Batoclimab for CIDP

Recruiting at 134 trial locations

Overseen By Central Study Contact

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Immunovant Sciences GmbH

Must be taking: Immunoglobulin, Corticosteroids

Disqualifiers: Diabetes, Myelopathy, Hereditary neuropathy, 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

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

Compensation: Varies

Number of Visits: Unknown

277 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a multi-center, randomized, quadruple-blind, placebo-controlled study to evaluate the efficacy and safety of batoclimab in adult participants with active CIDP. The study includes an up to 4-week Screening Period, an up to 12-week Washout Period, a 12-week Randomized Treatment Period (Period 1), an up to 24-week Randomized Withdrawal Period (Period 2), an up to 52-week Long-term Extension (LTE) Period (optional), and Safety Follow-up 4 weeks after the last dose of study treatment. The total study duration will be up to approximately 109 weeks. Eligible participants will be assigned to one of four cohorts based upon their baseline CIDP treatment (Cohorts A and D - immunoglobulin \[Ig\] or plasma exchange \[PLEX\]; Cohort B - corticosteroids; Cohort C - naive or untreated in previous 3-24 months) and whether they meet diagnosis according to the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) criteria (Cohorts A, B, and C) or clinical criteria only (Cohort D) at the time of screening.

Will I have to stop taking my current medications?

The trial includes a 12-week washout period (time without taking certain medications), which suggests you may need to stop some current treatments. However, the protocol does not specify exactly which medications must be stopped, so it's best to discuss this with the trial team.

What data supports the effectiveness of the drug Batoclimab for treating CIDP?

Research indicates that targeting the neonatal Fc receptor (FcRn) with therapies like Batoclimab can effectively reduce circulating IgG levels, which is beneficial in autoimmune diseases. This approach has shown promise in clinical trials for autoimmune conditions, suggesting potential effectiveness for CIDP. ^{1 2 3 4 5}

Is Batoclimab generally safe for humans?

The research articles provided do not contain specific safety data for Batoclimab or its related names in humans. ^{1 2 3 6 7}

How does the drug Batoclimab work differently from other treatments for CIDP?

Batoclimab is unique because it is an anti-FcRn monoclonal antibody that works by blocking the neonatal Fc receptor (FcRn), which extends the half-life of IgG antibodies. This mechanism helps reduce the levels of pathogenic IgG antibodies in the body, potentially offering a novel approach to treating CIDP compared to traditional therapies. ^{1 2 3 8 9}

Eligibility Criteria

Adults over 18 with active Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP) who meet specific diagnostic criteria can join this trial. They should not have other types of polyneuropathies, IgM paraproteinemia, certain forms of CIDP like Distal or Sensory CIDP, poorly controlled diabetes, central demyelination signs, or be on high doses of corticosteroids.

Inclusion Criteria

Cohorts A and B: Motor nerve conduction criteria strongly supportive of demyelination

I have been diagnosed with a form of CIDP according to specific guidelines.

My tests show early signs of nerve damage.

Exclusion Criteria

I have a specific type of chronic inflammatory nerve disease.

I have diabetes.

I am taking prescribed oral corticosteroids regularly.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

up to 4 weeks

Washout

Participants undergo a washout period to clear previous treatments

up to 12 weeks

Randomized Treatment

Participants receive randomized treatment with batoclimab or placebo

12 weeks

Randomized Withdrawal

Participants undergo a withdrawal period to assess relapse rates

up to 24 weeks

Long-term Extension (optional)

Participants may opt into continuation of treatment long-term

up to 52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Batoclimab

Trial Overview The study tests Batoclimab's effectiveness and safety in treating CIDP. Participants will receive either Batoclimab at two different dosages or a placebo via subcutaneous injections for up to one year with various periods including washout and long-term extension phases.

Participant Groups

Group I: Withdrawal Period 2: Cohort D, Placebo Experimental Treatment 1 Intervention

Group II: Withdrawal Period 2: Cohort D, Dose 2 Experimental Treatment 1 Intervention

Group III: Withdrawal Period 2: Cohort C, Placebo Experimental Treatment 1 Intervention

Group IV: Withdrawal Period 2: Cohort C, Dose 2 Experimental Treatment 2 Interventions

Group V: Withdrawal Period 2: Cohort B, Placebo Experimental Treatment 1 Intervention

Group VI: Withdrawal Period 2: Cohort B, Dose 2 Experimental Treatment 2 Interventions

Group VII: Withdrawal Period 2: Cohort A, Placebo Experimental Treatment 1 Intervention

Group VIII: Withdrawal Period 2: Cohort A, Dose 2 Experimental Treatment 2 Interventions

Group IX: Treatment Period 1: Cohort D, Dose 2 Experimental Treatment 1 Intervention

Group X: Treatment Period 1: Cohort D, Dose 1 Experimental Treatment 1 Intervention

Group XI: Treatment Period 1: Cohort C, Dose 2 Experimental Treatment 1 Intervention

Group XII: Treatment Period 1: Cohort C, Dose 1 Experimental Treatment 1 Intervention

Group XIII: Treatment Period 1: Cohort B, Dose 2 Experimental Treatment 1 Intervention

Group XIV: Treatment Period 1: Cohort B, Dose 1 Experimental Treatment 1 Intervention

Group XV: Treatment Period 1: Cohort A, Dose 2 Experimental Treatment 1 Intervention

Group XVI: Treatment Period 1: Cohort A, Dose 1 Experimental Treatment 1 Intervention

Group XVII: LTE Period: Without Relapse in Period 2: Dose 2 Experimental Treatment 1 Intervention

Participants will receive Dose 2 for all 52 weeks.

Group XVIII: LTE Period: With Relapse in Period 2: Dose 1 and Dose 2 Experimental Treatment 2 Interventions

Participants will receive Dose 1 for the initial 4 weeks only and Dose 2 for the remaining 48 weeks.

Batoclimab is already approved in European Union, United States for the following indications:

Approved in European Union as Batoclimab for:

• Thyroid Eye Disease (TED)

Approved in United States as Batoclimab for:

• Thyroid Eye Disease (TED)

Who Is Running the Clinical Trial?

Immunovant Sciences GmbH

Lead Sponsor

Trials

12

Recruited

1,400+

Findings from Research

Engineered human IgG1 antibodies with enhanced binding to the Fc receptor FcRn showed significantly longer half-lives in mice expressing human FcRn, indicating that modifying antibodies can improve their effectiveness in the bloodstream.

These modified antibodies not only persisted longer but also effectively reduced the half-life of other antibodies, suggesting potential for treating autoimmune diseases by promoting the clearance of harmful IgG antibodies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Enhanced half-life of genetically engineered human IgG1 antibodies in a humanized FcRn mouse model: potential application in humorally mediated autoimmune disease. Petkova, SB., Akilesh, S., Sproule, TJ., et al.[2022]

A new panel of highly specific monoclonal antibodies (mAbs) against human FcRn has been developed, which can help researchers study the expression and function of this important receptor in the body.

These mAbs can selectively block the binding of IgG antibodies and serum albumin to FcRn, providing a valuable tool for exploring therapeutic strategies that target FcRn to enhance drug delivery and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Monoclonal antibodies directed against human FcRn and their applications. Christianson, GJ., Sun, VZ., Akilesh, S., et al.[2022]

References

1. Netherlands pubmed.ncbi.nlm.nih.gov

Robust recombinant FcRn production in mammalian cells enabling oriented immobilization for IgG binding studies. [2018]

2. England pubmed.ncbi.nlm.nih.gov

Enhanced half-life of genetically engineered human IgG1 antibodies in a humanized FcRn mouse model: potential application in humorally mediated autoimmune disease. [2022]

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

Monoclonal antibodies directed against human FcRn and their applications. [2022]

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

Targeting the neonatal Fc receptor (FcRn) to treat autoimmune diseases and maternal-fetal immune cytopenias. [2022]

5. China pubmed.ncbi.nlm.nih.gov

Research progress on neonatal Fc receptor and its application. [2022]

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

Pharmacokinetics of humanized monoclonal anti-tumor necrosis factor-{alpha} antibody and its neonatal Fc receptor variants in mice and cynomolgus monkeys. [2010]

7. Germany pubmed.ncbi.nlm.nih.gov

Intravenous immunoglobulin mediates an increase in anti-platelet antibody clearance via the FcRn receptor. [2018]

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

Characterization and screening of IgG binding to the neonatal Fc receptor. [2022]

9. England pubmed.ncbi.nlm.nih.gov

FcRn: the neonatal Fc receptor comes of age. [2023]