Clinical trial NCT02017613: Safety and Efficacy Study of a Dual PI3K Delta/Gamma Inhibitor in Hematological Malignancies
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Lymphoma, B-Cell
T-Cell Lymphoma
Keywords: CLL, SLL, NHL, SLL, PTCL, CTCL, MM, AML
Lead sponsor: Rhizen Pharmaceuticals SA
Sponsor class: INDUSTRY
First posted: 2013-12-23
Enrollment: 35
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Maximum tolerated dose (MTD) and pharmacokinetics (PK) of RP6530
timeFrame: 28 days
Secondary outcomes:
measure: Clinical response following administration of RP6530
timeFrame: 8 weeks
Other outcomes:
Locations:
city: Paris
country: France
facility: Rhizen Trial Site
city: Milano
country: Italy
facility: Rhizen Trial Site 1
city: Milano
country: Italy
facility: Rhizen Trial Site 2
Study officials:
affiliation: Ospedale San Raffaele s.r.l.
name: Andrés JM Ferreri, MD
role: STUDY_CHAIR
affiliation: Humanitas Clinical and Research Centre
name: Carmelo Carlo-stella, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Hopital Necker-Enfants Malades
name: Richard Delarue, MD
role: PRINCIPAL_INVESTIGATOR
References: