SAR445088 for Chronic Inflammatory Demyelinating Polyneuropathy

Recruiting at 78 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

110 Participants Needed

Trial Summary

What is the purpose of this trial?

Primary Objectives: * Part A: Efficacy of SAR445088 across three subpopulations of CIDP patients: standard of care (SOC)-Treated, SOC-Refractory and SOC-Naive * Part B:Long-term safety and tolerability of SAR445088 in CIDP Secondary Objectives: * Part A: * Safety and tolerability of SAR445088 in CIDP * Immunogenicity of SAR445088 * Efficacy of SAR445088 with overlapping SOC (SOC-Treated group) * Part B: * Durability of efficacy during long-term treatment with SAR445088 in CIDP * Long-term immunogenicity of SAR445088 in CIDP

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.

What data supports the effectiveness of the drug SAR445088 for treating Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)?

While there is no direct data on SAR445088, similar treatments like intravenous immunoglobulin (IVIg) have shown significant improvements in neurological function for CIDP patients, suggesting potential effectiveness for SAR445088. ^{1 2 3 4 5}

What makes the drug SAR445088 unique for treating CIDP?

SAR445088, also known as Riliprubart or TNT-020, is unique because it offers different administration routes, including intravenous (IV) and subcutaneous (SC), which may provide flexibility in treatment options compared to traditional therapies like corticosteroids and intravenous immunoglobulin. This drug is being investigated specifically for its potential to address the underlying immune mechanisms of CIDP, which are not fully targeted by existing treatments. ^{1 2 3 6 7}

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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 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 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 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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of subcutaneous immunoglobulin IgPro20 in CIDP: PATH extension study. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Interferon beta-1a as an investigational treatment for CIDP. [2019]

3. France pubmed.ncbi.nlm.nih.gov

Regarding the past, what is the trial you have always been dreaming of in CIDP? [2017]

4. England 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. [2021]

5. England pubmed.ncbi.nlm.nih.gov

Intravenous immunoglobulin treatment in chronic inflammatory demyelinating polyneuropathy. A double-blind, placebo-controlled, cross-over study. [2022]

6. United States pubmed.ncbi.nlm.nih.gov

Intramuscular interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy. [2015]

7. United States pubmed.ncbi.nlm.nih.gov

Rituximab in chronic inflammatory demyelinating polyradiculoneuropathy with associated diseases. [2019]