Table 1 Ongoing and completed clinical trials with SMs.

A list of all identifiable completed or ongoing clinical trials from searching the ClinicalTrials.gov and ClinicalTrialsRegister.eu databases and studies published as research papers (PubMed) or abstracts (Web of Science) by January 2020. Indications and reported side effects are listed. If a compound has been evaluated for the same indication in different trials, then only the trial representing the highest phase is listed. Studies terminated early are excluded. AML, acute myelogenous leukemia; CRC, colorectal cancer; CRS, cytokine release syndrome; DLT, dose-limiting toxicity; HNSCC, head and neck squamous cell carcinoma; MDS, myelodysplastic syndrome; NSCLC, non–small cell lung cancer; RCC, renal cell carcinoma; SCLC, small-cell lung cancer; TNBC, triple-negative breast cancer; N/A, not available; pts., patients.

CompoundPhaseType of malignancyResultsClinical trial identifier
(trial status)
(publication)
ASTX660*1AMLN/ANCT04155580
(ongoing)
1/2Solid tumors, lymphomaN/ANCT02503423
(ongoing)
BI 891065*1Solid tumors, NSCLCN/ANCT03166631
(ongoing)
CUDC-427*
(formerly GDC-0917)
1Solid tumors42 pts. Well-tolerated at
5–600 mg/day
Decreased cIAP1 abundance in
PBMCs
NCT01226277
(completed)
(109)
DEBIO 1143*1Solid tumors, lymphoma51 pts. Well-tolerated <180
mg/day. At higher dosage
alanine aminotransferase
was increased in 5% of pts.
Decreased cIAP1 abundance
in PBMCs
NCT01078649
(completed)
(112)
1Pancreatic cancer, CRCN/ANCT03871959
(ongoing)
1bSolid tumors, NSCLCN/ANCT03270176
(ongoing)
1/2HNSCCN/ANCT02022098
(ongoing)
1/2SCLC, HNSCC,
gastrointestinal ovarian,
endometrial, peritoneal, and
cervical cancer
N/ANCT04122625
(ongoing)
LCL-161*1Solid tumors71 pts. Well-tolerated up to
1800 mg once/week with 9%
experiencing CRS at higher
concentrations, and in 6% of
pts. as a DLT.
cIAP1 degradation in
peripheral tissue
NCT01098838
(completed)
(111)
2TNBC209 pts. Well-tolerated in
combination with paclitaxel,
although notable toxicity at
1800 mg/week
NCT01617668
(completed)
(113)
2Multiple myeloma25 pts. Well-tolerated, but
CRS observed in 4 of 11 pts.
at 1800 mg/week. After dose
reduction to 1200 mg/week,
no further CRS was observed
NCT01955434
(completed)
(117)
1bCRC, NSCLC, TNBC, RCCN/ANCT02890069
(ongoing)
1/2SCLC, ovarian cancerN/ANCT02649673
(ongoing)
2LeukemiaN/ANCT02098161
(ongoing)
APG-13871/2Solid tumors, hematologic
malignancies
N/ANCT03386526
(ongoing)
Birinapant†1Solid tumors, lymphoma50 pts. Well-tolerated in
dosages <35 mg/m2. At
higher doses, CRS was
observed in 5 of 12 pts. and
Bell’s palsy in 2 of 3 pts.
receiving 63 mg/m2.
cIAP1 suppressed in PBMCs
NCT00993239
(completed)
(114)
1Ovarian cancer27 pts. Well-tolerated among
89% of pts. 1 pt. got
pancreatitis at an
accumulated dose of
78 mg/m2, and 1 pt. got
Bell’s palsy at 104 mg/m2
NCT01940172
(completed)
(115)
1/2Solid tumors176 pts. Well-tolerated with
ascending dose strategy
<35 mg/m2. Bell’s palsy was
observed in 8% of pts., yet
lower risk than in the
single-dosing group with no
ascending strategy
NCT01188499
(completed)
(110)
1/2MDS21 pts. Well-tolerated at
13 mg/m2 when combined
with 5-azacitidine with side
effects consistent with the
disease under investigation.
17% of evaluable pts.
developed Bell’s palsy
NCT01828346
(completed)
(116)
1HNSCCN/ANCT03803774
(ongoing)
1/2AMLN/ANCT01486784
(ongoing)
HGS1029/ AEG408261Solid tumors66 pts. Well-tolerated at
dosages up to 2.1 mg/m2
Dose-related decrease of
cIAP1 abundance in PBMCs
NCT00708006
(completed)
(108)

*Monovalent

†Bivalent