Trial Outcomes & Findings for Efficacy and Safety of Tenalisib (RP6530) in Patients With Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL) (NCT NCT04204057)

NCT ID: NCT04204057

Last Updated: 2024-08-13

Results Overview

Per Response Evaluation Criteria as defined by iwCLL guideline for CLL: Complete Response (CR), all parameters should be regressed to normal (lymph nodes ≥ 1.5 cm; spleen size \<13 cm; liver size normal; no constitutional symptoms; circulating lymphocyte count normal; platelet count ≥ 100 x 109 /L; Hemoglobin ≥ 11.0 g/dL). For partial response, at least two of the parameters (lymph nodes, liver and/or spleen size, constitutional symptoms, circulating lymphocyte count) and one parameter (platelet count, hemoglobin) need to improve if previously abnormal; Overall Response (OR) = CR + PR."

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

21 participants

Primary outcome timeframe

7 Months

Results posted on

2024-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	Tenalisib Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Overall Study STARTED	21
Overall Study COMPLETED	21
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy and Safety of Tenalisib (RP6530) in Patients With Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tenalisib n=21 Participants Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	11 Participants n=5 Participants
Age, Continuous	66.08 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	21 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Poland	9 participants n=5 Participants
Region of Enrollment Georgia	9 participants n=5 Participants
Region of Enrollment Bulgaria	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 7 Months

Population: Patients who received at least 1 dose of study medication and provided at least 1 post-baseline efficacy assessment

Outcome measures

Outcome measures
Measure	Tenalisib n=21 Participants Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Overall Response Rate (ORR)	33.3 percentage of participants Interval 14.59 to 56.97

PRIMARY outcome

Timeframe: 7 Months

Population: Patients who received at least 1 dose of study medication and provide at least post-baseline efficacy assessment

Duration of response (DOR): DOR is defined as the interval from the first documentation of CR/PR to the first documentation of definitive disease progression or death from any cause. Progression disease is defined using iwCLL criteria as at least one of the criteria of parameters (i.e., lymph nodes increase ≥ 50% from baseline or from response; liver and/or spleen size increase ≥ 50% from baseline or from response; any constitutional symptoms; circulating lymphocyte count increase ≥ 50% over baseline) or criteria of parameters (i.e., platelet count decrease of ≥ 50% over baseline secondary to CLL; hemoglobin decrease of ≥ 50% over baseline secondary to CLL) should be met.

Outcome measures

Outcome measures
Measure	Tenalisib n=21 Participants Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Duration of Response (DoR)	143 days Interval 87.0 to 148.0

SECONDARY outcome

Timeframe: 7 Months

Population: Patients who received at least 1 dose of study medication

Summary of Treatment-Emergent Adverse Events-(Causality All). Patients will be monitored for adverse events and both related and as well as non-related adverse events will be captured during the study. All adverse events (irrespective of causality) will be reported.

Outcome measures

Outcome measures
Measure	Tenalisib n=21 Participants Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Number of Participants With Treatment-emergent Adverse Events as Assessed by CTCAE Criteria v5.0	15 participants

SECONDARY outcome

Timeframe: 7 months

Population: Patients who received at least 1 dose of study medication and provide at least 1 post-baseline efficacy assessment

Progression-free survival (PFS): PFS is defined as the interval from first dose to first documentation of definitive disease progression or death from any cause.

Outcome measures

Outcome measures
Measure	Tenalisib n=21 Participants Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Progression Free Survival (PFS)	205 days Interval 198.0 to 205.0

Adverse Events

Tenalisib

Serious events: 3 serious events

Other events: 15 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Tenalisib n=21 participants at risk Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Blood and lymphatic system disorders Anemia	4.8% 1/21 • Number of events 1 • 7 months
Infections and infestations Pneumonia	9.5% 2/21 • Number of events 3 • 7 months

Other adverse events

Other adverse events
Measure	Tenalisib n=21 participants at risk Patients receive Tenalisib 800 mg BID, Orally in 28-Day cycle for 7 cycles Tenalisib: Tenalisib 800 mg BID, Orally
Blood and lymphatic system disorders Anemia	28.6% 6/21 • Number of events 10 • 7 months
Blood and lymphatic system disorders Neutropenia	23.8% 5/21 • Number of events 18 • 7 months
Blood and lymphatic system disorders Thrombocytopenia	9.5% 2/21 • Number of events 3 • 7 months
Gastrointestinal disorders Diarrhea	9.5% 2/21 • Number of events 2 • 7 months
Investigations Alanine aminotransferase increased	19.0% 4/21 • Number of events 7 • 7 months
Investigations Aspartate aminotransferase increased	19.0% 4/21 • Number of events 6 • 7 months
Investigations Blood alkaline phosphatase increased	9.5% 2/21 • Number of events 3 • 7 months

Additional Information

Prajak Barde MD

Rhizen Pharmaceuticals

Phone: +41325800175

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place