Phase 2b Study to Assess Efficacy and Safety of Batoclimab in Adult Participants with Active CIDP.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Immunovant Sciences GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

27 Jun 2023 → 18 Apr 2026

Decision date (initial)

2024-08-28

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2024-512646-42-00

EudraCT number

2022-002718-17

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Pharmacokinetic, Pharmacodynamic, Dose response, Efficacy

Period 2/ Cohort A: To evaluate the efficacy of batoclimab compared to placebo in maintaining clinical response as assessed by adjusted inflammatory neuropathy cause and treatment (Adj INCAT) score in participants receiving immune globulin (IVIg or SCIg) or plasma exchange (PLEX) treatment for CIDP at the time of screening.

Secondary objectives 3

1. Period 2 / Cohort A: To evaluate the efficacy of batoclimab compared to placebo in maintaining clinical response in participants receiving immune globulin (IVIg or SCIg) or PLEX treatment for CIDP at the time of screening.
2. Period 2 / Cohort A and B combined: To evaluate the efficacy of batoclimab compared to placebo in maintaining clinical response in participants receiving any CIDP treatment at the time of screening.
3. Period 2 / Cohorts A, B and C combined: To evaluate the efficacy of batoclimab compared to placebo in maintaining clinical response regardless of CIDP treatment history.

Conditions and MedDRA coding

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Are >= 18 years at the Screening Visit.
2. Have met clinical diagnostic criteria for typical CIDP, or one of the following CIDP variants: multifocal CIDP, focal CIDP, or motor CIDP in accordance with the EAN/PNS Guideline on the Diagnosis and Treatment of CIDP. Clinical criteria for typical CIDP and variants are as follows (either criteria must be met): a. Typical CIDP: All the following: - Progressive or relapsing, symmetric, proximal, and distal muscle weakness of upper and lower limbs, and sensory involvement of at least two limbs (at any point in the disease course) - Developing over at least 8 weeks - Absent or reduced tendon reflexes in all limbs b. CIDP variants: One of the following, but otherwise as in typical CIDP (tendon reflexes may be normal in unaffected limbs): - Multifocal CIDP: documented sensory loss and muscle weakness in a multifocal pattern, usually asymmetric, upper limb predominant - Focal CIDP: sensory loss and muscle weakness in only one limb - Motor CIDP: motor symptoms and signs without sensory involvement
3. Cohorts A and B - Have electrodiagnostic test results supporting the diagnosis of CIDP in accordance with the EAN/PNS Guideline on the Diagnosis and Treatment of CIDP (either criteria must be met): a. Motor nerve conduction criteria strongly supportive of demyelination. b. Motor nerve conduction criteria weakly supportive of demyelination and 2 or more of the following additional diagnostic criteria: - Objective improvement to an empiric trial of therapy with immunoglobulin treatment, plasma exchange (PLEX) or corticosteroids. - Diagnostic imaging by ultrasound or magnetic resonance imaging (MRI) supporting the diagnosis of CIDP by demonstrating nerve enlargement. - Cerebrospinal fluid (CSF) demonstrating albuminocytologic dissociation (i.e., elevated CSF protein level [defined as > 70 milligrams per deciliter {mg/dL} or more than 10 mg/dL greater than years of age for those aged 60 years and over] with normal CSF white blood cell [WBC] level). - Nerve biopsy demonstrating features supporting the diagnosis of CIDP such as edema, demyelination, and/or onion bulb formation.
4. Cohort C only: Have a diagnosis of CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP based on clinical criteria and motor nerve conduction criteria strongly supportive of demyelination (i.e., motor nerve conduction criteria weakly supportive of demyelination is insufficient diagnostic evidence for admission to Cohort C)
5. Cohort D only: Have met only clinical diagnostic criteria for typical CIDP, or one of the following CIDP variants: multifocal CIDP, focal CIDP, or motor CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP. Either inclusion criterion 2(a) or 2(b) must be met.
6. Additional inclusion criteria are defined in the protocol.

Exclusion criteria 8

1. Have current or prior history of immunoglobulin M (IgM) paraproteinemia with or without anti-myelin-associated-glycoprotein antibodies.
2. Have Distal CIDP, Sensory CIDP or are suspected of having a diagnosis of auto-immune nodopathy in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP.
3. Have polyneuropathy of causes other than CIDP including but not limited to: a. Multifocal motor neuropathy b. Hereditary demyelinating neuropathy c. Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes (i.e., POEMS) d. Lumbosacral radiculoplexus neuropathy e. Systemic illnesses including vitamin deficiency syndromes and paraneoplastic neuropathies f. Drug- or toxin-induced
4. Have diabetes mellitus (DM) and meets any of the following criteria: a. Does not meet inclusion criteria 2(a) and 3(a) b. In the opinion of the Investigator, there is evidence of poorly controlled DM preceding the diagnosis of CIDP. c. In the opinion of the Investigator, there is evidence of poorly controlled DM at screening.
5. Have a history of myelopathy or evidence of central demyelination.
6. Are receiving chronic oral corticosteroids monotherapy at a dose > 40 mg/day prednisolone/prednisone or its equivalent at the Screening Visit.
7. Are receiving chronic oral corticosteroid at a dose > 10 mg/day prednisolone/prednisone or equivalent in combination with immunoglobulin therapy or PLEX at the Screening Visit.
8. Additional exclusion criteria are defined in the protocol.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Period 2 / Cohort A: Proportion of participants who remain relapse-free at Week 36 where relapse is defined as a worsening (increase) of ≥ 1 point on the Adj INCAT score at any time point during Period 2 relative to Period 2 baseline which is sustained at a Follow-Up visit 1 week later.

Secondary endpoints 3

1. Period 2 / Cohort A: -Time to first relapse relative to Period 2 baseline - Change from Period 2 baseline to Week 36 in: Adj INCAT score, Inflammatory Rasch-built Overall Disability Scale (I-RODS), Mean Grip Strength, Medical Research Council (MRC) Sum Score, Overall Neuropathy Limitations Scale (ONLS)
2. Period 2 / Cohort A and B combined: Change from Period 2 baseline to Week 36 in: Adj INCAT score, I-RODS, Mean Grip Strength, MRC Sum Score, ONLS
3. Period 2 / Cohorts A, B and C combined: Proportion of participants who remain relapse-free at Week 36.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Immunovant Sciences GmbH

Sponsor organisation

Immunovant Sciences GmbH

Address

Viaduktstrasse 8

City

Basel Town

Postcode

4051

Country

Switzerland

Scientific contact point

Organisation

Immunovant Sciences GmbH

Contact name

Immunovant Clinical Trials

Public contact point

Organisation

Immunovant Sciences GmbH

Contact name

Immunovant Clinical Trials

Third parties 15

Locations

14 EU/EEA countries · 54 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 14 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 215 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications