Riliprubart for Chronic Inflammatory Demyelinating Polyneuropathy

(MOBILIZE Trial)

Recruiting at 177 trial locations

Overseen By Trial Transparency email recommended (Toll free for US & Canada)

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Sanofi

Must be taking: Immunosuppressants, corticosteroids

Must not be taking: Chemotherapy, B-cell depleting

Disqualifiers: Diabetes, serious infections, SLE, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

140 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called riliprubart for individuals with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), a condition where the immune system attacks the nerves, causing weakness and numbness. The study aims to determine if riliprubart is more effective than a placebo for those whose symptoms have not improved with standard treatments like immunoglobulins or steroids. Participants will receive either riliprubart or a placebo for a period, followed by a phase where everyone receives riliprubart. Ideal candidates for this trial are those diagnosed with CIDP who have not responded well to treatments such as immunoglobulins or steroids. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications. However, you can continue taking certain immunosuppressants like azathioprine, cyclosporine, or mycophenolate mofetil if they have been stable for at least 3 months. Low-dose oral corticosteroids are also allowed if stable for 3 months before screening.

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

Research has shown that riliprubart appears safe for people with chronic inflammatory demyelinating polyneuropathy (CIDP). In earlier studies, participants tolerated riliprubart well, experiencing only mild to moderate side effects such as headaches and nausea, common with many treatments.

No serious safety issues have been reported so far, which encourages those considering joining a trial. It is important to remember that riliprubart is still under investigation, but current results are promising for its safety in humans. ^{1 2 3 4 5}

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

Riliprubart is unique because it targets Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) in a new way. Unlike the standard treatments like corticosteroids, IVIG, and plasma exchange, which mainly suppress the immune system, riliprubart is designed to specifically modulate the immune response, potentially offering a more targeted approach with fewer side effects. Researchers are excited because this could mean more effective management of CIDP with better patient outcomes and improved quality of life.

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

Research has shown that riliprubart might help treat chronic inflammatory demyelinating polyneuropathy (CIDP). In earlier studies, riliprubart, which blocks a protein called C1s, showed promising results in easing symptoms for people with CIDP. Specifically, early data indicated that patients experienced better nerve function and less inflammation. In this trial, participants will receive either riliprubart or a placebo for 24 weeks, followed by an open-label extension phase with riliprubart for an additional 24 weeks. These findings support further research into riliprubart's potential as a treatment for CIDP. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with a condition called CIDP, which affects the nerves and doesn't respond to usual treatments like immunoglobulin or corticosteroids. Participants must meet specific diagnostic criteria and have certain levels of disability as measured by medical scales.

Inclusion Criteria

I've been on a stable dose of my immunosuppressant for at least 3 months.

I have been vaccinated against certain bacterial infections within the last 5 years.

I am following the required birth control measures.

Exclusion Criteria

History of attempted suicide or presence of suicidal ideation

Positive results for specific infectious diseases

My condition is a specific type of chronic inflammatory demyelinating polyneuropathy.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either riliprubart or placebo for 24 weeks

24 weeks

Open-label Extension

Participants receive riliprubart for an additional 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Riliprubart

Trial Overview The study tests Riliprubart against a placebo in people with refractory CIDP over approximately 109 weeks. It aims to see if Riliprubart is more effective than a non-active treatment in improving symptoms.

How Is the Trial Designed?

Group I: Riliprubart Arm Experimental Treatment 2 Interventions

Riliprubart for 24 weeks followed by open-label extension phase with riliprubart for 24 weeks

Group II: Placebo Arm Placebo Group 2 Interventions

Placebo for 24 weeks followed by open-label extension phase with riliprubart for 24 weeks

Riliprubart is already approved in China, European Union for the following indications:

Approved in China as Riliprubart for:

• Chronic inflammatory demyelinating polyneuropathy (CIDP) - Breakthrough Therapy status

Approved in European Union as Riliprubart for:

• Chronic inflammatory demyelinating polyneuropathy (CIDP) - Orphan Drug status

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

Published Research Related to This Trial

In a 32-week study involving 67 adults with IVIg-dependent CIDP, interferon beta-1a (IFNbeta-1a) did not significantly reduce the total IVIg dose compared to placebo, indicating no overall benefit for the treatment in this population.

However, exploratory analyses suggested that IFNbeta-1a may be beneficial for patients with more severe symptoms or those requiring high doses of IVIg, highlighting a potential targeted use for this therapy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intramuscular interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy. Hughes, RA., Gorson, KC., Cros, D., et al.[2015]

In a 48-week study involving 82 patients with chronic inflammatory demyelinating polyneuropathy (CIDP), subcutaneous IgPro20 (Hizentra) demonstrated long-term efficacy, with lower relapse rates at the higher dose of 0.4 g/kg (10% relapse) compared to 0.2 g/kg (48% relapse).

The treatment was generally safe, with 76% of patients experiencing mild to moderate adverse events, and no serious adverse events related to the treatment, indicating that IgPro20 can be a viable long-term option for managing CIDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of subcutaneous immunoglobulin IgPro20 in CIDP: PATH extension study. van Schaik, IN., Mielke, O., Bril, V., et al.[2023]

In a study of 13 Italian patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) who did not respond to conventional therapies, 9 patients showed improvement after treatment with rituximab, indicating its potential efficacy in this condition.

Responses to rituximab were notably better in patients with shorter disease duration, with improvements observed after a median of 2 months and lasting for about a year, suggesting that early intervention may enhance treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab in patients with chronic inflammatory demyelinating polyradiculoneuropathy: a report of 13 cases and review of the literature. Benedetti, L., Briani, C., Franciotta, D., et al.[2017]

In a study of 28 CIDP patients, 75% responded positively to rituximab, showing significant clinical improvement within a median of 6 months, and only two patients required retreatment over an average follow-up of 2 years.

Rituximab was found to be effective for CIDP patients with both autoimmune and haematological disorders, with better responses observed in patients with shorter disease duration and common forms of CIDP. Importantly, no major adverse events were reported, indicating a favorable safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab in chronic inflammatory demyelinating polyradiculoneuropathy with associated diseases. Roux, T., Debs, R., Maisonobe, T., et al.[2019]

A new trial will investigate the efficacy of the anti-B cell agent rituximab and the anti-T cell agent abatacept in treating chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), involving 180 participants across three groups, which is larger than any previous immunosuppressant trials for this condition.

The primary outcome will measure improvement in neuropathy limitations at 12 weeks, with a focus on simplifying the trial design to enhance recruitment and reduce costs, aiming to provide valuable insights into effective treatments for CIDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Regarding the past, what is the trial you have always been dreaming of in CIDP? Hughes, RA., Lunn, MP.[2017]

Citations

1. 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 ...

2. 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.

3. 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 ... Sanofi's complement C1s inhibitor, riliprubart, showed encouraging efficacy and safety for participants with chronic inflammatory demyelinating polyneuropathy ...

4. 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).

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06290128

NCT06290128 | A Study to Test the Effects and Safety of ... The purpose of the study is to evaluate efficacy of riliprubart compared to placebo in adult participants with CIDP whose disease is refractory to standard ...

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. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37119056/

An innovative phase 2 proof-of-concept trial design to ... This phase 2, proof-of-concept, multicenter, open-label trial will evaluate the efficacy, and safety of SAR445088 in 90 patients with CIDP across three groups.