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RO7126209

Phase 1

Alzheimers Disease | Small molecule | Neurology |Roche Holding AG|Last Updated: Jun 16, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials2
Total Enrollment277

FDA Designations

No designations recorded

Clinical trial landscape

RO7126209 · 2 trials · 1 indication

Phase 1 2
NCT04639050Brainshuttle AD: A Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7126209 Following Intravenous Infusion in Participants With Prodromal or Mild to Moderate Alzheimer's DiseaseAlzheimers Disease
ACTIVE NOT_RECRUITING241 Analytics
NCT04023994A Single Ascending Dose Study to Investigate the Safety, Tolerability, Immunogenicity and Pharmacokinetics of Intravenously Administered RO7126209 in Healthy ParticipantsAlzheimers Disease
COMPLETED36 Analytics
PHASE1ACTIVE NOT_RECRUITING
Brainshuttle AD: A Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7126209 Following Intravenous Infusion in Participants With Prodromal or Mild to Moderate Alzheimer's Disease
Alzheimers DiseaseUnlock trial analytics
PHASE1COMPLETED
A Single Ascending Dose Study to Investigate the Safety, Tolerability, Immunogenicity and Pharmacokinetics of Intravenously Administered RO7126209 in Healthy Participants
Alzheimers DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Part 1, 2, 3, and 4: Percentage of Participants With Adverse Events (AEs)
Part 1 and 2: Up to approximately 56 weeks; Part 3: Up to approximately 52 weeks; Part 4: Up to approximately 233 weeks
Part 3: Change From Baseline in Brain Amyloid Load as Measured by Amyloid Positron Emission Tomography (PET) Scan
Up to approximately 24 weeks
Percentage of Participants With Adverse Events (AEs)
Up to approximately 9 weeks

An Adverse Event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An Adverse Event can therefore be any unfavorable and unintended sign (including abnormal laboratory values or abnormal clinical test results), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as Adverse Events.

Secondary Endpoints

Part 1, 2, and 4: Change From Baseline in Brain Amyloid Load as Measured by Amyloid PET Scan
Part 1 and 2: Up to approximately 28 weeks; Part 4: Up to approximately 205 weeks
Part 1, 2, 3, and 4: Plasma Concentration of RO7126209
Part 1 and 2: Up to approximately 32 weeks; Part 3: Up to approximately 24 weeks; Part 4: Up to approximately 209 weeks
Part 1, 2, 3, and 4: Cerebral Spinal Fluid (CSF) Concentration of RO7126209
Part 1 and 2: Up to approximately 25 weeks; Part 3: Up to approximately 21 weeks; Part 4: Up to approximately 205 weeks
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1 (Dose Finding) Cohort 1: Dose Level 1 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 1 once every 4 weeks (Q4W) for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 1: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 1 Q4W for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 2: Dose Level 2 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 2, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 2: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 2, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 3: Dose Level 3 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 3, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 3: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 3, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 4: Dose Level 4 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 4, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 4: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 4, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 1 (Dose Finding) Cohort 5: Dose Level 5 of RO7126209EXPERIMENTALParticipants will receive a total of 2 doses of RO7126209 at dose level 5 Q4W, for 28 weeks followed by an 8-week safety follow-up period.
Part 1 (Dose Finding) Cohort 5: PlaceboPLACEBO_COMPARATORParticipants will receive a total of 2 doses of matching placebo to dose level 5 Q4W, for 28 weeks followed by an 8-week safety follow-up period.
Part 2 (Expansion) Cohort 1: Dose Level 3 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 3, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 2 (Expansion) Cohort 1: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 3, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 2 (Expansion) Cohort 2: Dose Level 4 of RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 at dose level 4, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 2 (Expansion) Cohort 2: PlaceboPLACEBO_COMPARATORParticipants will receive matching placebo to dose level 4, Q4W, for 28 weeks followed by a 28-week safety follow-up period.
Part 2 (Expansion) Cohort 3: Dose Level 5 of RO7126209EXPERIMENTALParticipants will receive a total of 2 doses of RO7126209 at dose level 5, Q4W, for 28 weeks followed by an 8-week safety follow-up period.
Part 2 (Expansion) Cohort 3: PlaceboPLACEBO_COMPARATORParticipants will receive a total of 2 doses of matching placebo to dose level 5, Q4W, for 28 weeks followed by an 8-week safety follow-up period.
Part 3 (Dose/Frequency/Pharmacodynamic (PD) Relationship): Dose Level 1: RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 in an open-label treatment period at dose level 1, Q4W, for 24 weeks followed by a 28-week safety follow-up period.
Part 3 Dose/Frequency/PD Relationship: Dose Level 2: RO7126209EXPERIMENTALParticipants will receive multiple doses of RO7126209 in an open-label treatment period at dose level 2, Q12W, for 24 weeks followed by a 28-week safety follow-up period.
Part 4: Open Label Extension (OLE)EXPERIMENTALParticipants who completed Part 1, 2, or 3 will receive RO7126209 for a maximum of 205 weeks.
PlaceboPLACEBO_COMPARATORIn Cohorts 1-5, there were ten participants in total who received placebo, two in each cohort.
RO7126209 (0.1 mg/kg)EXPERIMENTALHealthy volunteers will be administered a single intravenous dose of RO7126209 (0.1 mg/kg).
RO7126209 (0.4 mg/kg)EXPERIMENTALHealthy volunteers will be administered a single intravenous dose of RO7126209 (0.4 mg/kg).
RO7126209 (1.2 mg/kg)EXPERIMENTALHealthy volunteers will be administered a single intravenous dose of RO7126209 (1.2 mg/kg).
RO7126209 (3.6 mg/kg)EXPERIMENTALHealthy volunteers will be administered a single intravenous dose of RO7126209 (3.6 mg/kg).
RO7126209 (7.2 mg/kg)EXPERIMENTALHealthy volunteers will be administered a single intravenous dose of RO7126209 (7.2 mg/kg).

Interventions

NameTypeDescription
RO7126209DRUGRO7126209 will be administered intravenously as specified in each treatment arm.
PlaceboDRUGRO7126209-matching placebo will be administered intravenously as specified in each treatment arm.
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Eligibility Criteria

Age Range50 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites41

Key inclusion criteria for part 1, 2 and 3: * Ability to provide written consent signed by the participant * Availability of a person (referred to as the "study partner") who: consents to participate throughout the duration of study, in the Investigator's judgment, has frequent and sufficient conta...

Countries:United StatesAustraliaCanadaChileJapanPolandSouth KoreaSpainUnited Kingdom
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Frequently asked questions about RO7126209

What is RO7126209 used for in Alzheimer's disease?

RO7126209 is an investigational small molecule being developed by Roche Holding AG for the treatment of Alzheimer's disease. It is currently in Phase 1 clinical development, with studies evaluating its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy participants and in patients with prodromal or mild to moderate Alzheimer's disease.

Who makes RO7126209?

RO7126209 is being developed by Roche Holding AG, which trades under the ticker RHHBY. The company is conducting clinical trials to evaluate the drug's safety and efficacy in Alzheimer's disease, with studies taking place in multiple countries including the United States, Australia, Canada, Chile, Japan, Poland, South Korea, Spain, and the United Kingdom.

What phase is RO7126209 in?

RO7126209 is in Phase 1 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The drug is being studied in two Phase 1 trials: one completed single ascending dose study in healthy participants and one active multiple ascending dose study in patients with prodromal or mild to moderate Alzheimer's disease.

What clinical trials is RO7126209 in?

RO7126209 has two clinical trials. NCT04023994 is a completed Phase 1 single ascending dose study in healthy participants in the United States. NCT04639050 is an active Phase 1 multiple ascending dose study, known as Brainshuttle AD, in patients with prodromal or mild to moderate Alzheimer's disease across nine countries.

Is RO7126209 the same as Brainshuttle AD?

RO7126209 is the drug being tested in the Brainshuttle AD study, which is the title of the multiple ascending dose trial (NCT04639050). The study evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of RO7126209 following intravenous infusion in participants with prodromal or mild to moderate Alzheimer's disease.