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RO7020531

Phase 1

Healthy Participants | Small molecule | Other |Roche Holding AG|Last Updated: Feb 8, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment70

FDA Designations

No designations recorded

Clinical trial landscape

RO7020531 · 2 trials · 2 indications

Phase 1 2
NCT03530917A Study to Assess the Safety and Tolerability of Single and Multiple Ascending Doses of Oral RO7020531 in Chinese Healthy Participants.Healthy Participants
COMPLETED70 Analytics
NCT02956850A Study in Healthy Volunteers and in Participants With Chronic Hepatitis B to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of RO7020531Hepatitis B, Chronic
COMPLETED160 Analytics
PHASE1COMPLETED
A Study to Assess the Safety and Tolerability of Single and Multiple Ascending Doses of Oral RO7020531 in Chinese Healthy Participants.
Healthy ParticipantsUnlock trial analytics
PHASE1COMPLETED
A Study in Healthy Volunteers and in Participants With Chronic Hepatitis B to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of RO7020531
Hepatitis B, ChronicUnlock trial analytics

Study Endpoints

Primary Endpoints

Percentage of Participants With Adverse Events (AEs)
Screening up until 28 days after the last dose of study drug (up to 1 year).

An Adverse Event (AE) is defined as 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, any new disease or exacerbation of an existing disease, recurrence of an intermittent medical condition, any deterioration in a laboratory value or other clinical test or adverse events that are related to a protocol-mandated intervention, including those that occur prior to assignment of study treatment.

Part 1: Percentage of SAD Participants With Adverse Events (AEs)
From randomization up to Day 29

An AE is any untoward medical occurrence in a clinical investigation healthy volunteer (HV)/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, any new disease or exacerbation of an existing disease (a worsening in the character, frequency, or severity of a known condition and recurrence of an intermittent medical condition (e.g., headache) not present at baseline.

Part 1: Percentage of MAD Participants With AEs
From randomization up to Day 41

An AE is any untoward medical occurrence in a clinical investigation healthy volunteer (HV)/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, any new disease or exacerbation of an existing disease (a worsening in the character, frequency, or severity of a known condition and recurrence of an intermittent medical condition (e.g., headache) not present at baseline.

Part 2: Percentage of Chronic Hepatitis B Participants With AEs
From randomization up to Week 12

An AE is any untoward medical occurrence in a 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, any new disease or exacerbation of an existing disease (a worsening in the character, frequency, or severity of a known condition and recurrence of an intermittent medical condition (e.g., headache) not present at baseline.

Part 1: Percentage of SAD Participants With Laboratory Abnormalities Based on Hematology, Clinical Chemistry, Coagulation and Urinalysis Test Results
From randomization up to Day 8

For all laboratory parameters included, there exists a Roche predefined standard reference range. Laboratory values falling outside this standard reference range were labeled "H" for high or "L" for low in HV/participant listings of laboratory data.

Part 1: Percentage of MAD Participants With Laboratory Abnormalities Based on Hematology, Clinical Chemistry, Coagulation and Urinalysis Test Results
From randomization up to Day 20

For all laboratory parameters included, there exists a Roche predefined standard reference range. Laboratory values falling outside this standard reference range were labeled "H" for high or "L" for low in HV/participant listings of laboratory data.

Part 2: Percentage of Chronic Hepatitis B Participants With Laboratory Abnormalities Based on Hematology, Clinical Chemistry, Coagulation and Urinalysis Test Results
From randomization up to Week 12

For all laboratory parameters included, there exists a Roche predefined standard reference range. Laboratory values falling outside this standard reference range were labeled "H" for high or "L" for low in HV/participant listings of laboratory data.

Part 1: Percentage of SAD Participants With Abnormalities in Electrocardiograph (ECG) Parameters
From randomization up to Day 8

Triplicate 12-lead ECGs were obtained after the participant has been in a supine position for at least 10 minutes. Clinically significant RO7020531-related changes included confirmation of mean QTc 500 milliseconds (msec) or 60 msec longer than the pre-dose baseline.

Part 1: Percentage of MAD Participants With Abnormalities in ECG Parameters
From randomization up to Day 20

Triplicate 12-lead ECGs were obtained after the participants has been in a supine position for at least 10 minutes. Clinically significant RO7020531-related changes included confirmation of mean QTc 500 msec or 60 msec longer than the pre-dose baseline.

Part 2: Percentage of Chronic Hepatitis B Participants With Abnormalities in ECG Parameters
From randomization up to Week 12

Triplicate 12-lead ECGs were obtained after the participant has been in a supine position for at least 10 minutes. Clinically significant RO7020531-related changes included confirmation of mean QTc 500 msec or 60 msec longer than the pre-dose baseline.

Part 1: Percentage of SAD Participants With Abnormalities in Vital Signs
From randomization up to Day 8

Vital signs include blood pressure, pulse rate, respiratory rate and body temperature. Blood pressure, respiratory rate and pulse rate were obtained after the participant had been in a supine or sitting position for at least 5 minutes. Blood pressure measurement were performed in triplicate (can be as short as 20 second to 1 minute interval between measurements).

Part 1: Percentage of MAD Participants With Abnormalities in Vital Signs
From randomization up to Day 20

Vital signs include blood pressure, pulse rate, respiratory rate and body temperature. Blood pressure, respiratory rate and pulse rate were obtained after the participant had been in a supine or sitting position for at least 5 minutes. Blood pressure measurement were performed in triplicate (can be as short as 20 second to 1 minute interval between measurements).

Part 2: Percentage of Chronic Hepatitis B Participants With Abnormalities in Vital Signs
From randomization up to Week 12

Vital signs include blood pressure, pulse rate, respiratory rate and body temperature. Blood pressure, respiratory rate and pulse rate were obtained after the participant had been in a supine or sitting position for at least 5 minutes. Blood pressure measurement were performed in triplicate (can be as short as 20 second to 1 minute interval between measurements).

Secondary Endpoints

Maximum Observed Plasma Concentration (Cmax) for RO7020531, Main Active Metabolite (RO7011785) and Prodrug Metabolites (RO7018822 and RO7033805)
SAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24, 36, 48 hours (h) Post-dose Days 1, 2; MAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24h Post-dose Days 1, 2 and Days 13, 14; Pre-dose, 2, 6, 24hr Post-dose Days 3, 5, 7, 9 and 11.
Area Under the Plasma Concentration Versus Time Curve up to the Last Measurable Concentration (AUClast) for RO7020531, Main Active Metabolite (RO7011785) and Prodrug Metabolites (RO7018822 and RO7033805)
SAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24, 36, 48 hours (h) Post-dose Days 1, 2; MAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24h Post-dose Days 1, 2 and Days 13, 14; Pre-dose, 2, 6, 24hr Post-dose Days 3, 5, 7, 9 and 11.
Area Under the Plasma Concentration Versus Time Curve Extrapolated to Infinity (AUCinf) for RO7020531, Main Active Metabolite (RO7011785) and Prodrug Metabolites (RO7018822 and RO7033805)
SAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24, 36, 48 hours (h) Post-dose Days 1, 2; MAD: Pre-dose, 0.25, 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12, 18, 24h Post-dose Days 1, 2 and Days 13, 14; Pre-dose, 2, 6, 24hr Post-dose Days 3, 5, 7, 9 and 11.
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Single Ascending Dose (SAD): PlaceboEXPERIMENTALIn SAD Cohorts 1-4, there will be eight participants in total receiving placebo, two in each cohort.
SAD: Cohort 1EXPERIMENTALEight participants will be administered 40mg RO7020531 orally on Day 1.
SAD: Cohort 2EXPERIMENTALEight participants will be administered 100mg RO7020531 orally on Day 1.
SAD: Cohort 3EXPERIMENTALEight participants will be administered 140mg RO7020531 orally on Day 1.
SAD: Cohort 4EXPERIMENTALEight participants will be administered 170mg RO7020531 orally on Day 1.
Multiple Ascending Dose (MAD): PlaceboEXPERIMENTALIn MAD Cohorts 1-3, there will be six participants in total receiving placebo, two in each cohort.
MAD: Cohort 1EXPERIMENTALEight participants will be administered 100mg RO7020531 orally on Day 1 and every other day (QOD) for 14 days.
MAD: Cohorts 2 and 3EXPERIMENTALSixteen participants will be administered 150mg RO7020531 orally on Day 1 and every other day (QOD) for 14 days.
Part I: SAD in Healthy VolunteersEXPERIMENTALHealthy volunteers will receive single dose of RO7020531 or matching placebo orally on Day 1 of each cohort. A planned dose-escalation sequence for SAD is 3 milligrams (mg), 10 mg, 20 mg, 40 mg, 60 mg, 100 mg, 140 mg, and 170 mg.
Part I: MAD in Healthy VolunteersEXPERIMENTALHealthy volunteers will receive RO7020531 (100 mg, 140 mg, and 170 mg as selected based on safety, pharmacokinetic (PK) and pharmacodynamic (PD) data of SAD cohorts) or matching placebo orally every other day (QOD) from Day 1 through to Day 13.
Part II: CHB ParticipantsEXPERIMENTALCHB participants will receive RO7020531 (150 mg and 170 mg as selected based on safety, PK and PD data of MAD cohorts) or matching placebo orally QOD from Day 1 through to Day 41, unless in Cohort 4 in case of once a week (QW) dosing as dose modification.

Interventions

NameTypeDescription
RO7020531DRUG4 SAD Cohorts with individual dosages of 40, 100, 140 and 170 mg hard capsules and 3 MAD Cohorts with dosages of 100 and 150mg hard capsules, will be administered orally as per the dosing schedules described above.
PlaceboDRUGPlacebo hard capsules will be administered orally as per the dosing schedules described above.
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Eligibility Criteria

Age Range18 Years to 60 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria * Chinese healthy male and female participants. Healthy status is defined by absence of evidence of any active or chronic disease following a detailed medical and surgical history, a complete physical examination including vital signs, 12-lead ECG, hematology, blood chemistry, an...

Countries:Hong KongBulgariaItalyNetherlandsNew ZealandTaiwanThailandUnited Kingdom
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Frequently asked questions about RO7020531

What is RO7020531 used for?

RO7020531 is an investigational small molecule being studied for chronic Hepatitis B. It is being evaluated in healthy participants and in participants with chronic Hepatitis B to assess safety, tolerability, pharmacokinetics, and pharmacodynamics after single and multiple doses.

Who makes RO7020531?

RO7020531 is being developed by Roche Holding AG, traded on the OTC market under the ticker RHHBY. The company is conducting clinical trials to evaluate the drug's safety and tolerability in healthy participants and in those with chronic Hepatitis B.

What phase is RO7020531 in?

RO7020531 is in Phase 1 clinical development. It is investigational and has not been approved by regulatory authorities. Two Phase 1 trials have been completed, one involving healthy volunteers and participants with chronic Hepatitis B, and another in Chinese healthy participants.

What clinical trials is RO7020531 in?

RO7020531 has been studied in two completed Phase 1 trials. NCT02956850 assessed safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers and participants with chronic Hepatitis B across multiple countries. NCT03530917 evaluated single and multiple ascending doses in Chinese healthy participants.

Is RO7020531 the same as any other drug?

No alternative names for RO7020531 have been disclosed in the clinical trial records. The drug is identified solely by its code name RO7020531 in the studies conducted by Roche.