Compassionate Use Study of Tenalisib (RP6530)

An Open Label, Compassionate Use Study of Tenalisib (RP6530) in Patients Currently Receiving Treatment on Tenalisib Trials in Hematological Malignancies

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.

Study Overview

• Status: Completed
• Conditions: Hematological Malignancies
• Intervention / Treatment: Drug: Tenalisib

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Georgia
  • Tbilisi, Georgia
    • Ltd. M.Zodelava Hematology Centre
  • Tbilisi, Georgia
    • Medivest - Institute of Hematology and Transfusiology
• Poland
  • Chorzów, Poland, 41-503
    • Silesian Healthy Blood Clinic Grosicki, Grosicka Sp.J.
• United States
  • California
    • San Francisco, California, United States, 94143
      • University of California, Hellen Diller Family Comprehensive Cancer Center
  • Colorado
    • Denver, Colorado, United States, 80045
      • Division of Hematology, University of Colorado,
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic Taussig Cancer Institute
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tenalisib; Participants receive Tenalisib (RP6530) BID Orally	Drug: Tenalisib; BID Orally; Other Names: RP6530

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	2 years
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.	2 years

Collaborators and Investigators

• Sponsor: Rhizen Pharmaceuticals SA

Study record dates

Study Major Dates

• Study Start (Actual): November 28, 2018
• Primary Completion (Actual): March 25, 2023
• Study Completion (Actual): March 25, 2023

Study Registration Dates

• First Submitted: October 15, 2018
• First Submitted That Met QC Criteria: October 16, 2018
• First Posted (Actual): October 18, 2018

Study Record Updates

• Last Update Posted (Actual): May 8, 2024
• Last Update Submitted That Met QC Criteria: April 15, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Compassionate Use; Hematological Malignancies; Tenalisib; RP6530
• Additional Relevant MeSH Terms: Neoplasms by Site; Hematologic Diseases; Neoplasms; Hematologic Neoplasms; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Phosphoinositide-3 Kinase Inhibitors; Tenalisib
• Other Study ID Numbers: RP6530-1803

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No