Trial Outcomes & Findings for Efficacy and Safety Study of Selinexor in Relapsed or Refractory Peripheral T-cell Lymphoma or Cutaneous T-cell Lymphoma (NCT NCT02314247)

Last Updated: 2023-01-26

Results Overview

Overall Response (OR) = Complete Response (CR) + Partial Response (PR). Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment. Progression was defined as the first occurrence of progressive disease (PD) per the revised response criteria. Clinical disease progression in the absence of formal criteria for PD must be documented by a physician.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

16 participants

Primary outcome timeframe

Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Results posted on

2023-01-26

Participant Flow

Participant milestones

Participant milestones
Measure	Selinexor (PTCL) Assigned to 60 mg dose twice weekly	Selinexor (CTCL) Assigned to 60 mg dose twice weekly	Selinexor (CTCL) 8 Doses/Cycle Assigned to 60 mg dose twice weekly for weeks 1-4
Overall Study STARTED	9	6	1
Overall Study COMPLETED	0	0	0
Overall Study NOT COMPLETED	9	6	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Selinexor (PTCL) Assigned to 60 mg dose twice weekly	Selinexor (CTCL) Assigned to 60 mg dose twice weekly	Selinexor (CTCL) 8 Doses/Cycle Assigned to 60 mg dose twice weekly for weeks 1-4
Overall Study Adverse Event	2	1	0
Overall Study Withdrawal by Subject	1	3	1
Overall Study Progression of Disease	6	2	0

Baseline Characteristics

Efficacy and Safety Study of Selinexor in Relapsed or Refractory Peripheral T-cell Lymphoma or Cutaneous T-cell Lymphoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)	Total n=16 Participants Total of all reporting groups
Age, Continuous	65 years n=5 Participants	74 years n=7 Participants	69 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants	5 Participants n=7 Participants	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	7 Participants n=7 Participants	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	6 Participants n=7 Participants	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Due to the limited enrollment in this study, only the Intent to Treat (ITT) Population, consisting of all patients who received at least 1 dose of study treatment (which is identical to the Safety Population), was evaluated for efficacy.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Overall Response Rate (ORR)	11.1 Percentage of participants Interval 1.2 to 36.8	14.3 Percentage of participants Interval 1.5 to 45.3

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat population

Patients who achieved CR (disappearance of all detectable evidence of disease). Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Best Overall Response: Complete Response (CR)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat (ITT) population

Patients whose best overall response to study treatment was PR (regression of measurable disease and no new sites). Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Best Overall Response: Partial Response (PR)	1 Participants	1 Participants

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat population

Patients whose best overall response to study treatment was SD (failure to attain criteria needed for CR or PR, or to meet criteria for PD). Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Best Overall Response: Stable Disease (SD)	2 Participants	4 Participants

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat population

Patients whose best overall response to study treatment was PD. Progression was defined as the first occurrence of progressive disease (PD). Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment. Clinical disease progression in the absence of formal criteria for PD must be documented by a physician.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Best Overall Response: Progressive Disease (PD)	2 Participants	0 Participants

PRIMARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat population

Patients who could not be assessed quantitatively for disease response for any reason.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Best Overall Response: Not Evaluable (NE)	4 Participants	2 Participants

SECONDARY outcome

Timeframe: Date of start of study treatment to date of progression. Patients without documented PD are censored on date of last radiologic assessment.

Population: Those participants with stable disease, including those with partial response, as a subset of the Intent to Treat (ITT) population

Duration of time from the date of start of study treatment to the date of disease progression. Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=3 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=5 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Duration of Stable Disease, Including Patients With Partial Response	120.5 Days Interval 111.0 to 130.0	112.0 Days Interval 85.0 to 225.0

SECONDARY outcome

Timeframe: Disease response was assessed at screening and every 8 weeks (patients with PTCL); or at Cycle 1 Day 1 and every 4 weeks (patients with CTCL), until disease progression.

Population: Intent to Treat (ITT) Population

Percentage of patients who have CR, PR, or SD lasting ≥ 8 weeks. Objective disease response assessment in PTCL patients was made according to the revised response criteria based on the International Working Group (IWG) guidelines for malignant lymphoma (Cheson, 2007). Objective disease response assessment in CTCL patients was assessed according to the revised CTCL Consensus Response Criteria (Olsen, 2011) using physical examination, including the Modified Severity Weighted Assessment Tool (mSWAT) for skin assessment. CTCL Global Response Score was used as a secondary efficacy assessment.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Disease Control Rate (DCR)	33.3 Percentage of participants Interval 12.9 to 59.9	71.4 Percentage of participants Interval 40.4 to 92.1

SECONDARY outcome

Timeframe: Study treatment start date to date of disease progression or date of death. Patients without documented PD are censored on date of last radiologic assessment.

Population: Intent to Treat population.

Duration of time from start of study treatment to date of disease progression or death from any cause.

Outcome measures

Outcome measures
Measure	Selinexor (PTCL) n=9 Participants Assigned to 60 mg dose, 6 doses/cycle	Selinexor (CTCL) n=7 Participants Assigned to 60 mg dose, 6 doses/cycle (6 patients); Assigned to 60 mg dose, 8 doses/cycle (1 patient)
Progression Free Survival (PFS)	111.0 Days Interval 41.0 to 130.0	112.0 Days Interval 85.0 to 225.0

Adverse Events

Selinexor (PTCL)

Serious events: 8 serious events

Other events: 9 other events

Deaths: 3 deaths

Selinexor (CTCL)

Serious events: 1 serious events

Other events: 7 other events

Deaths: 1 deaths

Serious adverse events

Other adverse events

Additional Information

Jatin Shah, MD

Karyopharm Therapeutics Inc.

Phone: (617) 658-0600

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Selinexor (PTCL) n=9 participants at risk Assigned to 60 mg; 6 doses/cycle	Selinexor (CTCL) n=7 participants at risk Assigned to 60 mg; 6 doses/cycle (6 patients) Assigned to 60 mg; 8 doses/cycle (1 patient)
Blood and lymphatic system disorders Thrombocytopenia	22.2% 2/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Blood and lymphatic system disorders Anemia	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Blood and lymphatic system disorders Febrile Neutropenia	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Infections and infestations Pneumonia	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Infections and infestations Wound Infection Staphylococcal	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Nervous system disorders Aphasia	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Nervous system disorders Lethargy	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Respiratory, thoracic and mediastinal disorders Dyspnea	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Respiratory, thoracic and mediastinal disorders Pulmonary Embolism	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Gastrointestinal disorders Diarrhea	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Gastrointestinal disorders Rectal Haemorrhage	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Injury, poisoning and procedural complications Fall	0.00% 0/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	14.3% 1/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Metabolism and nutrition disorders Decreased Appetite	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Musculoskeletal and connective tissue disorders Muscular Weakness	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Psychiatric disorders Psychotic Disorder	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Renal and urinary disorders Renal Impairment	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Skin and subcutaneous tissue disorders Pruritus	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
Vascular disorders Deep Vein Thrombosis	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
General disorders Fatigue	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.
General disorders Pyrexia	11.1% 1/9 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.	0.00% 0/7 • Day of onset after the first dose of study treatment through 30 days following the last dose of study treatment. All patients who received at least 1 dose of study treatment were included in the Safety Population.