Clinical Trial Results:
A Phase 2, Open label Study to Assess the Efficacy and Safety of Tenalisib
(RP6530), a Novel PI3K Dual δ/γ Inhibitor, in Patients with
Relapsed/Refractory Chronic Lymphocytic Leukemia (CLL)
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Summary
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EudraCT number |
2019-003622-25 |
Trial protocol |
PL |
Global end of trial date |
02 Oct 2020
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Results information
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Results version number |
v1(current) |
This version publication date |
21 Aug 2021
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First version publication date |
21 Aug 2021
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Other versions |
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Summary report(s) |
Summary clinical report |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
RP6530-1901
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT04204057 | ||
WHO universal trial number (UTN) |
- | ||
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Sponsors
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Sponsor organisation name |
Rhizen Pharmaceuticals AG
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Sponsor organisation address |
Steinentorstrasse 23,, Basel, India, 4051
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Public contact |
Ajit Nair / SVP & Head Clinical R&D, Rhizen Pharmaceuticals AG, +41 4033241041, kr@rhizen.com
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Scientific contact |
Ajit Nair / SVP & Head Clinical R&D, Rhizen Pharmaceuticals AG, 9703031098 4033241041, kr@rhizen.com
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
22 Apr 2021
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
02 Oct 2020
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Global end of trial reached? |
Yes
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Global end of trial date |
02 Oct 2020
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Was the trial ended prematurely? |
No
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General information about the trial
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Main objective of the trial |
To assess the anti-tumor activity of Tenalisib as determined by the
objective response rate (ORR) and duration of response (DoR)
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Protection of trial subjects |
The DRC was constituted by the sponsor to review the safety and efficacy data. The committee consisted of PI of respective sites, sponsor representative, sponsor’s medical expert, and a statistician. The DRC reviewed the efficacy and safety data at regular intervals to assess the safety and efficacy of the study drug and provided the recommendations.
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Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
19 Nov 2019
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
No
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
Poland: 9
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Country: Number of subjects enrolled |
Bulgaria: 3
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Country: Number of subjects enrolled |
Georgia: 9
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Worldwide total number of subjects |
21
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EEA total number of subjects |
12
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
9
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From 65 to 84 years |
12
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85 years and over |
0
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Recruitment
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Recruitment details |
This was a Phase II, open-label, Simon’s two-stage study design to evaluate the efficacy and safety of Tenalisib in 61 patients. Eligible patients were enrolled in order of their eligibility | ||||||||||||||
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Pre-assignment
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Screening details |
All consented subjects who undergo at least one post-consent procedure were given a unique screening number that will be used to identify the subject for all procedures that occur prior to dosing or allocation. A total of 27 patients were screened in the study, of which 21 patients were eligible and received at least one dose of Tenalisib. | ||||||||||||||
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Period 1
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Period 1 title |
No (overall period)
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Is this the baseline period? |
Yes | ||||||||||||||
Allocation method |
Not applicable
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Blinding used |
Not blinded | ||||||||||||||
Blinding implementation details |
Non-randomized open-label study
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Arms
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Arm title
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Single arm | ||||||||||||||
Arm description |
Tenalisib (800 mg BID) | ||||||||||||||
Arm type |
Experimental | ||||||||||||||
Investigational medicinal product name |
Tenalisib
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Investigational medicinal product code |
RP6530
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Other name |
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Pharmaceutical forms |
Tablet
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Routes of administration |
Oral use
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Dosage and administration details |
Tenalisib (800 mg BID) daily in a 28-day cycle
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Baseline characteristics reporting groups
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Reporting group title |
No
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Reporting group description |
Patients who have treated with at least one dose of Tenalisib | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Subject analysis sets
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Subject analysis set title |
Safety analysis set
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Subject analysis set type |
Safety analysis | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Subject analysis set description |
A patient who have received at least one dose of study drug i.e. Tenalisib
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End points reporting groups
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Reporting group title |
Single arm
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Reporting group description |
Tenalisib (800 mg BID) | ||
Subject analysis set title |
Safety analysis set
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Subject analysis set type |
Safety analysis | ||
Subject analysis set description |
A patient who have received at least one dose of study drug i.e. Tenalisib
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End point title |
overall response rate (ORR) [1] | ||||||||
End point description |
ORR is defined as the sum of complete response (CR) and partial response (PR) rates as defined by the iwCLL guideline for
CLL (Hallek et al. 2018)
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End point type |
Primary
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End point timeframe |
8 months
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| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: A priory hypothesis was set based on Simon-two-stage design that the response in the subjects in Part-1 is less than 8 then null hypotheses will be accepted and part-2 will not be initiated. Therefore, no additional statistics were performed to test this hypothesis. |
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End point title |
Duration of response (DoR) [2] | ||||||||
End point description |
DoR is defined as the interval from the first documentation of CR/PR to first documentation of definitive disease progression or death from any cause.
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End point type |
Primary
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End point timeframe |
The DoR was calculated up to a predefined cut-off date (i.e. LPLV)
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| Notes [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: A priory hypothesis was set based on Simon-two-stage design that the response in the subjects in Part-1 is less than 8 then null hypotheses will be accepted and part-2 will not be initiated. Therefore, no additional statistics were performed to test this hypothesis. |
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Adverse events information
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Timeframe for reporting adverse events |
up-to a predefined cut-off date (i.e., up-to LPLV)
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Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
23.1
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Reporting groups
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Reporting group title |
Safety analysis set
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Reporting group description |
Patients who have treated at least one dose of study drug | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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| Were there any global substantial amendments to the protocol? No | |||
Interruptions (globally) |
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| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| After evaluating the anti-tumor effect in stage 1 by the DRC committee (fewer than 8 responses seen in stage 1), as per the Simon two-stage design of the study protocol, the study would be discontinued and stage 2 was not initiated. | |||
