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CTX-009

Phase 1

Glioblastoma | Small molecule | Oncology |COMPASS Pathways Plc - American Depository Shares|Last Updated: Mar 23, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment54

FDA Designations

No designations recorded

Clinical trial landscape

CTX-009 · 1 trial · 1 indication

Phase 1 1
NCT07392957Safety and Efficacy of CTX-009 With or Without CTX-471 for Recurrent GlioblastomaGlioblastoma
NOT YET_RECRUITING54 Analytics
PHASE1NOT YET_RECRUITING
Safety and Efficacy of CTX-009 With or Without CTX-471 for Recurrent Glioblastoma
GlioblastomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Phase IB Arm 1: Toxicity as measured by number of participants with adverse events
Start of treatment through 60 days after treatment (estimated to be 14 months)

Adverse events will be graded according to CTCAE v5.0

Phase IB Arm 1: Recommended Phase 2 Dose (RP2D)
Start of treatment through completion of cycle 1 (each cycle is 28 days)

RP2D will be determined from the phase IB portion of Arm 1 by assessing tolerability. Tolerability is defined as ≤1 among patients experiencing excessive dose limiting toxicities (DLTs). The dose level in phase IB determined to be tolerable is the RP2D.

Phase IB Arm 2: Toxicity as measured by number of participants with adverse events
Start of treatment through 60 days after treatment (estimated to be 14 months)

Adverse events will be graded according to CTCAE v5.0

Phase II Arm 1: Overall survival rate at 12 months (OS12)
12 months

Overall survival is defined from time of treatment start to time of death due to any cause or latest follow-up, whichever is earlier, with an inference focus on 12-month overall survival.

Phase II Arm 2: Overall survival rate at 12 months (OS12)
12 months

Overall survival is defined from time of treatment start to time of death due to any cause or latest follow-up, whichever is earlier, with an inference focus on 12-month overall survival.

Secondary Endpoints

Overall response rate (ORR)
Start of treatment to end of treatment (estimated total time to be 12 months)
Duration of response (DoR)
Start of treatment to disease progression/recurrence (estimated total time to be 36 months)
Median progression-free survival (mPFS)
Start of treatment to disease progression (estimated to be 36 months)
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase IB Arm 1: CTX-009 monotherapyEXPERIMENTALCTX-009 will be given intravenously at the assigned dose level on an outpatient basis every 2 weeks of a 28-day cycle.
Phase IB Arm 2: CTX-009 and CTX-471 combination therapyEXPERIMENTALCTX-009 will be given intravenously at the dose determined to be the recommended phase II dose (RP2D) in Arm 1 every 2 weeks, and CTX-471 will be given intravenously at a dose of 0.3 mg/kg every 2 weeks (day 1 and day 15). On days when both drugs are given, CTX-009 will be given first, followed by a 30-minute observation period, followed by CTX-471. Cycles will be 28 days.
Phase II Expansion Arm 1: CTX-009 monotherapyEXPERIMENTALCTX-009 will be given intravenously at the recommended phase 2 dose (RP2D) determined from Phase IB on an outpatient basis every 2 weeks of a 28-day cycle.
Phase II Expansion Arm 2: CTX-009 and CTX-471 combination therapyEXPERIMENTALCTX-009 will be given intravenously at the dose determined to be the recommended phase II dose (RP2D) every 2 weeks (day 1 and day 15). CTX-471 will be given intravenously at a dose of 0.3 mg/kg every 2 weeks (day 1 and day 15). On days when both drugs are given, CTX-009 will be given first, followed by a 30-minute observation period, followed by CTX-471. Cycles will be 28 days.

Interventions

NameTypeDescription
CTX-009DRUGCTX-009 will be given intravenously over the course of 60 minutes (+/- 5 minutes) on an outpatient basis every 2 weeks of a 28-day cycle.
CTX-471DRUGCTX-471 will be given intravenously over the course of 30 minutes (-5/+10) on an outpatient basis every 2 weeks of a 28-day cycle.
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Eligibility Criteria

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

Inclusion Criteria: * Histologically or radiographically confirmed recurrent CNS WHO grade 4 IDH wild-type glioma following standard of care treatment including radiation, chemotherapy, and/or tumor-treating fields. No more than 2 recurrences are allowed. * At least 18 years of age. * KPS performan...

Countries:United States
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Frequently asked questions about CTX-009

What is CTX-009 used for in glioblastoma?

CTX-009 is an investigational small molecule being studied for the treatment of glioblastoma, a type of brain cancer. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being evaluated in patients with recurrent glioblastoma.

What does CTX-009 target?

CTX-009 is a small molecule, but its specific molecular target has not been disclosed. The drug is being studied in a biomarker-selected patient population, suggesting that its mechanism of action may be linked to a particular biomarker. However, the exact target is not publicly available.

Who is developing CTX-009?

CTX-009 is being developed by COMPASS Pathways Plc, a biopharmaceutical company listed on the NASDAQ under the ticker symbol CMPS. The company is conducting clinical trials to evaluate the safety and efficacy of CTX-009 in patients with recurrent glioblastoma.

What phase is CTX-009 in?

CTX-009 is currently in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 trial is designed to assess the safety and efficacy of CTX-009, with or without another investigational drug, CTX-471, in patients with recurrent glioblastoma.

What clinical trials is CTX-009 in?

CTX-009 is being studied in a Phase 1 clinical trial with the identifier NCT07392957. The trial, titled 'Safety and Efficacy of CTX-009 With or Without CTX-471 for Recurrent Glioblastoma,' is not yet recruiting and plans to enroll 54 participants in the United States. The study is controlled and randomized.

Is CTX-009 the same as CTX-471?

No, CTX-009 and CTX-471 are different investigational drugs. The Phase 1 clinical trial NCT07392957 is evaluating CTX-009 both alone and in combination with CTX-471 for the treatment of recurrent glioblastoma. CTX-009 is the primary drug being studied, while CTX-471 is an additional agent being tested in combination.