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Pitavastatin

Phase 1

Acute Myeloid Leukemia | Small molecule | Oncology |United Therapeutics Corporation|Last Updated: Sep 11, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment6

FDA Designations

No designations recorded

Clinical trial landscape

Pitavastatin · 1 trial · 6 indications

Phase 1 1
NCT04512105Pitavastatin in Combination With Venetoclax for Chronic Lymphocytic Leukemia or Acute Myeloid LeukemiaAcute Myeloid Leukemia
COMPLETED6 Analytics
PHASE1COMPLETED
Pitavastatin in Combination With Venetoclax for Chronic Lymphocytic Leukemia or Acute Myeloid Leukemia
Acute Myeloid Leukemia
NCT045121056
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Study Endpoints

Primary Endpoints

Maximum Tolerated Dose for PIT administered with VEN-containing Standard of Care (SOC) regimens
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.

Determination of the maximum tolerated dose (MTD) will be utilized to evaluate the safety and tolerability of adding to PIT to treatment with stable doses of VEN ± anti-CD20 antibodies (patients with CLL) or VEN with hypomethylating agents (patients with AML).

Recommended Phase 2 Dose for PIT administered with VEN-containing SOC regimens
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.

Determination of the recommended Phase 2 dose (RP2D) will be utilized to evaluate the safety and tolerability of adding to PIT to treatment with stable doses of VEN ± anti-CD20 antibodies (patients with CLL) or VEN with hypomethylating agents (patients with AML).

Identifying Dose Limiting Toxicities (DLTs) for PIT administered with VEN-containing SOC regimens
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.

To evaluate the safety and tolerability of administering PIT in combination with VEN-containing SOC in patients with AML or CLL .

Identifying overall Adverse Event Profile of PIT when given with VEN-containing SOC regimens
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.

To evaluate the adverse events are based on the CTCAE (NCI Common Terminology Criteria for Adverse Events) Version 5.0.

Complete Response Rate
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.

Primary efficacy endpoint is the complete response rate of subjects who receive PIT when given with VEN-containing SOC regimens. The 2018 International Working Group for Chronic Lymphocytic Leukemia (iwCLL) and 2017 European LeukemiaNet (ELN) definitions of Complete Response (CR) will be utilized to determine the CR rate.

Secondary Endpoints

Partial Response Rates
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.
Stable Disease Rates
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.
Progressive Disease Rates
From the start date of treatment until 30 days after removal of treatment due to disease progression, toxicity, delay of treatment, or withdrawal of treatment, whichever came first, an average of 2 years.
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Dose Level -1 (DL-1)EXPERIMENTALPatients receive Pitavastatin (PIT) 1 mg PO daily. For CLL patients, they will also receive stabilized Venetoclax (VEN) 400mg PO daily. For AML patients, they will VEN mg PO daily when dosing in combination with azacitidine or decitabine. The 1 mg/day dose level will be held in reserve to allow dose reduction in those patients who cannot tolerate DL1.
Dose Level 1 (DL1)EXPERIMENTALPatients receive Pitavastatin (PIT) 2 mg PO daily. For CLL patients, they will also receive stabilized Venetoclax (VEN) 400mg PO daily. For AML patients, they will VEN mg PO daily when dosing in combination with azacitidine or decitabine. This is the starting dose level for the study.
Dose Level 2 (DL2)EXPERIMENTALPatients receive Pitavastatin (PIT) 4 mg PO daily. For CLL patients, they will also receive stabilized Venetoclax (VEN) 400mg PO daily. For AML patients, they will VEN mg PO daily when dosing in combination with azacitidine or decitabine. If DL1 is well tolerated, the next cohort will progress to this dose level.

Interventions

NameTypeDescription
PitavastatinDRUGGiven PO
VenetoclaxDRUGGiven PO
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Pathologically confirmed AML or CLL, otherwise eligible for VEN-containing therapy at Screening 1. Newly diagnosed patients with AML deemed ineligible for intensive induction chemotherapy (age 75 and older or \< 75 years of age with comorbidities that preclude the use of ...

Countries:United States
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Frequently asked questions about Pitavastatin

What is Pitavastatin used for in Acute Myeloid Leukemia?

Pitavastatin is being studied as a treatment for Acute Myeloid Leukemia (AML). It is a small molecule being developed by United Therapeutics Corporation. In a completed Phase 1 clinical trial, pitavastatin was evaluated in combination with venetoclax for patients with AML or chronic lymphocytic leukemia.

What does Pitavastatin target?

Pitavastatin is a small molecule being investigated in oncology. It was studied in a Phase 1 clinical trial for Acute Myeloid Leukemia and chronic lymphocytic leukemia. The trial did not specify a molecular target, but the drug is being evaluated for its potential anti-cancer effects in combination with venetoclax.

Who makes Pitavastatin?

Pitavastatin is being developed by United Therapeutics Corporation, a biopharmaceutical company. The company is investigating the drug for use in Acute Myeloid Leukemia. The Phase 1 clinical trial for this indication was conducted in the United States.

What phase is Pitavastatin in for Acute Myeloid Leukemia?

Pitavastatin is in Phase 1 clinical development for Acute Myeloid Leukemia. It is an investigational drug and has not been approved for this indication. A Phase 1 trial studying pitavastatin in combination with venetoclax for AML has been completed.

What clinical trials is Pitavastatin in?

Pitavastatin was studied in a completed Phase 1 clinical trial with the identifier NCT04512105. The trial evaluated pitavastatin in combination with venetoclax for patients with Acute Myeloid Leukemia or chronic lymphocytic leukemia. The study enrolled 6 participants and was conducted in the United States.

Is Pitavastatin the same as any other drug?

Pitavastatin is the drug name used in the clinical trial for Acute Myeloid Leukemia. No alternative names for this drug were provided in the trial information. The drug is being developed by United Therapeutics Corporation.