A Phase 1 interventional study of GDC-8264 in Acute Graft-versus-host Disease, sponsored by Genentech, Inc.. Completed at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-05-28.
Sponsored by Genentech, Inc. · Phase 1, Interventional, and Treatment
The primary purpose of the study is to assess the safety and pharmacokinetics (PK) of GDC-8264 in participants with acute graft-versus-host disease (aGVHD).
806 studies on the registry are indexed under Graft vs Host Disease; 138 are open to participants now.
This study's enrollment of 7 is below the median of 35 across 637 interventional studies indexed under Graft vs Host Disease.
Browse Graft vs Host Disease studies →Genentech, Inc. is the lead sponsor of 507 studies on the registry; 23 are open to participants now.
Of its 90 completed or terminated interventional studies of FDA-regulated products, 50 (56%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Participants will receive oral GDC-8264, 35 milligrams (mg), once daily (QD) for 28 days.
Drug: GDC-8264
Participants will receive oral GDC-8264, 75 mg, PO, QD for 28 days.
Drug: GDC-8264
GDC-8264 tablets will be administered as per the schedule specified in the respective arms.
Number of Participants With Adverse Events (AEs)
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.
Time frame: From signing informed consent form until 28 days after the final dose of GDC-8264 (up to 9 months)
Plasma Concentration of GDC-8264
Plasma concentration of GDC-8264 at specified timepoints was determined.
Time frame: 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
Maximum Plasma Concentration (Cmax) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Time to Reach Maximum Plasma Concentration (Tmax) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Area Under the Plasma Concentration-time Curve From 0 to 24 Hours (AUC0-24) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Terminal Half-life (T1/2) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Apparent Clearance (CL/F) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Apparent Volume of Distribution (Vz/F) of GDC-8264
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 frame: Day 1 and SS visit (any time between Day 4 to Day 28)
Overall Response Rate (ORR) on Day 29
ORR=percentage of participants with complete response (CR), very good partial response (VGPR), or partial response (PR) as determined by investigator. CR=all target organs evaluated as Mount Sinai Acute GVHD International Consortium (MAGIC) aGVHD Stage 0 \[Skin- no GVHD rash; Liver - bilirubin \<2 milligrams/decilitres (mg/dL); Upper gastrointestinal (GI) tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: \< 500 mL/day or \<3 episodes/day (adults), \<10 mL/kilograms (kg)/day or \<4 episodes/day\]. VGPR=resolution of signs \& symptoms i.e. no rash/residual erythematous rash involving \< 25% of body surface, without (w/o) bullae; bilirubin \<2 mg/dL or \<25% of baseline at screening; toleration of food/enteral feeding; predominantly formed stools; no overt GI bleeding/abdominal cramping; no more than occasional nausea/vomiting. PR=improvement in one/more target organs (skin, liver, upper GI tract, lower GI tract) involved with aGVHD symptoms w/o worsening in others.
Time frame: Up to Day 29
Duration of Response (DOR)
DOR was defined astime from response (CR, VGPR or PR) on Day 29 to aGVHD progression from nadir in any organ, new systemic therapy for aGVHD, or death from any cause (whichever occurs first), as determined by the investigator. CR=all target organs evaluated as MAGIC aGVHD Stage 0 \[Skin- no GVHD rash; Liver - bilirubin \<2 mg/dL; Upper GI tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: \< 500 mL/day or \<3 episodes/day (adults), \<10 mL/kg/day or \<4 episodes/day\]. VGPR=resolution of signs \& symptoms i.e. no rash or residual erythematous rash involving \< 25% of body surface, without bullae; bilirubin \<2 mg/dL or \<25% of baseline at screening; toleration of food/enteral feeding; predominantly formed stools; no overt GI bleeding or abdominal cramping; no more than occasional nausea or vomiting. PR=improvement in one or more target organs (skin, liver, upper GI tract, lower GI tract) involved with aGVHD symptoms without worsening in others.
Time frame: From Day 29 up to end of study (up to 9 months)
Percentage of Participants With aGVHD Flares by Day 56
aGVHD flare was defined as an increase in aGVHD target organ stage by at least one stage for at least 3 days that requires either addition of a new line of systemic treatment or increase in corticosteroid dose \> 0.25 mg/kg/day.
Time frame: Baseline up to Day 56
Percentage of Participants With Non-relapse Mortality (NRM) by Day 180
NRM was defined as any death that occurred after onset of aGVHD that was not attributable to relapse of the underlying primary disease was considered a non-relapse death.
Time frame: Baseline up to Day 180
A total of 7 participants took part in the study across 4 investigative sites in the United States from 06 April 2023 to 15 January 2024.
| Milestone | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Started | 4 | 3 |
| Completed | 1 | 0 |
| Not completed | 3 | 3 |
| Withdrew: Death | 3 | 1 |
| Withdrew: Study terminated by sponsor | 0 | 1 |
| Withdrew: Physician decision | 0 | 1 |
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.
| Participants | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | 4 | 3 |
Plasma concentration of GDC-8264 at specified timepoints was determined.
| nanograms/millilitres (ng/mL) | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Predose on Day 1 | NA ± NA | NA ± NA |
| 1.5 hours Postdose on Day 1 | 28.3 ± 141.5 | 698 ± 17.0 |
| 2 hours Postdose on Day 1 | 151 ± NA | 835 ± 10.3 |
| 3 hours Postdose on Day 1 | 89.7 ± 321.6 | 858 ± 57.2 |
| 4 hours Postdose on Day 1 | 123 ± 141.1 | 781 ± 33.8 |
| 6 hours Postdose on Day 1 | 227 ± NA | 700 ± 52.5 |
| 12 hours Postdose on Day 1 | 24.0 ± 28439.7 | 361 ± 106.5 |
| 24 hours Postdose on Day 1 | 12.2 ± 5241.5 | 144 ± 116.0 |
| Predose on Day 4 | 58.9 ± 167.3 | 167 ± 78.1 |
| 1.5 hours Postdose on Day 4 | 400 ± 27.2 | 195 ± 158.2 |
| 2 hours Postdose on Day 4 | 529 ± 31.8 | 218 ± 181.5 |
| 3 hours Postdose on Day 4 | 433 ± 19.2 | 318 ± 223.3 |
| 4 hours Postdose on Day 4 | 374 ± 8.8 | 393 ± 175.7 |
| 6 hours Postdose on Day 4 | 274 ± 6.7 | 539 ± 127.1 |
| 12 hours Postdose on Day 4 | 129 ± 43.1 | 373 ± 82.6 |
| 24 hours Postdose on Day 4 | 43.2 ± 143.2 | 140 ± 79.9 |
| Predose on Day 8 | 39.5 ± 138.0 | 266 ± 70.1 |
| Predose on Day 15 | 6.47 ± NA | 294 ± 54.6 |
| Predose on Day 22 | 6.50 ± NA | 276 ± 180.1 |
| Predose on Day 29 | 61.5 ± 297.2 | 1.51 ± NA |
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.
| ng/mL | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 270.00 ± NA | 991.21 ± 33.7 |
| SS PK Day | 541.48 ± 31.1 | 1480.00 ± NA |
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.
| hours | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 3.4 (3.4 to 3.4) | 2.30 (1.6 to 3.0) |
| SS PK Day | 2.17 (1.5 to 3.1) | 3.2 (3.2 to 3.2) |
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.
| nanograms*hours/millilitres (ng*hr/mL) | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 3250.37 ± NA | 10528.86 ± 57.8 |
| SS PK Day | 4534.44 ± 24.4 | 19405.83 ± NA |
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.
| hours | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 7.03 ± NA | 7.82 ± 27.2 |
| SS PK Day | 6.84 ± 50.2 | 8.70 ± NA |
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.
| millilitres/hours (mL/hr) | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 9608.61 ± NA | 6131.74 ± 69.5 |
| SS PK Day | 6871.73 ± 32.5 | 3216.19 ± NA |
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.
| millilitres (mL) | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Day 1 | 97480.88 ± NA | 69212.58 ± 37.3 |
| SS PK Day | 67784.28 ± 21.0 | 40355.80 ± NA |
ORR=percentage of participants with complete response (CR), very good partial response (VGPR), or partial response (PR) as determined by investigator. CR=all target organs evaluated as Mount Sinai Acute GVHD International Consortium (MAGIC) aGVHD Stage 0 \[Skin- no GVHD rash; Liver - bilirubin \<2 milligrams/decilitres (mg/dL); Upper gastrointestinal (GI) tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: \< 500 mL/day or \<3 episodes/day (adults), \<10 mL/kilograms (kg)/day or \<4 episodes/day\]. VGPR=resolution of signs \& symptoms i.e. no rash/residual erythematous rash involving \< 25% of body surface, without (w/o) bullae; bilirubin \<2 mg/dL or \<25% of baseline at screening; toleration of food/enteral feeding; predominantly formed stools; no overt GI bleeding/abdominal cramping; no more than occasional nausea/vomiting. PR=improvement in one/more target organs (skin, liver, upper GI tract, lower GI tract) involved with aGVHD symptoms w/o worsening in others.
| percentage of participants | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Overall Response Rate (ORR) on Day 29 | 100 | 66.7 |
DOR was defined astime from response (CR, VGPR or PR) on Day 29 to aGVHD progression from nadir in any organ, new systemic therapy for aGVHD, or death from any cause (whichever occurs first), as determined by the investigator. CR=all target organs evaluated as MAGIC aGVHD Stage 0 \[Skin- no GVHD rash; Liver - bilirubin \<2 mg/dL; Upper GI tract - no intermittent nausea, vomiting/anorexia; Lower GI tract - stool output: \< 500 mL/day or \<3 episodes/day (adults), \<10 mL/kg/day or \<4 episodes/day\]. VGPR=resolution of signs \& symptoms i.e. no rash or residual erythematous rash involving \< 25% of body surface, without bullae; bilirubin \<2 mg/dL or \<25% of baseline at screening; toleration of food/enteral feeding; predominantly formed stools; no overt GI bleeding or abdominal cramping; no more than occasional nausea or vomiting. PR=improvement in one or more target organs (skin, liver, upper GI tract, lower GI tract) involved with aGVHD symptoms without worsening in others.
| days | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Duration of Response (DOR) | 1.00 (1.0 to 1.0) | 4.00 (4.0 to 4.0) |
aGVHD flare was defined as an increase in aGVHD target organ stage by at least one stage for at least 3 days that requires either addition of a new line of systemic treatment or increase in corticosteroid dose \> 0.25 mg/kg/day.
| percentage of participants | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Percentage of Participants With aGVHD Flares by Day 56 | 50 | 0 |
NRM was defined as any death that occurred after onset of aGVHD that was not attributable to relapse of the underlying primary disease was considered a non-relapse death.
| percentage of participants | GDC-8264 35 mg | GDC-8264 75 mg |
|---|---|---|
| Percentage of Participants With Non-relapse Mortality (NRM) by Day 180 | 0 | 66.7 |
Collected over From signing informed consent form until 28 days after the final dose of GDC-8264 (up to 9 months). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| GDC-8264 35mg | 3/4 (75%) | 4/4 (100%) | 4/4 (100%) |
| GDC-8264 75mg | 2/3 (66.7%) | 3/3 (100%) | 3/3 (100%) |
| Event | GDC-8264 35mg | GDC-8264 75mg |
|---|---|---|
| Multiple organ dysfunction syndromeGeneral disorders | 0/4 | 1/3 |
| PyrexiaGeneral disorders | 0/4 | 1/3 |
| PneumoniaInfections and infestations | 0/4 | 1/3 |
| SepsisInfections and infestations | 1/4 | 1/3 |
| Upper respiratory tract infectionInfections and infestations | 0/4 | 1/3 |
| ViraemiaInfections and infestations | 0/4 | 1/3 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 0/4 | 1/3 |
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 0/4 | 1/3 |
| Abdominal painGastrointestinal disorders | 1/4 | 0/3 |
| DeathGeneral disorders | 1/4 | 0/3 |
| Event | GDC-8264 35mg | GDC-8264 75mg |
|---|---|---|
| FatigueGeneral disorders | 1/4 | 3/3 |
| Blood creatinine increasedInvestigations | 3/4 | 2/3 |
| HypotensionVascular disorders | 3/4 | 1/3 |
| Abdominal distensionGastrointestinal disorders | 0/4 | 2/3 |
| Anal incontinenceGastrointestinal disorders | 0/4 | 2/3 |
| Upper respiratory tract infectionInfections and infestations | 0/4 | 2/3 |
| Decreased appetiteMetabolism and nutrition disorders | 0/4 | 2/3 |
| HyperphosphataemiaMetabolism and nutrition disorders | 1/4 | 2/3 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 0/4 | 2/3 |
| Confusional statePsychiatric disorders | 1/4 | 2/3 |
Safety population included participants who received at least one dose of GDC-8264, with participants grouped according to treatment received.
| Age, Continuous(years) | GDC-8264 35 mg | GDC-8264 75 mg | Total |
|---|---|---|---|
| Mean | 61.3 ± 12.7 | 57.3 ± 6.8 | 59.6 ± 10.0 |
| Sex: Female, Male(Participants) | GDC-8264 35 mg | GDC-8264 75 mg | Total |
|---|---|---|---|
| Female | 2 | 2 | 4 |
| Male | 2 | 1 | 3 |
| Ethnicity (NIH/OMB)(Participants) | GDC-8264 35 mg | GDC-8264 75 mg | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 2 | 3 |
| Not Hispanic or Latino | 2 | 1 | 3 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | GDC-8264 35 mg | GDC-8264 75 mg | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 0 | 1 |
| White | 2 | 3 | 5 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 1 | 0 | 1 |
Documents are hosted by the registry — open the source record to download them.
Plan to share: No — For eligible studies, qualified researchers may request access to individual patient level clinical data. See Roche's commitment to transparency of clinical study information here: https://go.roche.com/data_sharing
This study is completed, as verified in May 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Genentech, Inc.