Actively Recruiting
A Phase 2 Study of Paxalisib (GDC-0084) in Recurrent or Refractory Primary Central Nervous System Lymphoma (PCNSL)
Led by Lakshmi Nayak, MD · Updated on 2025-06-26
25
Participants Needed
2
Research Sites
52 weeks
Total Duration
On this page
Sponsors
L
Lakshmi Nayak, MD
Lead Sponsor
K
Kazia Therapeutics Limited
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating a drug called Paxalisib (GDC-0084) as a possible treatment for patients with recurrent or refractory primary central nervous system lymphoma (PCNSL). This open-label Phase 2 study aims to determine how well Paxalisib works and assess its safety in about 25 adult participants with this condition. The FDA has approved Paxalisib for other uses but not specifically for PCNSL. Participants will receive Paxalisib orally each day in cycles lasting 28 days. Treatment can continue for up to 24 months as long as there are no serious side effects or disease progression. The study includes screening for eligibility, treatment, regular evaluations, and follow-up visits throughout the study period. During the study, participants will undergo various assessments including MRI scans and laboratory tests to monitor disease status and side effects. Researchers will track the number of participants who respond to treatment, how long the response lasts, overall survival, progression-free survival, and any adverse events. The total duration of participation can be up to 24 months, with ongoing safety monitoring and follow-up visits.
CONDITIONS
Brief Title
Paxalisib (GDC-0084) In Recurrent Or Refractory PCNSL
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to understand and willing to sign informed consent
- Signed IRB/IEC approved written informed consent form before any study procedures
- Willing and able to comply with visits, treatment, and study requirements
- At least 18 years old at signing informed consent
- Karnofsky Performance Status of 70 or higher
- Histologically confirmed recurrent or refractory primary DLBCL CNS lymphoma
- Evidence of recurrent or refractory disease on MRI with measurable or evaluable enhancing disease
- Recovered to grade 1 or baseline from prior therapy toxic effects; grade 2 neuropathy allowed
- Dexamethasone dose of 8 mg/day or less, stable or decreasing dose for 2 weeks before screening
- Able to undergo MRI
- Adequate blood counts, kidney and liver function as defined in the protocol
- Negative pregnancy test for women of childbearing potential within 72 hours before registration
- Use of highly effective contraception during treatment and for 28 days after last dose if applicable
You will not qualify if you...
- Unable to undergo brain MRI
- Active systemic disease
- Uncontrolled intercurrent illness
- Prior exposure to mTOR or PI3K inhibitors
- Prior malignancy requiring active treatment, except certain treated cancers with disease free for 3 or more years
- Recent systemic anti-cancer therapy or radiotherapy within 4 weeks or 5 half-lives
- Difficulty swallowing oral medication or significant gastrointestinal disease affecting absorption
- Known HIV infection, prior PML, or active significant infections
- Known hypersensitivity to Paxalisib or its components
- Need for treatment with strong CYP3A4 inhibitors or inducers
- Uncontrolled medical comorbidities including severe lung, bowel diseases, or grade 2 or higher diarrhea
- Uncontrolled diabetes or hypertension despite treatment
- Positive hepatitis B or C PCR testing
- Pregnant or breastfeeding
- Concurrent participation in another therapeutic trial
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 months
Participants receive Paxalisib orally each day in 28-day treatment cycles. Treatment continues for up to 24 months as long as there are no serious side effects or disease progression.
Monthly visits for treatment cycles
Duration - Duration varies after treatment ends
Participants are monitored for safety and disease status after completing treatment.
Visits scheduled as needed for assessments
Trial Site Locations
Total: 2 locations
1
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
2
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Actively Recruiting
Research Team
L
Lakshmi Nayak, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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