Phase 1
Completed N=7
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 Disease
Source: ClinicalTrials.gov NCT05673876 ↗
Enrolled (actual)
7
Serious AEs
100.0%
Results posted
Apr 2025
Primary outcomePrimary: Number of Participants With Adverse Events (AEs) — 4; 3 Participants
Summary
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).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs) |
4; 3 | — |
| PRIMARY Plasma Concentration of GDC-8264 |
NA; NA; 28.3; 698; 151; 835 | — |
| PRIMARY Maximum Plasma Concentration (Cmax) of GDC-8264 |
270.00; 991.21; 541.48; 1480.00 | — |
| PRIMARY Time to Reach Maximum Plasma Concentration (Tmax) of GDC-8264 |
3.4; 2.30; 2.17; 3.2 | — |
| PRIMARY Area Under the Plasma Concentration-time Curve From 0 to 24 Hours (AUC0-24) of GDC-8264 |
3250.37; 10528.86; 4534.44; 19405.83 | — |
| PRIMARY Terminal Half-life (T1/2) of GDC-8264 |
7.03; 7.82; 6.84; 8.70 | — |
| PRIMARY Apparent Clearance (CL/F) of GDC-8264 |
9608.61; 6131.74; 6871.73; 3216.19 | — |
| PRIMARY Apparent Volume of Distribution (Vz/F) of GDC-8264 |
97480.88; 69212.58; 67784.28; 40355.80 | — |
| SECONDARY Overall Response Rate (ORR) on Day 29 |
100; 66.7 | — |
| SECONDARY Duration of Response (DOR) |
1.00; 4.00 | — |
| SECONDARY Percentage of Participants With aGVHD Flares by Day 56 |
50; 0 | — |
| SECONDARY Percentage of Participants With Non-relapse Mortality (NRM) by Day 180 |
0; 66.7 | — |
Eligibility Criteria
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 corticosteroids for aGVHD at a dose of prednisone ≥2 milligrams per kilograms per day (mg/kg/day) by orally (PO) or methylprednisolone ≥2 mg/kg/day intravenously (or equivalent) in divided doses at diagnosis and up to 3 days prior to or on the same day as initiation of GDC-8264 (Day 1), with no taper planned prior to Day 3
Exclusion Criteria
- Evidence of relapsed, progressing, or persistent malignancy, or treatment for relapse after transplant, or requirement for rapid immune suppression withdrawal as pre-emergent treatment of early malignancy relapse
- Prior receipt of more than one allogeneic HSCT
- Prior receipt of solid organ transplantation that are target organs for aGVHD (e.g., liver transplant)
- Prior systemic treatment for aGVHD, except for the standard of care corticosteroid treatment initiated as part of this trial
- Diagnosis of chronic GVHD or overlap syndrome
- Uncontrolled active infection (i.e., progressive symptoms related to infection despite treatment, or persistently positive blood cultures despite treatment, or any other evidence of severe sepsis)
- Severe organ dysfunction (e.g., acute liver failure, renal failure requiring dialysis, ventilator support, or vasopressor therapy)
- Initiation or planned use of a marketed small molecule (excluding corticosteroids) or biologic therapy as treatment for aGVHD from the start of screening through the treatment period
Data sourced from ClinicalTrials.gov (NCT05673876). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.