SAR445088 for Chronic Inflammatory Demyelinating Polyneuropathy

No longer recruiting at 84 trial locations

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

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Bioverativ, a Sanofi company

Must be taking: Immunoglobulins, Corticosteroids

Must not be taking: Antidepressants, Chemotherapy

Disqualifiers: Diabetes, Infections, SLE, others

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

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

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment called SAR445088 (also known as BIVV020) for individuals with chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that affects the nerves and causes weakness and numbness. The goal is to evaluate the effectiveness of SAR445088 in various groups of CIDP patients, including those already on standard treatments, those who do not respond to standard treatments, and those who have not yet tried them. The trial will also assess the long-term safety of this treatment. Individuals diagnosed with CIDP who continue to experience symptoms despite treatment may be suitable candidates for this trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants, offering an opportunity to contribute to significant advancements in CIDP treatment.

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

The trial does not specify if you need to stop your current medications. However, if you are in the SOC-Refractory group, certain immunosuppressant drugs are allowed if taken for at least 6 months and at a stable dose for 3 months before screening. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that SAR445088 is likely to be safe for humans?

Research has shown that SAR445088, also known as riliprubart, has undergone safety testing in individuals with chronic inflammatory demyelinating polyneuropathy (CIDP). Studies have found that this treatment is generally well-tolerated. In a recent study, no major safety issues emerged after one year of use. Participants did not experience significant side effects, suggesting the treatment is safe for long-term use in CIDP. These findings indicate that SAR445088 is a promising option, with a safety profile that supports its use in clinical trials. ^{1 2 3 4 5}

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

Unlike standard treatments for Chronic Inflammatory Demyelinating Polyneuropathy (CIDP), which typically involve corticosteroids, immunosuppressants, or intravenous immunoglobulin, SAR445088 is unique because it targets the underlying inflammation differently. Researchers are excited because SAR445088 offers both intravenous (IV) and subcutaneous (SC) administration options, potentially providing more flexibility and convenience for patients. Additionally, the treatment's novel mechanism could offer better efficacy or fewer side effects than current options, which is promising for improving long-term outcomes for those living with CIDP.

What evidence suggests that SAR445088 could be an effective treatment for chronic inflammatory demyelinating polyneuropathy?

Research shows that SAR445088, also known as riliprubart, offers promising results for treating chronic inflammatory demyelinating polyneuropathy (CIDP). In earlier studies, riliprubart effectively blocked a part of the immune system called C1s complement, which contributes to CIDP. This blocking led to noticeable improvements in symptoms for CIDP patients. Early data suggests that the treatment is both effective and safe for long-term use. In this trial, participants will receive SAR445088 in various treatment arms, exploring different dosing strategies and combinations with standard-of-care therapies. These findings indicate that SAR445088 could be a helpful option for those dealing with CIDP. ^{1 2 3 4 5}

Who Is on the Research Team?

Clinical Sciences & Operations

Principal Investigator

Sanofi

Are You a Good Fit for This Trial?

Adults over 18 with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) who are either untreated, have not responded well to standard treatments, or cannot continue those treatments due to side effects. Participants must be able to consent and women of childbearing age must use effective contraception.

Inclusion Criteria

My condition did not improve after 12 weeks of standard treatment.

I am either new to treatment, have had standard treatment, or my condition didn't improve with standard treatment.

SOC-Naive (all criteria a-c must be met): a) Participants without previous treatment for CIDP or participants who received immunoglobulins (IVIg or SCIg) or corticosteroids but were stopped for reasons other than lack of response or side effects. b) Not treated with immunoglobulins (IVIg or SCIg) or corticosteroids for at least 6 months prior to screening. c) INCAT score: 2-9 (a score of 2 should be exclusively from leg disability component of INCAT).

Exclusion Criteria

Pregnant (defined as positive β-HCG blood test) or lactating females.

I haven't been hospitalized for serious infections in the last 30 days and don't have any active infections needing treatment.

I haven't taken strong immune or cancer drugs in the last 6 months.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

up to 6 weeks

Treatment Part A

Participants receive SAR445088 for 24 weeks. Includes overlap period with SOC therapy for some groups.

24 weeks

Safety Follow-up Part A

Participants who do not enroll into Part B attend a final safety follow-up visit.

22 weeks

Treatment Part B (Extension)

Participants continue receiving SAR445088 for an additional 52 weeks.

52 weeks

Safety Follow-up Part B

Participants who do not enroll into Part C attend a safety follow-up visit.

22 weeks

Treatment Part C

Participants continue receiving SAR445088 until the end of the study.

End of Study Follow-up

Final safety follow-up visit occurs 22 weeks after the last dose for the last participant.

22 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BIVV020
• BIVV020/SAR445088
• SAR445088 (IV)
• SAR445088 (SC)

Trial Overview The trial is testing SAR445088 for CIDP patients. It's given via IV or SC injections. The study has two parts: Part A tests the drug's effectiveness in different subpopulations; Part B looks at long-term safety and how well it works over time.

How Is the Trial Designed?

Group I: SOC-Treated Low Dose Experimental Treatment 2 Interventions

Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects SOC therapy; Weeks 13-24: SAR445088. Participants who do not enroll into Part B will attend a final safety follow-up visit that will take place 22 weeks after Week 24 (\~Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility, will be given the option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (\~week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study. Participants who discontinue at any time in Part C will attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose.

Group II: SOC-Treated Initial Dose Experimental Treatment 2 Interventions

Part A: Eligible participants will receive SAR445088 for 24 weeks. Weeks 1-12 (overlap period): Participants will be given SAR445088 with superimposing effects of SOC therapy; Weeks 13-24: SAR445088 given. Participants who do not enroll into Part B will attend final safety follow-up visit at 22 weeks after Week 24 (\~Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given option of rolling into Part B, and continue receiving SAR445088 for 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (\~week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit at 22 weeks after the last SAR445088 dose.

Group III: SOC-Refractory Low Dose Experimental Treatment 2 Interventions

Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continued eligibility and will be given the option to roll into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose.

Group IV: SOC-Refractory Initial Dose Experimental Treatment 2 Interventions

Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately (\~)at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose (approximately week 98) if they will not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after the last SAR445088 dose.

Group V: SOC-Naive Experimental Treatment 2 Interventions

Part A: Eligible participants will receive SAR445088 for 24 weeks. Participants who do not enroll into Part B will be asked to attend a final safety follow-up visit that will take place 22 weeks after Week 24 (approximately at Week 46). Part B: Participants who successfully complete Part A, will be reassessed for continuing eligibility and will be given the option of rolling into Part B, where they will continue receiving SAR445088 for an additional 52 weeks. At the end of the Part B treatment period, participants will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose (approximately week 98) if they do not continue in Part C. Part C: Participants from Part B who enter Part C will continue receiving SAR445088 until the end of study. Participants who discontinue at any time during Part C will be asked to attend a safety follow-up visit that will take place 22 weeks after last SAR445088 dose.

Who Is Running the Clinical Trial?

Bioverativ, a Sanofi company

Lead Sponsor

Trials

18

Recruited

1,000+

Published Research Related to This Trial

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 393 patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), 75% reported one or more comorbidities, which influenced treatment choices in nearly half of the cases, leading to less frequent use of corticosteroids.

Diabetes, monoclonal gammopathy of undetermined significance (MGUS), and other immune disorders were more common in CIDP patients compared to the general population, with diabetes specifically linked to higher disability scores and poorer treatment response.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Frequency of diabetes and other comorbidities in chronic inflammatory demyelinating polyradiculoneuropathy and their impact on clinical presentation and response to therapy. Doneddu, PE., Cocito, D., Manganelli, F., et al.[2021]

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]

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]

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. 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.

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

An innovative phase 2 proof-of-concept trial design to ... Methods: 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 ...

5. 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 ...