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Acute Graft-versus-host DiseasePhase 1NCT05673876

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)

A Phase 1 study testing GDC-8264 in people with Acute Graft-versus-host Disease, run by Genentech, Inc. at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Graft-versus-host Disease
What is being tested?
GDC-8264
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
7 participants
Where
4 sitesUnited States
Study dates
6 Apr 2023to 15 Jan 2024
Phase
Phase 1
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

4 sites in 1 country.

United States4 sites
  • Massachusetts General Hospital

    Boston, Massachusetts, 02114

  • City of Hope National Medical Center

    Duarte, California, 91010

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Columbia University Irving Medical Center

    New York, New York, 10032

Other trials in United States

About this study

Official title: A Phase Ib, Open-label, Randomized, Dose-finding, Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of GDC-8264 in Combination With Standard of Care in the Treatment of Acute Graft-versus-Host Disease in Patients Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation

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).

Being tested
GDC-8264

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GDC-8264Drug

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

Study groups in NCT05673876
Study groupRoleWhat they receive
GDC-8264, 35 mgExperimentalParticipants will receive oral GDC-8264, 35 milligrams (mg), once daily (QD) for 28 days.
Receives: Drug: GDC-8264
GDC-8264, 75 mgExperimentalParticipants will receive oral GDC-8264, 75 mg, PO, QD for 28 days.
Receives: Drug: GDC-8264

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: From signing informed consent form until 28 days after the final dose of GDC-8264 (up to 9 months)

  2. Plasma Concentration of GDC-8264

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

    Measured: 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

  3. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

  4. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

  5. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

  6. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

  7. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

  8. 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.

    Measured: Day 1 and SS visit (any time between Day 4 to Day 28)

Secondary outcomes (4)
  1. 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.

    Measured: Up to Day 29

  2. 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.

    Measured: From Day 29 up to end of study (up to 9 months)

  3. 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.

    Measured: Baseline up to Day 56

  4. 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.

    Measured: Baseline up to Day 180

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Jan 2023
  2. Study start6 Apr 2023
  3. Primary completion15 Jan 2024
  4. Study completion15 Jan 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
7 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05673876
Sponsor's protocol ID
GA43861
Other IDs
ISRCTN27200385
Status last verified
May 2025
First submitted
21 Dec 2022
Last updated
28 May 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
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

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing GDC-8264 in people with Acute Graft-versus-host Disease, run by Genentech, Inc. at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 24 Apr 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Adverse Events (AEs) (time frame: From signing informed consent form until 28 days after the final dose of GDC-8264 (up to 9 months)). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2025.

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