Clinical trial NCT05581199: To Assess Efficacy and Safety of Batoclimab in Adult Participants With Active CIDP
Official title: A Phase 2b, Multi-center, Randomized, Quadruple-blind, Placebo-controlled Study of Batoclimab Treatment in Adult Participants With Active Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Chronic Inflammatory Demyelinating Polyneuropathy
Keywords: Chronic Inflammatory Demyelinating Polyneuropathy
IMVT-1401
Monoclonal antibody
Autoimmune disorders
Lead sponsor: Immunovant Sciences GmbH
Sponsor class: INDUSTRY
First posted: 2022-10-14
Enrollment: 277
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion criteria:

All Cohorts:

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 Diagnosis and Treatment of CIDP. Clinical criteria for typical CIDP and variants are as follows (either criterion must be met):

   1. 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
   2. 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

   Cohorts A and B:
3. Have electrodiagnostic test results supporting the diagnosis of CIDP in accordance with the EAN/PNS Guideline on Diagnosis and Treatment of CIDP; for Cohorts A and B, either criterion must be met:

   1. Motor nerve conduction criteria strongly supportive of demyelination.
   2. 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 \[defined as \> 70 milligrams per deciliter {mg/dL} or \> 10 mg/dL greater than years of age for those aged 60 years and over\] 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.

   Cohort C only:
4. 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).

   Cohort D only:
5. 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.

Additional inclusion criteria are defined in the protocol.

Exclusion Criteria:

All Cohorts:

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:

   1. Multifocal motor neuropathy
   2. Hereditary demyelinating neuropathy
   3. Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein and skin change syndromes (i.e., POEMS)
   4. Lumbosacral radiculoplexus neuropathy
   5. Systemic illnesses including vitamin deficiency syndromes and paraneoplastic neuropathies
   6. Drug- or toxin-induced
4. Have diabetes mellitus (DM) and meets any of the following criteria:

   1. Does not meet inclusion criteria 2(a) and 3(a).
   2. In the opinion of the Investigator, there is evidence of poorly controlled DM preceding the diagnosis of CIDP.
   3. 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.

Additional exclusion criteria are defined in the protocol.

Primary outcomes:
measure: Period 2, Cohort A: Proportion of participants who remain relapse-free at Week 36
timeFrame: Week 36
Secondary outcomes:
measure: Period 2, Cohort A: Time to first relapse relative to Period 2 Baseline
timeFrame: Baseline (Week 12) to Week 36
measure: Period 2, Cohort A: Change from Period 2 Baseline in Adj INCAT score
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohort A: Change from Period 2 Baseline in Inflammatory Rasch-built Overall Disability Scale (I-RODS)
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohort A: Change from Period 2 Baseline in Mean grip strength
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohort A: Change from Period 2 Baseline in Medical Research Council (MRC) Sum Score
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohort A: Change from Period 2 Baseline in Overall Neuropathy Limitations Scale (ONLS)
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Adj INCAT score
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A and B combined: Change from Period 2 Baseline in I-RODS
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A and B combined: Change from Period 2 Baseline in Mean Grip Strength
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A and B combined: Change from Period 2 Baseline in MRC sum score
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A and B combined: Change from Period 2 Baseline in ONLS
timeFrame: Baseline (Week 12) and up to Week 36
measure: Period 2, Cohorts A, B, and C: Proportion of participants who remain relapse-free at Week 36
timeFrame: Week 36
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Site Number - 1603
state: Arizona
city: Carlsbad
country: United States
facility: Site Number - 1618
state: California
city: Los Angeles
country: United States
facility: Site Number - 1634
state: California
city: Orange
country: United States
facility: Site Number - 1619
state: California
city: San Francisco
country: United States
facility: Site Number - 1608
state: California
city: Aurora
country: United States
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state: Colorado
city: Fort Collins
country: United States
facility: Site Number - 1636
state: Colorado
city: New Haven
country: United States
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state: Connecticut
city: Washington
country: United States
facility: Site Number - 1630
state: District of Columbia
city: Boca Raton
country: United States
facility: Site Number - 1600
state: Florida
city: Jacksonville
country: United States
facility: Site Number - 1609
state: Florida
city: Orlando
country: United States
facility: Site Number - 1631
state: Florida
city: Orlando
country: United States
facility: Site Number - 1629
state: Florida
city: Ormond Beach
country: United States
facility: Site Number - 1617
state: Florida
city: Port Charlotte
country: United States
facility: Site Number - 1620
state: Florida
city: Rockledge
country: United States
facility: Site Number - 1633
state: Florida
city: Saint Petersburg
country: United States
facility: Site Number - 1604
state: Florida
city: O'Fallon
country: United States
facility: Site Number - 1607
state: Illinois
city: Kansas City
country: United States
facility: Site Number - 1602
state: Kansas
city: Nicholasville
country: United States
facility: Site Number - 1611
state: Kentucky
city: Brooklyn
country: United States
facility: Site Number - 1622
state: New York
city: New York
country: United States
facility: Site Number - 1605
state: New York
city: Chapel Hill
country: United States
facility: Site Number - 1635
state: North Carolina
city: Charlotte
country: United States
facility: Site Number - 1610
state: North Carolina
city: Portland
country: United States
facility: Site Number - 1614
state: Oregon
city: Philadelphia
country: United States
facility: Site Number - 1623
state: Pennsylvania
city: Philadelphia
country: United States
facility: Site Number - 1624
state: Pennsylvania
city: Austin
country: United States
facility: Site Number -1601
state: Texas
city: Houston
country: United States
facility: Site Number - 1606
state: Texas
city: San Antonio
country: United States
facility: Site Number - 1628
state: Texas
city: Richmond
country: United States
facility: Site Number - 1627
state: Virginia
city: Seattle
country: United States
facility: Site Number - 1632
state: Washington
city: Milwaukee
country: United States
facility: Site Number - 1613
state: Wisconsin
city: Rosario
country: Argentina
facility: Site Number - 7753
state: Santa Fe
city: Rosario
country: Argentina
facility: Site Number - 7751
state: Santa Fe
city: Tucumán
country: Argentina
facility: Site Number - 7752
state: Tucuman
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Site Number - 7750
city: Gent
country: Belgium
facility: Site Number - 4681
state: Oost-Vlaanderen
city: Leuven
country: Belgium
facility: Site Number - 4680
state: Vlaams Brabant
city: Brasília
country: Brazil
facility: Site Number - 9103
state: Distrito Federal
city: Curitiba
country: Brazil
facility: Site Number - 9101
state: Paraná
city: Ribeirão Preto
country: Brazil
facility: Site Number - 9100
state: São Paulo
city: Rio De Janeiro
country: Brazil
facility: Site Number - 9102
city: São Paulo
country: Brazil
facility: Site Number - 9105
city: Sofia
country: Bulgaria
facility: Site Number - 9111
state: Sofia-Grad
city: Sofia
country: Bulgaria
facility: Site Number - 9110
state: Sofia-Grad
city: Pleven
country: Bulgaria
facility: Site Number - 9112
city: Edmonton
country: Canada
facility: Site Number - 2600
state: Alberta
city: Vancouver
country: Canada
facility: Site Number - 2603
state: British Columbia
city: Copenhagen
country: Denmark
facility: Site Number - 4740
city: Turku
country: Finland
facility: Site Number - 3241
state: Varsinais-Suomi
city: München
country: Germany
facility: Site Number - 6704
state: Bayern
city: Bochum
country: Germany
facility: Site Number - 6705
state: Nordrhein-Westfalen
city: Dresden
country: Germany
facility: Site Number - 6700
state: Sachsen
city: Leipzig
country: Germany
facility: Site Number - 6702
state: Sachsen
city: Berlin
country: Germany
facility: Site Number - 6706
city: Patras
country: Greece
facility: Site Number - 6341
state: Achaïa
city: Athens
country: Greece
facility: Site Number - 6344
state: Attiki
city: Athens
country: Greece
facility: Site Number - 6345
state: Attiki
city: Alexandroupolis
country: Greece
facility: Site Number - 6343
state: Evros
city: Ioánnina
country: Greece
facility: Site Number - 6346
state: Ioannina
city: Heraklion
country: Greece
facility: Site Number - 6342
state: Irakleio
city: Larisa
country: Greece
facility: Site Number - 6347
city: Gussago
country: Italy
facility: Site Number - 6302
state: Brescia
city: Bologna
country: Italy
facility: Site Number - 6305
state: Emilia-Romagna
city: Udine
country: Italy
facility: Site Number - 6304
state: Friuli-Venezia Giulia
city: Roma
country: Italy
facility: Site Number - 6309
state: Lazio
city: Roma
country: Italy
facility: Site Number - 6306
state: Lazio
city: Bergamo
country: Italy
facility: Site Number - 6301
state: Lombardia
city: Milano
country: Italy
facility: Site Number - 6307
state: Lombardia
city: Pisa
country: Italy
facility: Site Number - 6303
state: Toscana
city: Siena
country: Italy
facility: Site Number - 6308
state: Toscana
city: Pavia
country: Italy
facility: Site Number - 6300
city: Seoul
country: Korea, Republic of
facility: Site Number - 9901
city: Seoul
country: Korea, Republic of
facility: Site Number - 9900
city: Oslo
country: Norway
facility: Site Number - 6491
city: Wroclaw
country: Poland
facility: Site Number - 3204
state: Dolnoslaskie
city: Bydgoszcz
country: Poland
facility: Site Number - 3211
state: Kujawsko-pomorskie
city: Lublin
country: Poland
facility: Site Number - 3210
state: Lubelskie
city: Lublin
country: Poland
facility: Site Number - 3206
state: Lubelskie
city: Mazurki
country: Poland
facility: Site Number - 3202
state: Lubelskie
city: Krakow
country: Poland
facility: Site Number - 3209
state: Malopolskie
city: Krakow
country: Poland
facility: Site Number - 3200
state: Malopolskie
city: Kraków
country: Poland
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state: Malopolskie
city: Gdańsk
country: Poland
facility: Site Number - 3205
state: Pomorskie
city: Katowice
country: Poland
facility: Site Number - 3201
state: Slaskie
city: Poznan
country: Poland
facility: Site Number - 3207
state: Wielkopolskie
city: Bydgoszcz
country: Poland
facility: Site Number - 3203
state: Województwo Kujawsko-pomorskie
city: Senhora Da Hora
country: Portugal
facility: Site Number - 3742
state: Porto
city: Vila Nova De Gaia
country: Portugal
facility: Site Number - 3745
state: Porto
city: Almada
country: Portugal
facility: Site Number - 3743
state: Setubal
city: Lisboa
country: Portugal
facility: Site Number - 3741
city: Porto
country: Portugal
facility: Site Number - 3744
city: Bucharest
country: Romania
facility: Site Number - 8406
state: Bucuresti
city: Targu Mures
country: Romania
facility: Site Number - 8401
state: Mures
city: Timişoara
country: Romania
facility: Site Number - 8403
state: Timis
city: Constanta
country: Romania
facility: SIte Number - 8400
city: Belgrade
country: Serbia
facility: Site Number - 8501
city: Belgrade
country: Serbia
facility: Site Number - 8502
city: Niš
country: Serbia
facility: Site Number - 8500
city: Novi Sad
country: Serbia
facility: Site Number - 8503
city: Liptovský Mikuláš
country: Slovakia
facility: Site Number - 8600
city: Martin
country: Slovakia
facility: Site Number - 8601
city: Prešov
country: Slovakia
facility: Site Number - 8603
city: Trnava
country: Slovakia
facility: Site Number - 8602
city: Hospitalet de Llobregat
country: Spain
facility: Site Number - 3701
state: Barcelona
city: Sant Cugat Del Vallès
country: Spain
facility: Site Number - 3704
state: Barcelona
city: San Sebastián
country: Spain
facility: Site Number - 3700
state: Gipuzkoa
city: Barcelona
country: Spain
facility: Site Number - 3703
city: Göteborg
country: Sweden
facility: Site Number - 4891
state: Vastra Gotalands Lan
city: Cambridge
country: United Kingdom
facility: Site Number - 7405
state: Cambridgeshire
city: Southampton
country: United Kingdom
facility: Site Number - 7402
state: Hampshire
city: Glasgow
country: United Kingdom
facility: Site Number - 7404
state: Lanarkshire
city: Preston
country: United Kingdom
facility: Site Number - 7403
state: Lancashire
city: Sheffield
country: United Kingdom
facility: Site Number - 7401
state: South Yorkshire
city: Manchester
country: United Kingdom
facility: Site Number - 7400
Study officials:
References: