Hematological Malignancies
Conditions
Keywords
Hematological Malignancies, Tenalisib, RP6530, Compassionate Use
Brief summary
Tenalisib has been evaluated as an investigational new drug in number of early clinical studies in patients with relapsed/refractory hematological malignancies and demonstrated acceptable safety and promising efficacy in these patients. Since these advanced relapsed/refractory patients have limited therapeutic options, it is reasonable to continue Tenalisib in responding patients post completion of their participation in previous clinical studies.
Interventions
DRUGTenalisib
BID Orally
Sponsors
Rhizen Pharmaceuticals SA
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Patients must be currently receiving treatment with Tenalisib on a previously approved protocol. 2. Patients must have had at least one efficacy evaluation in previous study and should have achieved either SD, PR or CR. 3. Patients must have completed at least 6 cycles of Tenalisib in previous study 4. Ability to swallow and retain oral medication. 5. Female patients of child-bearing potential must consent to use two medically acceptable methods of contraception. 6. Male patients must be willing to use adequate contraceptive measures 7. Willingness and ability to comply with trial and follow-up procedures. 8. Willingness to provide new written informed consent.
Exclusion criteria
1. Patient has been discontinued from their previous Tenalisib study 4 weeks prior to entering the compassionate use trial. 2. Patient progressed while receiving Tenalisib therapy in his/her previous study. 3. Pregnant or lactating woman. 4. Inability or unwillingness to comply with study and/or follow-up procedures outlined in the protocol. 5. Concurrent condition that in the investigator's opinion would jeopardize compliance with the protocol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-related Adverse Events | 2 years | To evaluate the safety and tolerability of Tenalisib as single agent. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 |
| Time to Disease Progression | 2 years | Number of patients with a time to progression. The time to progression is calculated from the day of enrollment in the study to disease progression or death due to any cause. |
Countries
Georgia, Poland, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Tenalisib (RP6530) Participants receive Tenalisib (RP6530) BID orally. | 17 |
| Total | 17 |
Baseline characteristics
| Characteristic | Tenalisib (RP6530) |
|---|---|
| Age, Continuous | 70.82 years STANDARD_DEVIATION 9.85 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 01 Participants |
| Race (NIH/OMB) Black or African American | 02 Participants |
| Race (NIH/OMB) More than one race | 00 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 00 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 00 Participants |
| Race (NIH/OMB) White | 14 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 11 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 17 |
| other Total, other adverse events | 16 / 17 |
| serious Total, serious adverse events | 5 / 17 |
Outcome results
Primary
Time to Disease Progression
Number of patients with a time to progression. The time to progression is calculated from the day of enrollment in the study to disease progression or death due to any cause.
Time frame: 2 years
Population: A total of 17 patients received at least one dose of Tenalisib.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Tenalisib (RP6530) | Time to Disease Progression | 8.4 Months |
Primary
Treatment-related Adverse Events
To evaluate the safety and tolerability of Tenalisib as single agent. Number of participants with treatment-related adverse events as assessed by CTCAE v4.0
Time frame: 2 years
Population: A total of 17 patients received at least one dose of Tenalisib.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Tenalisib (RP6530) | Treatment-related Adverse Events | 4 Participants |