Phase 2 Study of Paxalisib for Recurrent or Refractory PCNSL
This study is testing a drug called Paxalisib (GDC-0084) for people with primary central nervous system lymphoma (PCNSL) that has come back or not responded to previous treatments. Researchers want to see how well Paxalisib works in about 25 participants. Success in this study means seeing a certain number of participants respond to the treatment. You may be able to join if you are 18 or older and meet other specific health requirements. The study is currently unclear on its recruitment status.
- Study design
- This is an open-label, Phase 2 study involving about 25 participants. It aims to determine how effective Paxalisib (GDC-0084) is.
- What's involved
- You would take Paxalisib daily for cycles of 28 days, for up to 24 months, as long as there are no serious side effects and your disease doesn't worsen. This includes screening, study treatment, evaluations, and follow-up visits.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for up to 24 months to measure how well the treatment works.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT04906096
Paxalisib (GDC-0084) In Recurrent Or Refractory PCNSL
Recruiting
PHASE2Ages 18+InterventionalTreatmentLakshmi Nayak, MDInvestigator-initiated
~25 participants
Updated 2026-08-05 on ClinicalTrials.gov
What's tested:PAXALISIB
At a glance
Recruiting sites
1 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with Objective Response Rate (ORR)
Measured over Up to 24 Months
Conditions
NCT04906096
Where you'd take part
This study runs at 2 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Dana Farber Cancer Institute
Boston, Massachusettsstudy coordinator listed
Recruiting
Brigham and Women's Hospital
Boston, Massachusettsno site contact published
Not yet recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Study leadership
- Lakshmi Nayak, MD · PRINCIPAL_INVESTIGATOR · Dana-Farber Cancer Institute
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Want this trial checked against your situation?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Participants must be able to understand and willing to sign a written informed consent document.
Participant must have signed and dated an IRB/IEC approved written informed consent form in accordance with regulatory and institutional guidelines. This must be obtained before the performance of any protocol-related procedures that are not part of normal subject care.
Participant must be willing and able to comply with scheduled visits, treatment schedule, laboratory tests, and other requirements of the study.
Participants must be at least 18 years old on day of signing informed consent.
Participants must have a Karnofsky Performance Status (KPS) ≥ 70
Participants must have histologically confirmed R/R primary DLBCL CNS lymphoma (from brain biopsy, CSF or vitreous biopsy).
Participants should have evidence of refractory or recurrent disease on MRI with measurable or evaluable enhancing disease.
Participants must have recovered to ≤ grade 1 or pre-treatment baseline from clinically significant toxic effects of prior therapy; exception, participants with ≤ grade 2 neuropathy may be eligible.
Participant with dexamethasone requirement of ≤ 8mg/day or bioequivalent with corticosteroid usage at a stable or decreasing dose 2 weeks prior to screening.
Participants must be able to undergo MRI.
Participants must demonstrate adequate as defined below (all screening labs should be performed within 14 days of treatment initiation):
Hematology
White Blood Count (WBC) ≥ 2 K/µL
Platelet count ≥ 100 K/µL
Absolute Neutrophil Count ≥ 1.5 K/µL
Hemoglobin \> 9.0 g/dL or ≥ 5.6 mmol/L (Criteria must be met without erythropoietin dependency and without packed red blood cell (pRBC) transfusion within last 2 weeks)
Biochemistry
Serum creatinine ≤1.5 x institutional ULN OR Measured or calculated creatinine clearance ≥30 mL/min for participant with creatinine levels \>1.5 × institutional ULN (Creatinine clearance should be calculated per institutional standard)
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN (≤5 × ULN for participants with liver metastases)
Total bilirubin (TBILI) ≤ 1.5 x institutional ULN (except subjects with Gilbert Syndrome who must have a total bilirubin level of \< 3.0 x institutional ULN) OR Direct bilirubin ≤ULN for participants with total bilirubin levels \>1.5 × ULN)
Women of childbearing potential (WOCBP) must have a negative serum pregnancy within 72 hours prior to registration.
WOCBP who are sexually active must use highly effective methods of contraception during treatment and for 28 days after the last dose of paxalisib. For male subjects with a pregnant or non-pregnant WOCBP partner, contraception measures are required during treatment and for 28 days after the last dose of paxalisib.
Established use of oral, injected, or implanted hormonal methods of contraception
Correctly placed intrauterine device (IUD) or intrauterine system (IUS)
Male condom or female condom used WITH a spermicide (i.e., foam, gel, film, cream)
Male sterilization with appropriately confirmed absence of sperm in the post-vasectomy ejaculate
Bilateral tubal ligation or bilateral salpingectomy
Exclusion
Participants unable to undergo MRI brain.
Participants with active systemic disease.
Participants with uncontrolled intercurrent illness.
Participants with prior exposure to mTOR/PI3K inhibitors
Prior malignancy (or any other malignancy requiring active treatment), except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, superficial bladder cancer or other cancer from which the subject has been disease free for ≥ 3 years.
Participants who have received prior systemic anti-cancer therapy including investigational agents or radiotherapy within 4 weeks OR 5 half-lives prior to dosing, whichever is shorter. Note: Participants must have recovered from all AEs due to previous therapies to ≤Grade 1 or baseline. Participants with ≤Grade 2 neuropathy may be eligible.
Participants who have difficulty with or are unable to swallow oral medication or have significant gastrointestinal disease that would limit absorption of oral medication.
Known history of infection with HIV, prior history of PML or any active significant infection (eg, bacterial, viral, or fungal).
Known history of hypersensitivity or anaphylaxis to paxalisib including active product or excipient components.
Requires treatment with a strong cytochrome P450 3A4 (CYP3A4) inhibitor/inducer which may have an effect of the metabolism of paxalisib.
Participants with uncontrolled medical comorbidities per investigator discretion including but not limited to interstitial lung disease, severe dyspnea at rest or requiring oxygen therapy, pre-exisiting Crohn's disease or ulcerative colitis or pre-existing chronic condition resulting in baseline grade 2 or higher diarrhea.
Participants with type I diabetes mellitus, participants with uncontrolled type II diabetes mellitus,despite being on oral anti-diabetic medication. , participants with Type II diabetes mellitus that are well controlled on insulin . Uncontrolled diabetes is defined as HbA1c \>9% in addition to fasting glucose\>140mg/dL on at least 2 occasions within 14 days prior to registration
Participants with uncontrolled hypertension despite optimal medical management (per investigator's assessment).
Hepatitis B or C serologic status: subjects who are hepatitis B core antibody (anti-HBc) positive and who are hepatitis B surface antigen (HBsAg) negative will need to have a negative polymerase chain reaction (PCR) and must be willing to undergo DNA PCR testing during the study to be eligible. Those who are HBsAg positive or hepatitis B PCR positive will be excluded. Subjects who are hepatitis C antibody positive will need to have a negative PCR result to be eligible. Those who are hepatitis C PCR positive will be excluded.
Breast feeding or pregnant
Concurrent participation in another therapeutic trial.
What this trial measures
- Number of participants with Objective Response Rate (ORR)Up to 24 Months
The Objective Response (ORR) is defined as a complete, unconfirmed complete or partial response as determined by the investigator assessment using IPCG criteria