Autoimmune HepatitisPhase 2NCT04790916
Effect of RO7049665 on the Time to Relapse Following Steroid Tapering in Participants With Autoimmune Hepatitis (AIH)
A Phase 2 study testing RO7049665 in people with Autoimmune Hepatitis and Autoimmune Chronic Hepatitis, run by Hoffmann-La Roche at 14 sites in 8 countries.
Trial summary
The primary objective of the study is to evaluate the effect of RO7049665 on time to relapse following forced corticosteroid (CCS) tapering as measured by the hazard ratio between RO7049665 7.5 milligrams (mg) and placebo arm. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Autoimmune Hepatitis, Autoimmune Chronic Hepatitis
- What is being tested?
- RO7049665
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 14 sitesin 8 countries
- Study dates
- 19 Apr 2021to 18 Nov 2021
- Phase
- Phase 2
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants with a definite diagnosis of AIH (type 1, 2 and 3) as per simplified or revised original diagnostic criteria
- Participants who have been in biochemical remission for > 2 years (or less if according to the local practice) prior to randomization
- Participants who have been on stable treatment (corticosteroids [CCSs] +/- non-specific immunosuppressants [NSIs]) for at least 3 months prior to randomization and who have not had a dose increase in the previous 6 months prior to randomization
- No signs of liver inflammation on a liver biopsy taken no more than 12 months prior to randomization
- Participants with AIH who have previously not attempted (or not attempted in the last 3 years, if this is the local practice) to taper CCSs to 0 mg/day
- Body mass index within the range of 18-35 kilograms per meter square (kg/m^2)
- Women of childbearing potential who agree to remain abstinent or use at least one acceptable contraceptive method during the treatment period and for at least 28 days after the final dose of study drug
Who cannot join
- Participants with cirrhosis (F4 fibrosis by Fibroscan®) with significant impairment of liver function (Child Pugh category B or C)
- Any other autoimmune disease requiring immunomodulating treatment
- History of infection with hepatitis B, human immunodeficiency virus, active hepatitis C virus (HCV) infection, detection of replicating cytomegalovirus (CMV) or Epstein-Barr virus (EBV)
- Active infections requiring systemic therapy with antibiotic, antiviral, or antifungal treatment or febrile illness within 7 days before Day-1
- History of primary or acquired immunodeficiency
- Pregnant or lactating female participants
Show all 20Show fewer
- Symptomatic herpes zoster within 3 months prior to screening
- History of active or latent tuberculosis or a positive Quantiferon Gold test
- History of clinically significant severe drug allergies, multiple drug allergies, allergy to any constituent of the product, or intolerance to topical steroids
- Lymphoma, leukemia, or any malignancy within the past 5 years, except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years and in situ carcinoma of the cervix that was completely removed surgically. Breast cancer within the past 10 years
- Significant uncontrolled comorbidity, such as cardiac, pulmonary, renal, hepatic, endocrine, or gastrointestinal disorders
- Any condition or disease detected during the medical interview/physical examination that would render the participant unsuitable for the study, place the participant at undue risk, or interfere with the ability of the participant to complete the study in the opinion of the Investigator
- CCSs of <5 mg/day, or <2.5 mg CCSs plus immune suppressant, or <3 mg/day budesonide with or without immune suppressant
- CCSs >20 mg/day or >9 mg/day budesonide
- Non-specific immunosuppressant (NSI) daily dose higher than recommended standard of care therapy
- T or B cell-depleting therapy within the last 12 months or T- or B-cell number below normal due to depleting therapy
- Leukocyte apheresis within 12 weeks of screening
- Donation of blood or blood products in excess of 500 milliliters (mL) within 3 months prior to screening.
- Exposure to any investigational treatment within 6 months prior to Day 1
- Abnormal hematologic, hepatic enzyme, hepatic function, or biochemistry values
Trial locations and contacts
14 sites in 8 countries.
14 sites in 8 countries
South Korea3 sites
Pusan National University Hospital; division of pulmonology
Busan
Korea University Ansan Hospital
Gyeonggi-do, 15355
University of Ulsan College of Medicine - Asan Medical Center (AMC) - Asan Liver Center
Seoul, KOR
Canada2 sites
Universite de Montreal - Centre Hospitalier de l'Universite de Montreal CHUM - Hopital Saint-Luc
Montreal, Quebec, H2X 0A9
Toronto General Hospital
Toronto, Ontario, M5G 2C4
Italy2 sites
IRCCS Saverio De Bellis; Anatomia Patologica
Castellana Grotte, Apulia, 70013
Ospedale San Gerardo
Monza, Lombardy, 20900
Netherlands2 sites
Amsterdam UMC - location AMC
Amstermdam, 1105 AZ
Radboud Universiteit - Radboud Universitair Medisch Centrum Radboudumc
Nijmegen, 6525 GA
United Kingdom2 sites
King College Hospital NHS Foundation Trust
London, SE5 9RS
Nottingham University Hospitals NHS Trust - City Hospital
Nottingham, NG5 1PB
Australia1 site
The Alfred Hospital - Professor Stuart Roberts' Clinic - The Alfred Centre Location
Melbourne, Victoria, 3004
Germany1 site
Martin Zeitz Centrum für Seltene Erkrankungen ZSE Hamburg
Hamburg, 20246
About this study
Official title: A Double-Blind, Randomized, Parallel-Group, Phase 2 Study to Investigate the Effect of RO7049665 on the Time to Relapse Following Steroid Tapering in Patients With Autoimmune Hepatitis
The primary objective of the study is to evaluate the effect of RO7049665 on time to relapse following forced corticosteroid (CCS) tapering as measured by the hazard ratio between RO7049665 7.5 milligrams (mg) and placebo arm.
- Conditions
- Autoimmune Hepatitis, Autoimmune Chronic Hepatitis
- Being tested
- RO7049665, Placebo
- Medical subject headings
- Hepatitis, Autoimmune
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RO7049665Drug
RO7049665, subcutaneous injection, Q2W.
PlaceboOther
RO7049665-matching placebo, subcutaneous injection, Q2W.
| Study group | Role | What they receive |
|---|---|---|
| RO7049665 3.5 mg | Experimental | Participants will receive RO7049665 3.5 mg, administered as subcutaneous (SC) injection, every 2 weeks (Q2W) until participants experience relapse or the study is closed. Receives: Drug: RO7049665 |
| RO7049665 7.5 mg | Experimental | Participants will receive RO7049665 7.5 mg, administered as SC injection, Q2W until participants experience relapse or the study is closed. Receives: Drug: RO7049665 |
| Placebo | Placebo comparator | Participants will receive RO7049665-matching placebo, administered as SC injection, Q2W until participants experience relapse or the study is closed. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Relapse for RO7049665 7.5 mg Versus Placebo
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: From randomization (Day 1) up to relapse or end of the study (up to approximately 25 months)
Secondary outcomes (6)
Change From Baseline in Alanine Aminotransferase (ALT)
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: Up to end of the study (up to approximately 25 months)
Change From Baseline in Aspartate Aminotransferase (AST)
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: Up to end of the study (up to approximately 25 months)
Change From Baseline in Immunoglobulin G (IgG)
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: Up to end of the study (up to approximately 25 months)
Time to Relapse for RO7049665 3.5 mg Versus Placebo
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: From Randomization (Day 1) up to relapse or end of the study (up to approximately 25 months)
Percentage of Participants With Adverse Events (AEs)
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: Up to end of the study (up to approximately 25 months)
Number of Participants With Anti-drug Antibody (ADA) Emergence and Neutralizing Potential
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
Measured: Up to end of the study (up to approximately 25 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Mar 2021
- Study start19 Apr 2021
- Primary completion18 Nov 2021
- Study completion18 Nov 2021
Why it stopped: A lack of efficacy was seen with RO7049665 in a study of ulcerative colitis. This reduces the likelihood that the drug is effective in autoimmune hepatitis.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Autoimmune HepatitisStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04790916
- Sponsor's protocol ID
- BP42698
- Other IDs
- 2020-003990-23
- Status last verified
- Nov 2022
- First submitted
- 8 Mar 2021
- Last updated
- 18 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research\_and\_development/who\_we\_are\_how\_we\_work/clinical\_trials/our\_commitment\_to\_data\_sharing.htm).
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Randomized, Parallel-Group, Phase 2 Study to Investigate the Effect of RO7049665 on the Time to Relapse Following Steroid Tapering in Patients With Autoimmune Hepatitis
- ClinicalTrials.gov ID
- NCT04790916
- Acronym / sponsor ID
- BP42698
- Conditions
- Autoimmune Hepatitis, Autoimmune Chronic Hepatitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: A lack of efficacy was seen with RO7049665 in a study of ulcerative colitis. This reduces the likelihood that the drug is effective in autoimmune hepatitis.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 19 Apr 2021 (actual)
- Primary completion
- 18 Nov 2021 (actual)
- Study completion
- 18 Nov 2021 (actual)
- First posted
- 10 Mar 2021 (actual)
- Results
- Posted 18 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of the study is to evaluate the effect of RO7049665 on time to relapse following forced corticosteroid (CCS) tapering as measured by the hazard ratio between RO7049665 7.5 milligrams (mg) and placebo arm.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| RO7049665 3.5 mg | Experimental | Drug: RO7049665Participants will receive RO7049665 3.5 mg, administered as subcutaneous (SC) injection, every 2 weeks (Q2W) until participants experience relapse or the study is closed. |
| RO7049665 7.5 mg | Experimental | Drug: RO7049665Participants will receive RO7049665 7.5 mg, administered as SC injection, Q2W until participants experience relapse or the study is closed. |
| Placebo | Placebo comparator | Other: PlaceboParticipants will receive RO7049665-matching placebo, administered as SC injection, Q2W until participants experience relapse or the study is closed. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Participants with a definite diagnosis of AIH (type 1, 2 and 3) as per simplified or revised original diagnostic criteria
- Participants who have been in biochemical remission for > 2 years (or less if according to the local practice) prior to randomization
- Participants who have been on stable treatment (corticosteroids [CCSs] +/- non-specific immunosuppressants [NSIs]) for at least 3 months prior to randomization and who have not had a dose increase in the previous 6 months prior to randomization
- No signs of liver inflammation on a liver biopsy taken no more than 12 months prior to randomization
- Participants with AIH who have previously not attempted (or not attempted in the last 3 years, if this is the local practice) to taper CCSs to 0 mg/day
Key exclusion criteria (20 listed)
- Participants with cirrhosis (F4 fibrosis by Fibroscan®) with significant impairment of liver function (Child Pugh category B or C)
- Any other autoimmune disease requiring immunomodulating treatment
- History of infection with hepatitis B, human immunodeficiency virus, active hepatitis C virus (HCV) infection, detection of replicating cytomegalovirus (CMV) or Epstein-Barr virus (EBV)
- Active infections requiring systemic therapy with antibiotic, antiviral, or antifungal treatment or febrile illness within 7 days before Day-1
- History of primary or acquired immunodeficiency
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RO7049665RO7049665, subcutaneous injection, Q2W. | Drug | RO7049665 3.5 mg, RO7049665 7.5 mg |
| PlaceboRO7049665-matching placebo, subcutaneous injection, Q2W. | Other | Placebo |
Primary outcome
Time to Relapse for RO7049665 7.5 mg Versus Placebo
Time frame: From randomization (Day 1) up to relapse or end of the study (up to approximately 25 months)
The study was terminated by the Sponsor. Only 2 participants were enrolled in this study. Based on the low enrollment number, no data is reported here in order to protect and maintain participant privacy/confidentiality.
6 secondary outcomes
Change From Baseline in Alanine Aminotransferase (ALT)
Time frame: Up to end of the study (up to approximately 25 months)
Change From Baseline in Aspartate Aminotransferase (AST)
Time frame: Up to end of the study (up to approximately 25 months)
Change From Baseline in Immunoglobulin G (IgG)
Time frame: Up to end of the study (up to approximately 25 months)
Time to Relapse for RO7049665 3.5 mg Versus Placebo
Time frame: From Randomization (Day 1) up to relapse or end of the study (up to approximately 25 months)
Percentage of Participants With Adverse Events (AEs)
Time frame: Up to end of the study (up to approximately 25 months)
Number of Participants With Anti-drug Antibody (ADA) Emergence and Neutralizing Potential
Time frame: Up to end of the study (up to approximately 25 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to individual patient level data through the clinical study data request platform (www.vivli.org). Further details on Roche's criteria for eligible studies are available here (https://vivli.org/ourmember/roche/). For further details on Roche's Global Policy on the Sharing of Clinical Information and how to request access to related clinical study documents, see here (https://www.roche.com/research_and_development/who_we_are_how_we_work/clinical_trials/our_commitment_to_data_sharing.htm).
Related trials for Autoimmune Hepatitis
RecruitingPhase 3
A Trial of Inebilizumab in Participants With Autoimmune Hepatitis
Autoimmune Hepatitis, AIH
RecruitingPhase 2
Belimumab in Autoimmune Hepatitis
Autoimmune Hepatitis
Enrolling by invitationObservational study
Comparative Autoantibody and Immunologic Cell Marker Study
Autoimmune Diseases, Lupus Erythematosus, Systemic
Not yet recruitingObservational study
Thyroid Disorders in Pediatric Autoimmune Hepatitis
Thyroid Disease, Autoimmune Hepatitis
RecruitingObservational study
A-LiNK: Improving Outcomes in Autoimmune Liver Disease
Autoimmune Hepatitis, Primary Sclerosing Cholangitis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
14 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing RO7049665 in people with Autoimmune Hepatitis and Autoimmune Chronic Hepatitis, run by Hoffmann-La Roche at 14 sites in 8 countries.
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 7 inclusion and 20 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: stopped early, as of the sponsor's last update on 18 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in South Korea, Canada, Italy, Netherlands, United Kingdom and 3 more countries. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 18 Sep 2023). 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?
Time to Relapse for RO7049665 7.5 mg Versus Placebo (time frame: From randomization (Day 1) up to relapse or end of the study (up to approximately 25 months)). 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 18 Sep 2023.