NCT05918055

Eltanexor (KPT-8602) With Inqovi (Decitabine-Cedazuridine) in High-Risk Myelodysplastic Syndromes

Terminated Phase 1, PHASE2 Results posted Last updated 2 December 2025

What this trial tests

Phase 1, PHASE2 trial testing KPT-8602 in Myelodysplastic Syndromes in 3 participants. Terminated before completion.

Timeline

14 November 2023

Primary endpoint
10 March 2025

12 March 2025

Quick facts

Lead sponsor	National Cancer Institute (NCI)
Phase	Phase 1, PHASE2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	3
Start date	14 November 2023
Primary completion	10 March 2025
Estimated completion	12 March 2025
Sites	1 location across United States

Drugs / interventions tested

• KPT-8602 — full drug profile →
• Inqovi — full drug profile →
• Bone marrow aspirate
• Bone marrow biopsy
• ECG

Conditions studied

• Myelodysplastic Syndromes — all drugs for Myelodysplastic Syndromes →

Sponsor

National Cancer Institute (NCI)

Who can join

Adults 18 to 120, any sex, with Myelodysplastic Syndromes. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Phase 1: Recommended Phase 2 Dose (RP2D) of Eltanexor (KPT-8602) in Combination With Inqovi (Decitabine-Cedazuridine) in Adult Participants With Higher-Myelodysplastic Syndromes (MDS) Primary · From day 1 of study drug through 28 days after the first dose

If no DLTs are observed at the highest planned dose level for evaluation (dose level 2), dose escalation will stop, and this will be considered the recommended phase 2 dose (RP2D) of Eltanexor (KPT-8602). A DLT is defined as a treatment-related toxicity based on Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

Group	Value	95% CI
All Participants in Phase I	NA

Phase 1: Number of Participants Who Have Grades 3 and/or 4 Dose-limiting Toxicity (DLT) at the Recommended Phase 2 Dose (RP2D) Primary · First 28 days of study treatment

Participants enrolled in phase I will have the grades and types of toxicity reported at each dose level to determine the RP2D. Grade 1 is mild. Grade 2 is moderate. Grade 3 is severe. Grade 4 is life-threatening. Grade 5 is death related to adverse event. Toxicity was assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0).

Grade 3 Platelet Count Decreased

Group	Value	95% CI
Phase I:Dose escalation of Eltanexor (KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level 1	1
Phase I:Dose escalation of Eltanexor(KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level -1	0

Grade 4 Platelet Count Decreased

Group	Value	95% CI
Phase I:Dose escalation of Eltanexor (KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level 1	1
Phase I:Dose escalation of Eltanexor(KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level -1	0

Grade 4 Neutrophil Count Decreased

Group	Value	95% CI
Phase I:Dose escalation of Eltanexor (KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level 1	1
Phase I:Dose escalation of Eltanexor(KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level -1	0

Phase 1: Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). Secondary · From the first study intervention through 30 days after the study agent (s) was/were administered, an average of 8.5 months.

Here is the number of participants with serious and/or non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent

Group	Value	95% CI
Phase I:Dose escalation of Eltanexor (KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level 1	2
Phase I:Dose escalation of Eltanexor(KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level -1	2
Participant Enrolled But Not Treated	0

Adverse events — posted to ClinicalTrials.gov

Time frame: All-Cause Mortality was monitored/assessed an average of 8.5 months. Adverse Events were monitored/assessed from the first study intervention through 30 days after the study agent (s) was/were administered, an average of 8.5 months.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Phase I:Dose Escalation of Eltanexor (KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level 1

Serious: 0/2 (0%)

Deaths: 0/2

Phase I:Dose Escalation of Eltanexor(KPT-8602) for High-Risk Myelodysplastic Syndromes-Dose Level -1

Serious: 0/2 (0%)

Deaths: 1/2

Participant Enrolled But Not Treated

Serious: 0/1 (0%)

Deaths: 0/1

Outside Courses

Serious: 0/1 (0%)

Deaths: 0/2

Other adverse events (60 terms — click to expand)

Reaction	System	Phase I:Dose Escalation of…	Phase I:Dose Escalation of…	Participant Enrolled But N…	Outside Courses
Anemia	Blood and lymphatic system disorders	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—
Hypomagnesemia	Metabolism and nutrition disorders	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—
Oral hemorrhage	Gastrointestinal disorders	—	—	—	—
Pain	General disorders	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—
Allergic rhinitis	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—
Anxiety	Psychiatric disorders	—	—	—	—
Bloating	Gastrointestinal disorders	—	—	—	—
Chills	General disorders	—	—	—	—
Concentration impairment	Nervous system disorders	—	—	—	—
Dehydration	Metabolism and nutrition disorders	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—
Dry eye	Eye disorders	—	—	—	—
Dry mouth	Gastrointestinal disorders	—	—	—	—
Dysgeusia	Nervous system disorders	—	—	—	—
Dysuria	Renal and urinary disorders	—	—	—	—
Eosinophilia	Blood and lymphatic system disorders	—	—	—	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Fatigue	General disorders	—	—	—	—
Febrile neutropenia	Blood and lymphatic system disorders	—	—	—	—
Fever	General disorders	—	—	—	—
Gastroesophageal reflux disease	Gastrointestinal disorders	—	—	—	—
Gastrointestinal disorders - Other, specify: Stomach discomfort after taking Deferasirox.	Gastrointestinal disorders	—	—	—	—
General disorders and administration site conditions - Other, specify: Port occlusion	General disorders	—	—	—	—
Gum infection	Infections and infestations	—	—	—	—
Headache	Nervous system disorders	—	—	—	—
Hematuria	Renal and urinary disorders	—	—	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—	—	—
Hyponatremia	Metabolism and nutrition disorders	—	—	—	—
Hypotension	Vascular disorders	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—
Malaise	General disorders	—	—	—	—
Memory impairment	Nervous system disorders	—	—	—	—
Metabolism and nutrition disorders - Other, specify: Hepatic steatosis	Metabolism and nutrition disorders	—	—	—	—
Metabolism and nutrition disorders - Other, specify	Metabolism and nutrition disorders	—	—	—	—

Data from ClinicalTrials.gov NCT05918055 adverse events section.

Sponsor's own description

Background:

Myelodysplastic syndromes (MDS) are diseases that affect the bone marrow. They can inhibit the blood formation process and reduce blood cell counts. High-risk MDS can lead to leukemia. People with high-risk MDS have a low survival rate. Better treatments are needed.

Objective:

To test a study drug Eltanexor (KPT-8602), combined with another drug (Inqovi), in people with MDS.

Eligibility:

Adults aged 18 years and older with high-risk MDS that did not respond to treatment.

Design:

Participants will be screened. They will have a physical exam. They will have blood and urine tests and tests of their heart function. They may have a bone marrow biopsy: Their hip will be numbed; then a needle will be inserted to draw out a sample of soft tissue from inside the bone. They will answer questions about their quality of life. Genetic tests may be performed.

KPT-8602 and Inqovi are both tablets taken by mouth. Participants will take these drugs at home on a 28-day cycle. They will take Inqovi once a day on days 1 to 5. They will take KPT-8602 on a schedule assigned by the researcher. Participants will be given a drug diary to record each dose.

Participants will visit the clinic for an exam at least once in each cycle. Some tests, including the bone marrow biopsy, may be repeated.

Participants will continue treatment for at least 6 cycles. If their disease improves, they may continue taking the drugs after 6 cycles.

Participants will have follow-up visits at the clinic for about 8 years.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

1. Acute Myeloid Leukemia in Older Patients: From New Biological Insights to Targeted Therapies.
   Niscola P, Gianfelici V, Catalano G, Giovannini M, et al · · 2024 · cited 10× · PMID 39590121 · DOI 10.3390/curroncol31110490
2. How I treat higher-risk MDS.
   Mina A, Komrokji R. · · 2025 · cited 6× · PMID 39808802 · DOI 10.1182/blood.2024025271
3. Novel-and Not So Novel-Inhibitors of the Multifunctional CRM1 Protein.
   Aumann WK, Kazi R, Harrington AM, Wechsler DS. · · 2024 · cited 3× · PMID 39161560 · DOI 10.3389/or.2024.1427497
4. Treating Hematological Malignancies With OR-2100, an Orally Bioavailable Prodrug of Decitabine.
   Watanabe T, Kidoguchi K, Kimura S. · · 2025 · cited 2× · PMID 39837580 · DOI 10.1111/cas.16452
5. Novel small-molecule therapies for myelodysplastic syndromes with IPSS-R ⩾3.5 in patients aged 60 or older: current landscape and challenges.
   Hou K, Dong X, Niu W. · · 2025 · cited 1× · PMID 40919259 · DOI 10.1177/20406207251371298
6. Selinexor in acute myeloid leukemia: therapeutic applications and current challenges.
   Qie Z, Ma L, Tan J, Peng X. · · 2025 · cited 1× · PMID 40463909 · DOI 10.3389/fphar.2025.1602911
7. Prognostic and functional role of the nuclear export receptor 1 (XPO1) in gastrointestinal cancers: a potential novel target?
   Sokolova V, Gruber R, Pammer LM, Kocher F, et al · · 2024 · cited 1× · PMID 39729162 · DOI 10.1007/s11033-024-10169-5