Study of the Safety and Efficacy of STI-6129 in Patients With Relapsed or Refractory Systemic AL Amyloidosis
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|ClinicalTrials.gov Identifier: NCT04316442|
Recruitment Status : Recruiting
First Posted : March 20, 2020
Last Update Posted : November 17, 2020
The STI-6129-001 study is a three-stage, multicenter, open-label, dose-finding, phase 1 trial. It is designed primarily to identify the recommended phase 2 dose (RP2D) of STI-6129 by assessing the safety, preliminary efficacy and pharmacokinetics of this anti-CD38-Duostatin 5.2 antibody-drug conjugate (ADC) for the treatment of relapsed or refractory systemic AL amyloidosis.
The patients that will be treated with STI-6129 in this trial are relapsed or refractory systemic AL amyloidosis patients who have received prior lines of treatment.
|Condition or disease||Intervention/treatment||Phase|
|Light Chain (AL) Amyloidosis||Biological: STI-6129||Phase 1|
This study is composed of three dosing plan stages. The initial stage of this trial is the dose-escalation stage. A standard dose-escalation 3+3 design will be utilized to identify a safe maximum tolerated dose (MTD) of STI-6129 in patients with relapsed or refractory systemic AL amyloidosis. After identification of the MTD, or the finding that the last dosing cohort is tolerated well (i.e., the maximum practical dose [MPD]), 12 patients will be enrolled to receive STI-6129 treatment at the MTD/MPD level to collect pharmacokinetic data ( the pharmacokinetic (PK) stage) to model a treatment schedule that achieves a stable effective serum concentration. Results from the dose-escalation stage and the pharmacokinetic stage will be analyzed to develop a treatment dose/schedule for treating 30 additional patients enrolled in the expansion stage.
Each patient enrolled will receive up to three 3-week cycles of STI-6129. After the treatment period, patients will be monitored for up to a year.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1, Open-Label, Dose-Escalation Study of the Safety and Efficacy of an Anti-CD38 Antibody Drug Conjugate (STI-6129) in Patients With Relapsed or Refractory Systemic AL Amyloidosis|
|Actual Study Start Date :||November 13, 2020|
|Estimated Primary Completion Date :||December 2021|
|Estimated Study Completion Date :||December 2021|
Experimental: STI-6129 infusion
Intravenous infusion to be given with prophylaxis for infusion reactions if necessary.
Three cycles of intravenous infusion of STI-6129 will be given (one infusion every three weeks).
Other Name: anti-CD38-Duostatin 5.2 antibody-drug conjugate (ADC)
- Safety and tolerability of STI-6129 in AL amyloidosis patients [ Time Frame: Baseline through study completion at up to 14 months ]Types, frequencies, and severities of adverse events (AEs) and the relationships of AEs to study drug; includes serious adverse events (SAEs), neurotoxicity, dose-limiting toxicities (DLTs), and laboratory abnormalities
- Overall hematological response rate according to the 2012 Consensus Round Table response criteria [ Time Frame: Baseline through study completion at up to 14 months ]Proportion of subjects with Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), No Response (NR) and Progressive Disease (PD)
- Organ response rates (cardiac, renal, hepatic, peripheral nervous system) according to the 2012 Consensus Round Table response criteria [ Time Frame: Baseline through study completion at up to 14 months ]Organ response rates (cardiac, renal, hepatic, peripheral nervous system) according to the 2012 Consensus Round Table response criteria
- Correlation of treatment response (organ responses and hematological response) with disease severity based on the 2012 revised Mayo Clinic staging system for AL amyloidosis [ Time Frame: Baseline through study completion at up to 14 months ]Correlation of treatment response (organ responses and hematological response) with disease severity based on the 2012 revised Mayo Clinic staging system for AL amyloidosis
- Plasma levels of the total antibody plus conjugated toxin (STI-6129) and the free toxin (Duostatin 5.2) [ Time Frame: Day 1 to day 63 ]Plasma levels of the total antibody plus conjugated toxin (STI-6129) and the free toxin (Duostatin 5.2) by ELISA and mass spectrophotometry assays, respectively, at pre-dose and various time points post-dose
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): NCT04316442
|Contact: Ying Yan, MD MS||858-203-4100 ext email@example.com|
|Contact: Mike Royal, MD JD MBA||858-203-4100 ext firstname.lastname@example.org|
|United States, New York|
|Columbia University - Herbert Irving Comprehensive Cancer Center||Recruiting|
|New York, New York, United States, 10032|
|Contact: Niha Mamillapalli 212-342-3849 email@example.com|
|Principal Investigator: Suzanne Lentzsch, MD PHD|
|Principal Investigator:||Suzanne Lentzsch, MD||Columbia University|