Safety and Efficacy Study of a Dual PI3K Delta/Gamma Inhibitor in Hematological Malignancies

NCT02017613 — Phase 1, Completed, 35 participants, Rhizen Pharmaceuticals SA

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A Phase I, Dose Escalation Study to Evaluate Safety and Efficacy of RP6530, a Dual PI3K Delta/Gamma Inhibitor, in Patients With Relapsed or Refractory Hematologic Malignancies

Registry	NCT02017613, acronym PI3K
Status	Completed
Phase	Phase 1
Study type	Interventional; na, single group, masking: none, treatment
Enrollment	35 (Actual)
Sponsor	Rhizen Pharmaceuticals SA (Industry)
Conditions	Lymphoma, B-Cell, T-Cell Lymphoma
Interventions	RP6530 (Drug)
Dates	start 2013-11-01, primary completion 2016-05-01, completion 2016-05-01, first posted 2013-12-23, last updated 2016-06-24
Locations	3 sites in France, Italy

-Summary

The objective of this study is to evaluate the safety and efficacy of RP6530, a dual PI3K delta/gamma inhibitor in patients with hematologic malignancies.

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+Detailed description

The Maximum tolerated dose (MTD) will be determined based on the safety, pharmacokinetic (PK) and efficacy data. Safety analyses include AE's, AE's related to the drug, SAE's, laboratory values, vitals/ ECG and dose limiting toxicity (DLT). PK include measurement of peak plasma concentration (Cmax), area under the plasma concentration versus the time curve (AUC), time of maximum concentration observed (Tmax). Efficacy analyses include overall response rate (ORR) and duration of response (DOR).

+Arms and interventions

Arm	Type	Interventions	Description
Single arm	Experimental	Drug: RP6530	RP6530 administered orally

RP6530 (Drug), also known as PI3k Delta/ Gamma inhibitor: Escalating doses starting at 25 mg BID

+Outcome measures

Primary outcomes

Maximum tolerated dose (MTD) and pharmacokinetics (PK) of RP6530 [28 days]: * To access maximum tolerated dose by clinical laboratory assessments, adverse events and dose limiting toxicities. * PK parameter AUC, Cmax, tmax, t1/2 will be determined.

Secondary outcomes

Clinical response following administration of RP6530 [8 weeks]: Overall response rate (ORR) and duration of response (DOR).

+Eligibility

Sex: All, ages: 18 Years to any

Inclusion Criteria:

* Refractory to or relapsed after at least 1 prior treatment line.
* ECOG performance status ≤2
* Patients must be ≥18 years of age
* Able to give a written informed consent.

Exclusion Criteria:

* Any cancer therapy in the last 4 weeks or limited palliative radiation <2 weeks
* Patients with HBV, HCV or HIV infection
* Autologous hematologic stem cell transplant within 3 months of study entry. Allogeneic hematologic stem cell transplant within 12 months.
* Previous therapy with GS-1101 (CAL-101, idelalisib), IPI-145, TGR-1202 or any drug that specifically inhibits PI3K/ mTOR (including temsirolimus, everolimus), AKT or BTK Inhibitor (including Ibrutinib).
* Patients on immunosuppressive therapy including systemic corticosteroids.
* Patients who are receiving chronic systemic anticoagulation therapy (warfarin sodium or heparin, etc.).
* Patients with known history of liver disorders.
* Patients with uncontrolled Diabetes Type I or Type II
* Any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study.
* Women who are pregnant or lactating.

+Locations (3)

Facility	City	Country
Rhizen Trial Site	Paris	France
Rhizen Trial Site 1	Milan	Italy
Rhizen Trial Site 2	Milan	Italy

+Investigators

Andrés JM Ferreri, MD, Study chair, Ospedale San Raffaele s.r.l.
Carmelo Carlo-stella, MD, Principal investigator, Humanitas Clinical and Research Centre
Richard Delarue, MD, Principal investigator, Hopital Necker-Enfants Malades