Long-term Safety and Tolerability Assessment of Batoclimab in Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

The primary objective of this study is to evaluate the long-term **safety** and **tolerability** of batoclimab in participants with **Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)**. This is clinically relevant as it aims to ensure that batoclimab, a solution for injection, can be safely administered over an extended period, which is crucial for managing a chronic condition like CIDP. The secondary objective is to assess the long-term clinical efficacy of batoclimab in these participants. Evaluating efficacy is important to determine the therapeutic benefits of batoclimab in improving or stabilizing the symptoms associated with CIDP over time.

The clinical trial involves a total of **73 participants** diagnosed with **Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)**. The study population includes both male and female subjects, with an age range that spans from young adults to older adults. Participants were selected based on their completion of a prior study, IMVT-1401-2401, and their potential benefit from continued access to the investigational drug, batoclimab, as assessed by the investigator. The trial does not include a vulnerable population. Participants are required to adhere to specific lifestyle considerations, such as the use of highly effective contraception methods for both male and female participants of childbearing potential, and restrictions on donating eggs or sperm during and after the study period. The trial aims to evaluate the long-term safety and tolerability of batoclimab in this specific patient group.

The clinical trial is designed to evaluate the long-term safety and tolerability of **batoclimab** in adult participants with **Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)**. This is a Phase 4, open-label extension study, which follows a therapeutic exploratory and confirmatory approach. The trial will involve participants who have completed a prior study, IMVT-1401-2401, or those who may benefit from continued access to batoclimab as determined by the investigator. The study is expected to commence recruitment on February 17, 2025, and conclude by November 12, 2027, with a maximum treatment period of 52 weeks.

Participants will receive batoclimab as a **solution for injection** administered subcutaneously. The trial will monitor the proportion of participants experiencing treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs leading to study treatment discontinuation. Secondary endpoints include changes from baseline in the Adjusted INCAT score and mean grip strength over time. The study will include an initial screening visit to confirm eligibility, followed by regular follow-up visits to assess safety and efficacy, and an end-of-study visit to conclude participation.

Participant involvement is expected to last up to 52 weeks, with conditions for early termination including the occurrence of significant adverse events or withdrawal of consent. Participants must agree to use effective contraception methods and refrain from donating eggs or sperm during the study and for 90 days after the final dose. The study prohibits participation in other interventional studies during the treatment period. The trial is not categorized as low intervention, and it does not involve a pediatric formulation. The study is conducted under the sponsorship of Immunovant Sciences GmbH, with batoclimab being the investigational product.

The clinical trial involves the administration of **Batoclimab**, an experimental medication developed by Immunovant Sciences GmbH. Batoclimab is a **solution for injection** intended for subcutaneous administration. The active substance, batoclimab, is a protein-based compound, specifically an anti-human FcRn receptor human monoclonal antibody, known by several synonyms including HL161BKN, HBM-9161, and RVT-1401. The maximum daily dose of batoclimab is 340 mg, with a total maximum dose of 17,680 mg over a treatment period of up to 52 weeks. The dosing schedule is designed to ensure optimal therapeutic outcomes while monitoring for safety and tolerability in participants with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).

In this study, no non-experimental treatments such as standard-of-care therapy, placebo, or comparator treatments are utilized. The focus is solely on evaluating the long-term safety and tolerability of batoclimab. Participant compliance with the dosing regimen is monitored throughout the trial to ensure adherence to the protocol and to accurately assess the medication's effects. The trial does not involve any pediatric formulations, and the product is not classified as an orphan drug. The administration of batoclimab is conducted under controlled conditions to maintain the integrity of the study and ensure participant safety.

Efficacy in the clinical trial will be assessed using both primary and secondary endpoints. The primary endpoint focuses on the safety profile of the treatment, specifically evaluating the proportion of participants experiencing treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs leading to discontinuation of the study treatment. Secondary endpoints will assess changes from baseline over time in specific clinical measures, including the **Adjusted INCAT** score and mean grip strength. These measures will provide insights into the functional improvements in participants with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) receiving Batoclimab. The collection and analysis of these efficacy parameters will be conducted at predetermined intervals throughout the study duration, ensuring a comprehensive evaluation of the treatment's impact on the disease.