaishali Sanchorawala, MD||Boston Medical Center|