A Pilot Trial of Atorvastatin in Tumor Protein 53 (p53) -Mutant and p53 Wild-Type Malignancies

Joaquina Baranda

Status and phase

Active, not recruiting

Phase 1

Conditions

Solid Tumor

Malignant Disease

Relapsed Hematologic Malignancy

Cancer

Myelodysplastic Syndromes

Acute Myeloid Leukemia

Treatments

Drug: Atorvastatin

Study type

Interventional

Funder types

Other

Identifiers

NCT03560882

IIT-2018-p53Atorva

Details and patient eligibility

About

This is a window-of-opportunity trial to determine if atorvastatin given for 1 to 4 weeks at a dose of 80 milligrams per day (mg/day) is sufficient to decrease the level of conformational mutant tumor protein 53 (p53) in malignant diseases (solid tumor and relapsed Acute Myeloid Leukemia (AML)).

Enrollment

50 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Ability of participant to understand this study, and participant to sign a written informed consent. Legally authorized representative is not allowed to sign consent for participant.
Participants with tumor protein 53 (TP53) immunohistochemistry (IHC)-positive tumors
Participants whose screening IHC shows TP53-IHC-negative including wild type (WT) and null.
Participants with histologic or cytologic confirmation of any malignant disease who are planning and eligible to undergo surgical resection. For participants with Solid Tumors Only
Participants with previously treated acute myeloid leukemia (AML) are eligible if they relapse and are in between two treatment regimens
No concurrent or recent (within 30 days) use of systemic therapy including chemotherapy, immunotherapy, hormonal therapy, cancer vaccine, or local therapy for the cancer.
Formalin-fixed paraffin-embedded (FFPE) tumor tissue deemed adequate for IHC analysis and next generation sequencing (NGS) are required. Bone marrow aspirate tissue samples from participants with AML are required.
Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 or 1
Adequate organ and marrow function
A negative urine or serum pregnancy test within 7 days before Day 1 dose of study medication, if female participant is of childbearing potential.
Women of child-bearing potential and men with partners of child-bearing potential must agree to practice sexual abstinence, or to use two forms of adequate contraception (hormonal AND barrier method of birth control) prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. If a woman becomes pregnant or suspects she is pregnant while participating in this study, she should inform her treating physician immediately.

Exclusion criteria

Current or anticipated use of other investigational agents while participating in this study.
Pregnant or breast feeding.
Diagnosis of squamous cell cancer of the oropharynx
Previous malignancies (except non-melanoma skin cancers, and the following in situ cancers: bladder, gastric, colon, endometrial, cervical/dysplasia, melanoma, or breast), unless a complete remission was achieved at least 2 years prior to study entry AND no additional therapy is required during the study period
Prior use of statins in the past 30 days.
History of rhabdomyolysis
Active liver disease
Participants who currently consume substantial quantities of alcohol (Male, more than 4 drinks a day, Female, more than 2 drinks a day)
Concurrent use of drugs associated with myopathy
Hypersensitivity to atorvastatin or any component of the formulation
Untreated hypothyroidism
Inability to comply with study and follow-up procedures as judged by the Investigator