Immunoglobulin G4 Related DiseasePhase 2NCT04520451
Open Label Two-Arm Study to Evaluate Rilzabrutinib in IgG4-Related Disease Participants
A Phase 2 study testing rilzabrutinib and Glucocorticoids in people with Immunoglobulin G4 Related Disease, run by Principia Biopharma, a Sanofi Company at 9 sites in 5 countries.
Trial summary
This is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: rilzabrutinib with glucocorticoids and (2) Active Comparator: glucocorticoids only. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immunoglobulin G4 Related Disease
- What is being tested?
- rilzabrutinib and Glucocorticoids
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 9 sitesin 5 countries
- Study dates
- 22 Aug 2020to 15 Oct 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Be male or female with age ≥18 years.
- Have a clinical diagnosis of IgG4-RD.
- Be willing to taper off an equivalent prednisone dose of between 20-40 mg/day in 2 weeks.
Who cannot join
- Currently or within 6 months of screening taking rituximab, other B-cell depleting agents, or alkylating agents unless B cell concentrations have been demonstrated by flow cytometry to return to normal values (defined as 5 cells per cubic mm).
- History of solid organ transplant
- Positive at Screening for HIV, hepatitis B, hepatitis C, or TB
- Female patients who are pregnant or nursing.
- NOTE: Other Inclusion/Exclusion criteria may apply.
Trial locations and contacts
9 sites in 5 countries.
9 sites in 5 countries
United States5 sites
Investigational Site Number 84016
Boston, Massachusetts, 02114
Investigational Site Number 84018
Detroit, Michigan, 48202
Investigational Site Number 84030
New York, New York, 10021
Investigational Site Number 84036
Portland, Oregon, 97239
Investigational Site Number 84019
Stanford, California, 94305
About this study
Official title: An Open Label, Two-Arm Study to Evaluate the Effect of Rilzabrutinib (PRN1008/SAR444671) on Safety and Disease Activity in ParticipantsWith IgG4-Related Disease
This is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: rilzabrutinib with glucocorticoids and (2) Active Comparator: glucocorticoids only.
Read the detailed description
4 weeks of screening, 12 weeks of main treatment, 12 weeks of cross-over (for GC-only group), 40 weeks of extension treatment and 4-week follow-up.
- Conditions
- Immunoglobulin G4 Related Disease
- Being tested
- rilzabrutinib, Glucocorticoids
- Keywords
- +IgG4, glucocorticoid, IgG4-RD, BTK, Bruton's
- Medical subject headings
- Immunoglobulin G4-Related Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rilzabrutinibDrug
Also called PRN1008/SAR444671
oral tablet
GlucocorticoidsDrug
oral tablet or capsule
| Study group | Role | What they receive |
|---|---|---|
| Rilzabrutinib + glucocorticoids | Experimental | Rilzabrutinib tablets, 400 mg twice daily from Week 0 to Week 12 plus glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 2 weeks on study) Receives: Drug: rilzabrutinib; Drug: Glucocorticoids |
| Glucocorticoids | Active comparator | Glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 12 weeks on study) Receives: Drug: Glucocorticoids |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment
Disease flare is defined as an increase in IgG4-RD responder index (RI) >2 or initiation of rescue treatment.
Measured: Up to 64 weeks
Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE
Measured: Up to 68 weeks
Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG
Measured: Up to 68 weeks
Secondary outcomes (6)
Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time
Measured: At Week 52
Proportion of patients with an IgG4-RD RI activity score = 0 at over time
Measured: Up to 64 weeks
Level and change from baseline of each subclass of the serological markers over time
serum protein electrophoresis to determine IgG subclasses and complement factors C3 and C4
Measured: Up to 64 weeks
Proportion of participants achieving reduction in baseline serum IgG4 level of 10% over time
Measured: Up to 64 weeks
Change from baseline in IgG4-RD RI over time
Measured: From baseline up to 64 weeks
Change from baseline in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time
Measured: From baseline up to 64 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Aug 2020
- Study start22 Aug 2020
- Primary completion15 Oct 2024
- Study completion15 Oct 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immunoglobulin G4 Related DiseaseStudies of the same condition, open and under way.
- 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
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Principia Biopharma, a Sanofi Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Massachusetts General Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04520451
- Sponsor's protocol ID
- ACT17125
- Other IDs
- PRN1008-017, U1111-1260-3972, 2022-002959-18
- Status last verified
- Oct 2025
- First submitted
- 11 Aug 2020
- Last updated
- 14 Oct 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Wallace ZS, Zhang Y, Perugino CA, Naden R, Choi HK, Stone JH; ACR/EULAR IgG4-RD Classification Criteria Committee. Clinical phenotypes of IgG4-related disease: an analysis of two international cross-sectional cohorts. Ann Rheum Dis. 2019 Mar;78(3):406-412. doi: 10.1136/annrheumdis-2018-214603. Epub 2019 Jan 5.
PubMed 30612117
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Two-Arm Study to Evaluate the Effect of Rilzabrutinib (PRN1008/SAR444671) on Safety and Disease Activity in ParticipantsWith IgG4-Related Disease
- ClinicalTrials.gov ID
- NCT04520451
- Acronym / sponsor ID
- ACT17125
- Conditions
- Immunoglobulin G4 Related Disease
- Keywords
- +IgG4, glucocorticoid, IgG4-RD, BTK, Bruton's
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Principia Biopharma, a Sanofi CompanyIndustry (pharma, biotech, devices)
- Collaborators
- Massachusetts General Hospital
- Study start
- 22 Aug 2020 (actual)
- Primary completion
- 15 Oct 2024 (actual)
- Study completion
- 15 Oct 2024 (actual)
- First posted
- 20 Aug 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a Phase 2a, multi-center, open-label, two-arm study of approximately 25 patients with active IgG4-related disease (IgG4-RD). The two arms include (1) Experimental: rilzabrutinib with glucocorticoids and (2) Active Comparator: glucocorticoids only.
Detailed description
4 weeks of screening, 12 weeks of main treatment, 12 weeks of cross-over (for GC-only group), 40 weeks of extension treatment and 4-week follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rilzabrutinib + glucocorticoids | Experimental | Drug: rilzabrutinib, Drug: GlucocorticoidsRilzabrutinib tablets, 400 mg twice daily from Week 0 to Week 12 plus glucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 2 weeks on study) |
| Glucocorticoids | Active comparator | Drug: GlucocorticoidsGlucocorticoids (20 to 40 mg/day prednisone equivalent tapered to 0 mg/day within 12 weeks on study) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Be male or female with age ≥18 years.
- Have a clinical diagnosis of IgG4-RD.
- Be willing to taper off an equivalent prednisone dose of between 20-40 mg/day in 2 weeks.
Key exclusion criteria (5 listed)
- Currently or within 6 months of screening taking rituximab, other B-cell depleting agents, or alkylating agents unless B cell concentrations have been demonstrated by flow cytometry to return to normal values (defined as 5 cells per cubic mm).
- History of solid organ transplant
- Positive at Screening for HIV, hepatitis B, hepatitis C, or TB
- Female patients who are pregnant or nursing.
- NOTE: Other Inclusion/Exclusion criteria may apply.
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 |
|---|---|---|
| rilzabrutinibAlso: PRN1008/SAR444671oral tablet | Drug | Rilzabrutinib + glucocorticoids |
| Glucocorticoidsoral tablet or capsule | Drug | Glucocorticoids, Rilzabrutinib + glucocorticoids |
Primary outcome
Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment
Time frame: Up to 64 weeks
Disease flare is defined as an increase in IgG4-RD responder index (RI) >2 or initiation of rescue treatment.
Incidence of SAE, AE leading to discontinuation and possible glucocorticoid-related AE
Time frame: Up to 68 weeks
Number of participants with Potentially clinically significant abnormalities (PCSAs) for clinical laboratory tests, vital signs and ECG
Time frame: Up to 68 weeks
6 secondary outcomes
Proportion of participants with reduction from baseline IgG4-RD RI activity score by ≥2 points over time
Time frame: At Week 52
Proportion of patients with an IgG4-RD RI activity score = 0 at over time
Time frame: Up to 64 weeks
Level and change from baseline of each subclass of the serological markers over time
Time frame: Up to 64 weeks
Proportion of participants achieving reduction in baseline serum IgG4 level of 10% over time
Time frame: Up to 64 weeks
Change from baseline in IgG4-RD RI over time
Time frame: From baseline up to 64 weeks
Change from baseline in IgG4-RD damage, as recorded on the damage portion of the IgG4-RD RI over time
Time frame: From baseline up to 64 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2025.
- Background: PubMed 30612117Wallace ZS, Zhang Y, Perugino CA, Naden R, Choi HK, Stone JH; ACR/EULAR IgG4-RD Classification Criteria Committee. Clinical phenotypes of IgG4-related disease: an analysis of two international cross-sectional cohorts. Ann Rheum Dis. 2019 Mar;78(3):406-412. doi: 10.1136/annrheumdis-2018-214603. Epub 2019 Jan 5.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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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.
The record lists no contact. Use the official record to reach the sponsor.
9 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing rilzabrutinib and Glucocorticoids in people with Immunoglobulin G4 Related Disease, run by Principia Biopharma, a Sanofi Company at 9 sites in 5 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 3 inclusion and 5 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 14 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Canada, France, Italy, Spain. 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 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of participants who are without disease flare following the first dose of rilzabrutinib until the end of treatment (time frame: Up to 64 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04520451, which also lists 1 linked publication.
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 14 Oct 2025.