Riliprubart vs IVIg for CIDP

(VITALIZE Trial)

Recruiting at 161 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: IVIg

Must not be taking: Immunosuppressants, Corticosteroids, Chemotherapy, others

Disqualifiers: Diabetes, Infections, Lupus, 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

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

160 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to compare the effectiveness of a new treatment, riliprubart (an experimental treatment), with the current standard treatment, IVIg, for people with CIDP (Chronic Inflammatory Demyelinating Polyneuropathy). CIDP is a nerve disorder that causes weakness and numbness. Participants will receive either riliprubart or continue with IVIg to determine which better manages their symptoms. Those on a stable dose of IVIg with ongoing CIDP symptoms might be suitable for this trial. 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 requires participants to be on a stable maintenance dosage of IVIg (a type of medication) and does not specify stopping other medications. However, certain treatments like plasma exchange, immunosuppressive medications, and specific complement system inhibitors must not have been used recently. It's best to discuss your current medications with the study team to ensure eligibility.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that riliprubart has promising safety results from earlier studies. People with chronic inflammatory demyelinating polyneuropathy (CIDP) who used riliprubart found it well-tolerated. Some studies reported mild side effects, but these were not serious. In one study, riliprubart was tested for a year, and the safety results were positive, indicating no major health problems for participants. Discuss these findings with a healthcare provider to understand their implications. ^{1 2 3 4 5}

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

Most treatments for chronic inflammatory demyelinating polyneuropathy (CIDP) involve immunoglobulin therapy, like IVIg, which helps to modulate the immune system. But riliprubart works differently, targeting specific pathways involved in the inflammation process. Researchers are excited about riliprubart because it has a novel mechanism of action that could provide more targeted relief with potentially fewer side effects. Additionally, riliprubart could offer a new option for patients who do not respond well to traditional therapies.

What evidence suggests that this trial's treatments could be effective for CIDP?

This trial will compare Riliprubart with IVIg for treating chronic inflammatory demyelinating polyneuropathy (CIDP). Research has shown that Riliprubart may help treat CIDP by blocking C1s, a part of the immune system that can harm nerves in CIDP. In early trials, patients using Riliprubart experienced symptom improvements, suggesting it can effectively manage CIDP. Additionally, ongoing research has demonstrated Riliprubart's promising safety and effectiveness over one year. These findings support Riliprubart's potential as a helpful treatment option for those with CIDP. ^{1 2 5 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) who have responded to IVIg treatment in the past. They must meet specific diagnostic criteria, have a stable dose of IVIg, and some ongoing disability from CIDP. Vaccinations against certain bacteria are required, and participants must agree to use contraception.

Inclusion Criteria

I am willing to switch my IVIg infusions to a hospital setting before the study starts.

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

My IVIg dose has been stable for the last 8 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either Riliprubart + Placebo IVIg or IVIg + Placebo Riliprubart 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's effectiveness compared to standard IVIg treatment in managing CIDP symptoms over up to 109 weeks. Participants will be randomly assigned either the new drug or usual treatment and monitored for changes in their condition.

How Is the Trial Designed?

Group I: Riliprubart Arm Experimental Treatment 2 Interventions

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

Group II: IVIg Arm Active Control 3 Interventions

IVIg (IVIg continuation) + Placebo riliprubart 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

Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) patients often start treatment with intravenous immunoglobulin (IVIg) at a standard dose of 2 g/kg, but most require ongoing maintenance therapy due to limited sustained improvement.

There is significant variability in IVIg maintenance regimens among doctors, and the lack of evidence-based guidelines may lead to either under- or overtreatment, highlighting the need for standardized protocols to optimize therapy for CIDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Maintenance IV immunoglobulin treatment in chronic inflammatory demyelinating polyradiculoneuropathy. Kuitwaard, K., Fokkink, WR., Brusse, E., et al.[2018]

In a study of 41 patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), intravenous immunoglobulin (IVIg) was found to be more effective than intravenous methylprednisolone (IVMP), with 87.5% of IVIg patients improving compared to 54.2% of IVMP patients.

While both treatments had a high rate of relapse after discontinuation (85.7% for IVIg and 76.9% for IVMP), the median time to relapse was significantly longer for IVMP (14 months) compared to IVIg (4.5 months), suggesting that IVMP may provide a longer duration of benefit despite a lower initial response rate.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Frequency and time to relapse after discontinuing 6-month therapy with IVIg or pulsed methylprednisolone in CIDP. Nobile-Orazio, E., Cocito, D., Jann, S., et al.[2022]

Hizentra, a subcutaneous immunoglobulin therapy, offers significant cost savings of approximately $32,447 per patient annually compared to intravenous immunoglobulin (IVIG) for the maintenance treatment of chronic inflammatory demyelinating polyneuropathy (CIDP).

The analysis suggests that switching 10% of patients from IVIG to Hizentra could save a hypothetical 25-million-member health plan about $2.3 million, highlighting Hizentra's economic advantages and reduced patient burden due to self-administration and fewer adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Budget Impact Model of Maintenance Treatment of Chronic Inflammatory Demyelinating Polyneuropathy with IgPro20 (Hizentra) Relative to Intravenous Immunoglobulin in the United States. Mallick, R., Carlton, R., Van Stiphout, J.[2023]

In a pivotal phase III trial, maintenance therapy with subcutaneous immunoglobulin (IgPro20) for 24 weeks significantly reduced relapse rates in patients with chronic inflammatory demyelinating polyneuropathy (CIDP) compared to placebo, demonstrating its efficacy as a treatment option.

IgPro20 was well tolerated with low rates of systemic adverse events, and over half of the patients preferred it over their previous intravenous immunoglobulin (IVIg) therapy, indicating it may be a more convenient and safer alternative for long-term management of CIDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Immune Globulin Subcutaneous (Human) 20% (Hizentra®): A Review in Chronic Inflammatory Demyelinating Polyneuropathy. Lamb, YN., Syed, YY., Dhillon, S.[2022]

In a study involving 41 patients with chronic inflammatory demyelinating polyneuropathy (CIDP), switching from intravenous immunoglobulin (IVIg) to an equivalent dose of subcutaneous immunoglobulin (IgPro20) maintained stability in clinical outcomes such as disability and muscle strength over 6 months.

A significant 88% of patients preferred the subcutaneous IgPro20 treatment over IVIg after the switch, indicating not only efficacy but also improved patient satisfaction with the new treatment method.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Switch from intravenous to subcutaneous immunoglobulin IgPro20 in CIDP patients: a prospective observational study under real-world conditions. Gingele, S., Koch, M., Saparilla, AC., et al.[2022]

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

A study of riliprubart against the usual treatment ... The purpose of the study is to evaluate effectiveness and safety of riliprubart compared to intravenous immunoglobulin (IVIg) in adult participants with ...

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. 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 ...