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GDC-8264

Phase 1

Acute Graft-versus-host Disease | Small molecule | Immunology |Roche Holding AG|Last Updated: May 28, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials1
Total Enrollment7

FDA Designations

No designations recorded

Clinical trial landscape

GDC-8264 · 1 trial · 1 indication

Phase 1 1
NCT05673876A Study to Assess the Safety and Pharmacokinetics of GDC-8264 in Combination With Standard of Care in Participants With Acute Graft-Versus-Host Disease (aGVHD)Acute Graft-versus-host Disease
COMPLETED7 Analytics
PHASE1COMPLETED
A Study to Assess the Safety and Pharmacokinetics of GDC-8264 in Combination With Standard of Care in Participants With Acute Graft-Versus-Host Disease (aGVHD)
Acute Graft-versus-host DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants With Adverse Events (AEs)
From signing informed consent form until 28 days after the final dose of GDC-8264 (up to 9 months)

An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product, regardless of causal attribution. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.

Plasma Concentration of GDC-8264
Predose, 1.5, 2, 3, 4, 6, 12, and 24 hours post-dose on Days 1 and 4; Predose on Days 8, 15, 22, 29

Plasma concentration of GDC-8264 at specified timepoints was determined.

Maximum Plasma Concentration (Cmax) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

Steady-state (SS) PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Time to Reach Maximum Plasma Concentration (Tmax) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

SS PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Area Under the Plasma Concentration-time Curve From 0 to 24 Hours (AUC0-24) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

SS PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Terminal Half-life (T1/2) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

SS PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Apparent Clearance (CL/F) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

SS PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Apparent Volume of Distribution (Vz/F) of GDC-8264
Day 1 and SS visit (any time between Day 4 to Day 28)

SS PK visit was Day 4 if the participant was hospitalized or Day 8 if the participant was an outpatient. If the SS PK visit samples could not be obtained on Day 4/8, the SS PK visit may have occurred on any dosing day between Day 4 and 28.

Secondary Endpoints

Overall Response Rate (ORR) on Day 29
Up to Day 29
Duration of Response (DOR)
From Day 29 up to end of study (up to 9 months)
Percentage of Participants With aGVHD Flares by Day 56
Baseline up to Day 56
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
GDC-8264, 35 mgEXPERIMENTALParticipants will receive oral GDC-8264, 35 milligrams (mg), once daily (QD) for 28 days.
GDC-8264, 75 mgEXPERIMENTALParticipants will receive oral GDC-8264, 75 mg, PO, QD for 28 days.

Interventions

NameTypeDescription
GDC-8264DRUGGDC-8264 tablets will be administered as per the schedule specified in the respective arms.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Diagnosis of post-allogeneic hematopoietic stem cell transplantation (HSCT) aGVHD at screening * Evidence of engraftment post-transplant * Diagnosis of high-risk aGVHD, per refined Minnesota high-risk aGVHD criteria during screening * Initiation of treatment with systemic cort...

Countries:United States
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Frequently asked questions about GDC-8264

What is GDC-8264 used for?

GDC-8264 is an investigational small molecule being developed for the treatment of acute Graft-versus-host Disease (aGVHD). It is intended to be used in combination with standard of care therapy in patients with this condition. The drug is currently in Phase 1 clinical development.

Who makes GDC-8264?

GDC-8264 is being developed by Roche Holding AG, which trades under the ticker RHHBY. The company is conducting clinical trials to evaluate the safety and pharmacokinetics of this investigational drug in patients with acute Graft-versus-host Disease.

What phase is GDC-8264 in?

GDC-8264 is in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities. A Phase 1 study has been completed to assess its safety and pharmacokinetics in combination with standard of care for acute Graft-versus-host Disease.

What clinical trials is GDC-8264 in?

GDC-8264 has one completed clinical trial, identified as NCT05673876. This Phase 1 study assessed the safety and pharmacokinetics of GDC-8264 in combination with standard of care in participants with acute Graft-versus-host Disease. The trial enrolled 7 participants in the United States.

Is GDC-8264 the same as any other drug?

GDC-8264 is the primary name for this investigational small molecule. No alternative names have been reported for this drug in the available clinical trial information. It is being studied specifically for acute Graft-versus-host Disease.