Phase 2
Completed N=10
A Dose Escalation Study of Carfilzomib Taken With Thalidomide and Dexamethasone in Relapsed AL Amyloidosis
Amyloidosis
Source: ClinicalTrials.gov NCT02545907 ↗
Enrolled (actual)
10
Serious AEs
30.0%
Results posted
Apr 2021
Primary outcomePrimary: Number of Participants With Dose-Limiting Toxicities as Assessed by Reported Data — 0; 1 Participants
Summary
This study evaluates the safety and efficacy of carfilzomib used in combination with thalidomide and dexamethasone in patients with relapsed AL amyloidosis. The trial begins with a dose escalation phase, in which the maximum tolerated and recommended dose will be determined. The trial will then open into an expansion phase in which the combination efficacy is assessed.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Dose-Limiting Toxicities as Assessed by Reported Data |
0; 1 | — |
| PRIMARY Number of Participants Experiencing Grade 3 or 4 Toxicity as Assessed by CTCAE v4.0. |
2; 3 | — |
| SECONDARY Clonal Response Rate Within 3 Months, at 3 Months, Within 6 Months and at 6 Months as Determined by Paraprotein and Free Light Chain Assessment. |
1; 5; 1; 5; 2; 5 | — |
| SECONDARY Amyloidotic Organ Response Rate Within 3 Months and 6 Months Based on Biochemical, Electrocardiographical, and Radiographical Assessment. |
0; 0; 0; 0; 3; 7 | — |
| SECONDARY Time to Amyloidotic Organ Response Based on Reported Data. |
0; 0 | — |
| SECONDARY Number of Deaths at 6 Months Based on Reported Data. |
0; 0 | — |
| SECONDARY Number of Patients Progression-free at 6 Months Based on Reported Data. |
3; 7 | — |
| SECONDARY Maximum Response Determined by Paraprotein and Free Light Chain Assessment. |
1; 0; 0; 1; 2; 1 | — |
| SECONDARY Time to Maximum Response Based on Reported Data. |
5.3 | — |
| SECONDARY Number of Patients Withdrawing From Treatment Based on Reported Data. |
0; 0 | — |
| SECONDARY Number of Patients Experiencing Dose Delays Based on Reported Data. |
2; 1 | — |
| SECONDARY Compliance Profile of KTD Based on Reported Chemotherapy Compliance Data. |
1; 5 | — |
Eligibility Criteria
Inclusion Criteria
- Patients with the following characteristics are eligible for this study:
- Aged 18 years or greater
- Diagnosis of systemic AL amyloidosis with:
- exclusion of genetic mutations associated with hereditary amyloidosis and immunohistochemical exclusion of AA and TTR amyloidosis as appropriate.
- Amyloid related organ dysfunction or organ syndrome
- Measurable clonal disease
- Clonal relapse after previous chemotherapy or stem cell transplant OR refractory clonal disease to previous chemotherapy or stem cell transplant
- Capable of providing written, informed consent and willing to follow study protocol
- Life expectancy ≥ 6 months
- ECOG performance status of 0.07 and NT-proBNP >700 pMol/L OR NT-proBNP >1000 pMol/L OR supine SBP <100 mm of Hg
- Myocardial infarction in the preceeding 6 months or unstable angina or conduction abnormalities uncontrolled by medication or devices
- Concurrent active malignancies, except surgically removed basal cell carcinoma of the skin or other in situ carcinomas
- Pregnant, lactating or unwilling to use adequate contraception
- Systemic infection unless specific anti-infective therapy is employed.
- Known or suspected HIV infection
- Contraindication to any of the required concomitant drugs or supportive treatments. Any other clinically significant medical disease or condition or psychiatric illness that, in the Investigator's opinion, may interfere with protocol adherence or a participant's ability to give informed consent
- Previous experimental agents or approved anti-tumour treatment within 3 months before the date of registration
- Known allergies to the IMPs
Data sourced from ClinicalTrials.gov (NCT02545907). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.