Barriers to recruitment on clinical trials for AL amyloidosis from 2018 to 2023 at a referral center
| Clinical trials . | AQUARIUS∗ . | ANDROMEDA† . | CAEL 301‡ . | CAEL 302§ . | AFFIRM-AL|| . | ETCTN10440¶ . | STI6129# . | DPd∗∗ . | OP201†† . | Total . |
|---|---|---|---|---|---|---|---|---|---|---|
| No. of enrolled patients | 4 | 8 | 2 | 8 | 1 | 2 | 3 | 3 | 0 | 31 |
| No. of prescreen failures (before informed consent) | 11 | 9 | 7 | 17 | 13 | 5 | 5 | 9 | 3 | 79 |
| Reasons for prescreen failure | ||||||||||
| Patient preference due to logistics of the trial | 7 | 3 | 1 | 4 | 4 | 0 | 0 | 2 | 0 | 21 |
| dFLC not meeting eligibility | 1 | 3 | 0 | 1 | 2 | 3 | 2 | 2 | 0 | 14 |
| eGFR not meeting eligibility | 0 | 0 | 0 | 1 | 1 | 2 | 1 | 0 | 1 | 6 |
| Cardiac biomarkers not meeting eligibility | 1 | 1 | 1 | 3 | 3 | 0 | 0 | 3 | 1 | 13 |
| Orthostatic hypotension | 0 | 1 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 2 |
| Myeloma by SLiM CRAB criteria | 2 | 0 | 2 | 2 | 1 | 0 | 0 | 0 | 0 | 7 |
| Other reasons‡‡ | 0 | 1 | 3 | 5 | 2 | 0 | 2 | 2 | 1 | 16 |
| Clinical trials . | AQUARIUS∗ . | ANDROMEDA† . | CAEL 301‡ . | CAEL 302§ . | AFFIRM-AL|| . | ETCTN10440¶ . | STI6129# . | DPd∗∗ . | OP201†† . | Total . |
|---|---|---|---|---|---|---|---|---|---|---|
| No. of enrolled patients | 4 | 8 | 2 | 8 | 1 | 2 | 3 | 3 | 0 | 31 |
| No. of prescreen failures (before informed consent) | 11 | 9 | 7 | 17 | 13 | 5 | 5 | 9 | 3 | 79 |
| Reasons for prescreen failure | ||||||||||
| Patient preference due to logistics of the trial | 7 | 3 | 1 | 4 | 4 | 0 | 0 | 2 | 0 | 21 |
| dFLC not meeting eligibility | 1 | 3 | 0 | 1 | 2 | 3 | 2 | 2 | 0 | 14 |
| eGFR not meeting eligibility | 0 | 0 | 0 | 1 | 1 | 2 | 1 | 0 | 1 | 6 |
| Cardiac biomarkers not meeting eligibility | 1 | 1 | 1 | 3 | 3 | 0 | 0 | 3 | 1 | 13 |
| Orthostatic hypotension | 0 | 1 | 0 | 1 | 0 | 0 | 0 | 0 | 0 | 2 |
| Myeloma by SLiM CRAB criteria | 2 | 0 | 2 | 2 | 1 | 0 | 0 | 0 | 0 | 7 |
| Other reasons‡‡ | 0 | 1 | 3 | 5 | 2 | 0 | 2 | 2 | 1 | 16 |
CRAB, hypercalcemia, renal failure, anemia and bone lesion; CrCl, creatinine clearance; dFLC, difference in involved and uninvolved light chain levels; eGFR, estimated glomerular filtration rate; IVSd, interventricular septal diameter; LVEF: left ventricular ejection fraction; NTproBNP, N-terminal prohormone of brain natriuretic peptide; PR, partial hematologic response; QTcF, corrected QT interval, SLiM, >60% bone marrow plasmacytosis, serum light chain ratio >100, MRI with 3 focal lesions.
AQUARIUS (NCT05250973): newly diagnosed, Mayo Clinic 2004 stage II and IIIA cardiac with or without other organ involvement, and dFLC ≥40 mg/L with an abnormal ratio.
ANDROMEDA (NCT03201965): newly diagnosed, dFLC ≥50 mg/L, NTproBNP ≤8500 pg/mL, and QTcF <500 milliseconds.
CAEL301 (NCT04504825): newly diagnosed, Mayo Clinic 2004 stage IIIB, dFLC >40 mg/L, and NTproBNP >8500 pg/mL.
CAEL 302 (NCT04512235): newly diagnosed, Mayo Clinic 2004 stage IIIA, dFLC >40 mg/L, and NTproBNP >650 and <8500 pg/mL.
AFFIRM-AL (NCT04973137): newly diagnosed, Mayo Clinic 2012 stage IV, and NTproBNP <8500 pg/mL.
ETCTN10440 (NCT04847453): relapsed after 1 line of treatment, Mayo Clinic 2004 stage I-IIIA, dFLC >20 mg/L, NTproBNP <8500 pg/mL, and CrCl ≥15 mL/min.
STI-6129 (NCT05692908): relapsed, refractory, dFLC >40 mg/L, and LVEF >40%.
DPd (NCT04270175): relapsed and/or refractory, eGFR >20 mL/min per 1.73 m2, prior daratumumab treatment, and prior pomalidomide exposure was allowed if PR or better was achieved and no disease progression occurred within 60 days of last dose received.
OP201 (NCT04115956): relapsed after 1 line of treatment, Mayo Clinic 2004 stage I to II.
For example, IVSd <12 mm; QTcF >500 milliseconds; LVEF 40%.