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Table 5. Summary of the results of published clinical studies of Selinexor in human malignancies
Tumor type Phase Remarks Reference/ID
Advanced solid tumors Phase I Selinexor single agent. 189 patients enrolled. [193]
Most common grade ≥ 3 adverse events: thrombocytopenia, fatigue ID: NCT01607905
2
and hyponatremia. RP2D 35 mg/m given twice weekly.
157 evaluable patients. 1 CR and 5 PR
Sarcoma Phase I Selinexor single agent. 54 patients enrolled. [194]
Most common grade ≥ 3 adverse events: fatigue, thrombocytopenia, ID: NCT01896505
anemia, lymphopenia, and leucopenia.
52 evaluable patients. SD: 33%
Pediatric refractory acute leukemia Phase I Selinexor combined with Fludarabine and Cytarabine. 18 patients en- [195]
rolled. ID: NCT02212561
2
Selinexor tolerable at doses up to 55 mg/m in pediatric patients.
15 evaluable patients. CR: 47%
Non-Hodgkin lymphoma Phase I Selinexor single agent. 79 patients enrolled. [196]
Most common grade ≥ 3 adverse events: thrombocytopenia, neutrope- ID: NCT01607892
nia, anemia, leukopenia, fatigue, and hyponatremia. RP2D 60 mg.
70 evaluable patients. OR: 31% (including 4 CR and 18 PR)
Acute myeloid leukemia Phase I Selinexor single agent. 95 patients enrolled. [197]
Most common grade ≥ 3 nonhematological adverse event: fatigue. No ID: NCT01607892
reported dose-limiting toxicities. RP2D 60 mg.
81 evaluable patients. OR: 14%
Phase I Selinexor combined with cytarabine and mitoxantrone. 20 patients [198]
enrolled. ID: NCT02573363
Serious adverse events 30%, including one fatal adverse event. RP2D:
80 mg.
20 evaluable patients. Overall response rate 70% (including 10 CR)
Multiple myeloma Phase I Dose-escalation phase: Selinexor as single agent in 25 patients en- [199]
rolled. ID: NCT01607892
Dose-expansion phase: Selinexor as single agent or combined with
dexamethasone. 59 patients enrolled.
Most common grade ≥ 3 adverse event: thrombocytopenia. RP2D: 80
mg plus 20 mg dexamethasone given twice weekly.
Objective response rate: 4% Selinexor as single agent, 50% when
combined with dexamethasone
Phase II Selinexor combined with dexamethasone. 79 patients (multi-refractory [200]
disease) enrolled.
Most common grade ≥ 3 adverse events: thrombocytopenia, anemia,
neutropenia, hyponatremia, leukopenia, and fatigue.
Overall response rate 21%
Castration-resistant prostate Phase II Selinexor as single agent. 14 patients (refractory to anti-androgenic [201]
cancer therapy) enrolled. ID: NCT02215161
Some activity (PR 25%), but poor tolerability.

ID: identifier at ClinicalTrials.gov; MTD: maximum-tolerated dose; RP2D: recommended Phase II dose; OR: objective response; CR:
complete response; PR: partial response; SD: stable disease

common grade 3 or 4 toxicities in this series were fatigue, thrombocytopenia, anemia, lymphopenia, and
leucopenia. In 52 evaluable patients, no objective responses were seen, but 17 patients achieved stable dis-
ease for ≥ 4 months. The authors concluded that Selinexor shows preliminary evidence of anticancer activ-
ity in sarcoma, and is well tolerated at a 60 mg flat dose.

[195]
Alexander et al. evaluated the combination of Selinexor with fludarabine and cytarabine in 18 pediatric
patients with relapsed or refractory leukemia. Selinexor was administered twice per week at several doses
2
2
between 30 mg/m and 70 mg/m . Dose-limiting reversible cerebellar toxicity was experienced by some
2
patients treated with the 70 mg/m dose. Seven of the 15 patients that were evaluable achieved complete
response. The authors concluded that Selinexor combined with fludarabine and cytarabine shows promis-
2
ing response rates in pediatric patients with relapsed or refractory leukemia. A 55 mg/m dose Selinexor is
tolerable in this combination.
[196]
Kuruvilla et al. evaluated Selinexor in 79 patients with different subtypes of non-Hodgkin lymphoma
2
(NHL). In the dose-expansion phase of the study, Selinexor was administered at doses of 35 mg/m or