Targeting N-myristoylation for therapy of B-cell lymphomas.
Erwan Beauchamp, Megan C Yap, Aishwarya Iyer...
https://pubmed.ncbi.nlm.nih.gov/33093447Actively Recruiting
Phase 1
Age: 18Years +
All Genders
ID06613217
A Phase 1 Study of Oral PCLX-001 (Zelenirstat) in Relapsed/Refractory Acute Myeloid Leukemia
Led by Pacylex Pharmaceuticals · Updated on 2025-03-10
35
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
Sponsors
P
Pacylex Pharmaceuticals
Lead Sponsor
O
Ozmosis Research Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying oral zelenirstat (PCLX-001) in patients with relapsed or refractory acute myeloid leukemia (R/R AML) to find the safest and most effective dose. This Phase 1 trial includes two parts: Dose Escalation to find the minimum safe and biologically effective dose, and Dose Expansion to further evaluate safety and preliminary activity at that dose. Patients have previously received treatment for AML and are unable to benefit from other therapies. The study involves daily oral doses of zelenirstat taken in 28-day cycles, starting at 40 mg and increasing through several dose levels up to 280 mg. In Dose Escalation, small groups of patients receive ascending doses to monitor safety and drug effects, with adjustments based on observed toxicities. Dose Expansion will treat about 20 patients at the determined safe and effective dose to assess further safety and clinical response. Participants will be monitored through regular evaluations including toxicity checks, pharmacokinetic and pharmacodynamic assessments, and laboratory tests to track liver, kidney, and heart function. Their ability to take oral medication and use contraception is required. Researchers will measure the recommended dose for expansion and overall clinical responses over approximately 18 months. The study started in March 2025 and follows patients until September 2026.
CONDITIONS
Brief Title
Study of Oral PCLX-001 in R/R Acute Myeloid Leukemia
Who Can Participate
Age: 18Years +
All Genders
Eligibility Criteria
You may qualify if you...
- Ability to understand and willing to sign written informed consent before any study procedures
- Male or female patients aged 18 years or older
- Diagnosed with AML as per 2016 WHO classification
- Received at least one prior therapy for AML
- Not eligible for other therapies expected to provide clinical benefit
- ECOG performance status of 0, 1, or 2
- No use of chemotherapeutic or anti-leukemic agents during the study except specified exceptions
- Adequate liver function: total bilirubin ≤1.5x ULN except for hemolysis or Gilbert's syndrome; ALT and AST ≤2.5x ULN or ≤5x ULN for malignant liver involvement
- Adequate kidney function: eGFR >60 mL/min and creatinine ≤1.5x ULN
- Adequate cardiac function: LVEF ≥50% by echocardiography or MUGA scan
- Ability to take oral medication
- Women of childbearing potential must have a negative pregnancy test within 7 days before study drug start
- Women of childbearing potential and fertile men must agree to use two reliable contraception methods during the study and for 6 months after
- Male patients with partners of reproductive potential must use condoms and an additional contraception method during and 6 months after treatment
You will not qualify if you...
- Diagnosis of acute promyelocytic leukemia
- Known hypersensitivity to study drugs or agents used with the study
- History of significant cardiac disease including NYHA class > II heart failure, unstable or recent angina, recent myocardial infarction, or uncontrolled arrhythmias
- Uncontrolled arterial hypertension
- Moderate or severe liver impairment (Child-Pugh class B or C)
- Known uncontrolled HIV infection
- Active hepatitis B or C infection requiring treatment
- Infections of CTCAE Grade 2 not responding to therapy or serious active infections above Grade 2
- Uncontrolled seizure disorder requiring certain strong medications
- Previous or concurrent cancer distinct from AML except specified low-risk cancers
- Inability to swallow oral medications
- Malabsorption conditions affecting drug absorption
- Breastfeeding women
- Unstable acute toxic effects from prior cancer treatments
- Active radiation or antineoplastic therapy for another cancer at screening
- Prior treatment assignment in this study
- Participation in another clinical study with investigational drugs
- Substance abuse or conditions interfering with study participation
- Significant ECG abnormalities such as advanced heart block or QTc prolongation unless approved by investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
1
2
3
Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - Up to 18 months
Participants receive daily oral doses of PCLX-001 on 28-day cycles to determine the minimum safe and biologically-effective dose and to evaluate safety and preliminary clinical activity.
Daily dosing on 28-day cycles with visits as per study schedule
Trial Site Locations
Total: 1 location
1
MD Anderson Cancer Centre
Houston, Texas, United States, 77030
Actively Recruiting
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Research Team
P
Pacylex Pharmaceuticals
H
Heit, MSc
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
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Published Research Related To This Trial
Novel, First-in-Human, Oral PCLX-001 Treatment in a Patient with Relapsed Diffuse Large B-Cell Lymphoma.
Randeep Sangha, Neal M Davies, Afshin Namdar...
https://pubmed.ncbi.nlm.nih.gov/35323358Multiomics analysis identifies oxidative phosphorylation as a cancer vulnerability arising from myristoylation inhibition.
Erwan Beauchamp, Jay M Gamma, Christopher R Cromwell...
https://pubmed.ncbi.nlm.nih.gov/38715059