Riliprubart for CIDP

(LTS Trial)

Recruiting at 35 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Riliprubart

Disqualifiers: Pregnancy, Lupus, Hypersensitivity, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 3 years

300 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the long-term safety and effectiveness of riliprubart, a treatment for chronic inflammatory demyelinating polyneuropathy (CIDP), a nerve disorder. It serves as a follow-up study for individuals who have completed earlier phases of riliprubart trials and wish to continue the treatment. Participants will receive riliprubart through a prefilled pen injection for up to three years. The trial is suitable for those with CIDP who have finished specific previous studies with riliprubart. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to the potential availability of a new treatment.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It is best to discuss this with the study team or your doctor.

Is there any evidence suggesting that riliprubart is likely to be safe for humans?

Research has shown that riliprubart is generally safe based on past studies. Earlier research found it well-tolerated by people with chronic inflammatory demyelinating polyneuropathy (CIDP). Most side effects were mild to moderate, such as headaches or slight irritation at the injection site. Importantly, the available data has not found any serious side effects directly linked to the treatment. The ongoing study aims to further explore and confirm these safety findings over a longer period. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for CIDP?

Unlike the standard treatments for CIDP, which often include intravenous immunoglobulin (IVIG) or corticosteroids, Riliprubart is unique because it is administered via a subcutaneous injection using a prefilled pen. This method can offer more convenience and potentially fewer side effects compared to traditional intravenous infusions. Researchers are excited about Riliprubart because it could make managing CIDP easier and more comfortable for patients, with the possibility of improved adherence to treatment.

What evidence suggests that riliprubart might be an effective treatment for CIDP?

Research has shown that riliprubart, the treatment under study in this trial, may help treat chronic inflammatory demyelinating polyneuropathy (CIDP). In earlier studies, about half of the patients experienced improvements in symptoms and health markers over 24 weeks. Riliprubart also improved quality of life and reduced tiredness for participants. These findings suggest that riliprubart, which blocks a part of the immune system involved in inflammation, could be effective for some people with CIDP. ^{2 3 6 7 8}

Are You a Good Fit for This Trial?

This trial is for adults with Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) who have already been treated with riliprubart in earlier studies. They must be willing to use contraception and comply with study requirements.

Inclusion Criteria

I agree to use birth control as required during and after the study.

I have CIDP and completed treatment with riliprubart in specified studies.

I can sign and follow the study's consent form and its rules.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive riliprubart weekly for up to 3 years

3 years

12 visits (in-person)

Follow-up

Participants are monitored for safety for a minimum of 55 weeks after the last dose of riliprubart

55 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Riliprubart

Trial Overview The trial is testing the long-term safety and effectiveness of a medication called Riliprubart, administered through a prefilled pen, in individuals with CIDP over approximately 4 years.

How Is the Trial Designed?

Group I: riliprubart Experimental Treatment 1 Intervention

Participants receive subcutaneous injection with a riliprubart prefilled pen (PFP)

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University

Citations

1. sanofi.com sanofi.com/en/media-room/press-releases/2024/2024-06-25-20-30-00-2904145

Media Update: Riliprubart one-year results from phase 2 ... Additional results showed that riliprubart improved participant-reported fatigue and quality-of-life measurements as well as biomarkers ...

2. neurology.org neurology.org/doi/10.1212/WNL.0000000000210365

Phase 2 Efficacy and Safety of Riliprubart, a C1s- ... Objective:To report efficacy and safety results of riliprubart in chronic inflammatory demyelinating polyneuropathy (CIDP).

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06859099

NCT06859099 | Long-term Safety and Efficacy Study of ... The purpose of this study is to evaluate long-term safety and efficacy of riliprubart in adult participants with chronic inflammatory demyelinating ...

4. congress.sanofimedical.com congress.sanofimedical.com/s3fs-public/2024-04/Preliminary%20Efficacy%20and%20Safety%20Data%20from%20the%20Phase%202%20Trial%20of%20Riliprubart%20in%20CIDP_Oral%20Presentation.pdf?VersionId=hfNI7iUD27di.Uaem_cOA6GA0Rlxsnbr

Preliminary Efficacy and Safety Data from the Phase 2 Trial of CIDP, chronic inflammatory demyelinating polyneuropathy; EFNS, European ... These results demonstrate proof of concept for C1s inhibition with riliprubart in CIDP.

5. rarediseaseadvisor.com rarediseaseadvisor.com/news/riliprubart-improves-symptoms-half-soc-treated-refractory-cidp/

Riliprubart Improves Symptoms in Select Patients with CIDP Riliprubart improved symptoms and disease markers in roughly half of patients with CIDP over a 24-week period, a study found.

6. neurology.org neurology.org/doi/10.1212/WNL.0000000000204596

Preliminary Efficacy and Safety Data from the Phase 2 Trial ... Abstract. Objective: Report preliminary efficacy and safety results for riliprubart, a novel complement C1s-inhibitor, in people with CIDP.

7. sanofi.com sanofi.com/en/media-room/press-releases/2025/2025-06-30-05-00-00-3107129

Press Release: Riliprubart granted orphan drug ... Long-term, 76-week sustained efficacy and safety data from riliprubart's phase 2 study were presented at the Peripheral Nerve Society meeting in ...

8. sanofistudies.com sanofistudies.com/cwout/CW-V9DEXS//us/es/listing/312929/a-study-of-riliprubart/

Study on the Effectiveness and Safety of an Investigational ... A study of riliprubart against the usual treatment of intravenous immunoglobulin in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).