Trial Outcomes & Findings for Compassionate Use Study of Tenalisib (RP6530) (NCT NCT03711604)

Last Updated: 2024-05-08

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

17 participants

Primary outcome timeframe

2 years

Results posted on

2024-05-08

Participant Flow

Participant milestones

Participant milestones
Measure	Tenalisib (RP6530) Participants receive Tenalisib (RP6530) BID orally.
Overall Study STARTED	17
Overall Study COMPLETED	17
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Compassionate Use Study of Tenalisib (RP6530)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tenalisib (RP6530) n=17 Participants Participants receive Tenalisib (RP6530) BID orally.
Age, Continuous	70.82 years STANDARD_DEVIATION 9.85 • n=93 Participants
Sex: Female, Male Female	6 Participants n=93 Participants
Sex: Female, Male Male	11 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	01 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	00 Participants n=93 Participants
Race (NIH/OMB) Black or African American	02 Participants n=93 Participants
Race (NIH/OMB) White	14 Participants n=93 Participants
Race (NIH/OMB) More than one race	00 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	00 Participants n=93 Participants

PRIMARY outcome

Timeframe: 2 years

Population: A total of 17 patients received at least one dose of Tenalisib.

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

Outcome measures

Outcome measures
Measure	Tenalisib (RP6530) n=17 Participants Participants receive Tenalisib (RP6530) orally. Tenalisib (RP6530)
Treatment-related Adverse Events	4 Participants

PRIMARY outcome

Timeframe: 2 years

Population: A total of 17 patients received at least one dose of Tenalisib.

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.

Outcome measures

Outcome measures
Measure	Tenalisib (RP6530) n=17 Participants Participants receive Tenalisib (RP6530) orally. Tenalisib (RP6530)
Time to Disease Progression	8.4 Months Interval 2.77 to 24.01

Adverse Events

Tenalisib (RP6530)

Serious events: 5 serious events

Other events: 16 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Tenalisib (RP6530) n=17 participants at risk Participants receive Tenalisib (RP6530) BID orally.
Nervous system disorders Seizure	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Nervous system disorders Hemorrhage intracranial	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Infections and infestations Covid-19	11.8% 2/17 • Number of events 3 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Renal and urinary disorders Renal Cell Carcinoma	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.

Other adverse events

Other adverse events
Measure	Tenalisib (RP6530) n=17 participants at risk Participants receive Tenalisib (RP6530) BID orally.
Nervous system disorders Spinal cord compression	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Nervous system disorders Post herpetic neuralgia	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Nervous system disorders Peripheral neuropathy	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Nervous system disorders Dysgeusia	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Investigations Elevated Aspartate aminotransferase	11.8% 2/17 • Number of events 3 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Investigations Elevated Blood alkaline phosphotase	11.8% 2/17 • Number of events 5 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Investigations Neutrophile count decreased	11.8% 2/17 • Number of events 7 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Investigations Platelet count decreased	11.8% 2/17 • Number of events 9 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Investigations Elevated Troponin	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Exfoliative Rash	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Erythema	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Actinic keratosis	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Rash pruritic	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Rash	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Skin and subcutaneous tissue disorders Pruritis	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
General disorders Oedema	11.8% 2/17 • Number of events 2 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
General disorders Obstruction	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
General disorders Fatigue	11.8% 2/17 • Number of events 2 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Infections and infestations Covid-19	11.8% 2/17 • Number of events 2 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Gastrointestinal disorders Abdominal discomfort	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Gastrointestinal disorders Diarrhoea	11.8% 2/17 • Number of events 2 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Gastrointestinal disorders Constipation	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Gastrointestinal disorders Nausea	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Gastrointestinal disorders Vomiting	5.9% 1/17 • Number of events 2 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Blood and lymphatic system disorders Thrombocytopenia	11.8% 2/17 • Number of events 3 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Musculoskeletal and connective tissue disorders Back pain	11.8% 2/17 • Number of events 3 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Musculoskeletal and connective tissue disorders Arthralgia	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Cardiac disorders Myocardial infarction	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Cardiac disorders Supraventriculat extrasystoles	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Respiratory, thoracic and mediastinal disorders Pnuemonitis	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Respiratory, thoracic and mediastinal disorders Epistaxis	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Psychiatric disorders Mental status changes	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Psychiatric disorders Insomnia	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Basal cell carcinoma	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Vascular disorders Haematoma	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Injury, poisoning and procedural complications Wound	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Renal and urinary disorders Renal disorder	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.
Surgical and medical procedures Nephrectomy	5.9% 1/17 • Number of events 1 • 2 years Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and which does not necessarily have a causal relationship with this treatment. This includes AE, SAE and adverse event of special interest.

Additional Information

Dr.Prajak Barde

Rhizen Pharmaceuticals AG

Phone: 9820503970

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place