Clinical trial NCT06798012: A Study of TAK-411 in Adults With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Official title: A Phase 2, Open-label, Proof-of-Concept Study to Investigate the Efficacy, Safety, and Tolerability of TAK-411 in Adult Subjects With Chronic Inflammatory Demyelinating Polyradiculoneuropathy (The CASCA Study)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
Keywords: Drug Therapy
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2025-01-29
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: CIDP is an autoimmune disease. This means that the body's germ fighting (immune) system attacks itself. In CIDP, the immune system attacks the protective covering around the nerves called myelin. Over time, these nerves lose their ability to send signals to the muscles in the body. This leads to muscle weakness and loss of sensation in arms and legs among other symptoms. Participants with CIDP can be treated with a protein called immunoglobulin (or IG).

TAK-411 is a special type of immune globulin G (hsIgG) that has been chemically changed. It is made from IG that comes from human plasma. This study will test if TAK-411 can decrease inflammation and improve symptoms of CIDP.

The main aim of this study is to check how TAK-411 affects the physical functioning of adults with CIDP when compared with results of the placebo group of a historical trial.

Participants may be treated with TAK-411 for up to 1 year (51 weeks) and will be followed up for 3 weeks after last dose.

During the study, participants may visit their study clinic up to approximately 21 times.


Eligibility criteria: Key Inclusion Criteria

* The participant is at least 18 years of age, inclusive, at the time of signing the Informed Consent Form (ICF).
* The participant has a body weight of less than or equal to (\<=) 150 kilogram (kg).
* The participant has a documented diagnosis of typical CIDP, as confirmed by a neurologist specializing/experienced in neuromuscular diseases and consistent with the European Academy of Neurology/Peripheral Nerve Society (EAN/PNS) 2021 criteria.
* The participant has responded to IgG treatment in the past (documented partial or complete resolution of neurological symptoms and deficits).
* The participant has had disease activation within 18 months before screening, as documented in medical records and in the opinion of the investigator, defined as one of the following:

  1. Clinically meaningful deterioration of symptoms on interruption or dose reduction of IgG treatment.
  2. Clinically meaningful deterioration of symptoms requiring IgG treatment dose increase with subsequent clinical improvement.
  3. Clinically meaningful deterioration of symptoms at the end of IgG treatment dose interval with improvement after next dose administration.
* The participant is on a stable dose of immunoglobulin treatment intravenously (IGIV) treatment, defined as no change greater than 10 percentage (%) in frequency or dose of IGIV therapy within the 12 weeks before and throughout screening within the dose range of 0.4 to 2.4 grams per kilogram (g/kg) every 2 to 6 weeks (inclusive).
* The participant has an INCAT score greater than (\>) 2 at screening.

Key Exclusion Criteria

* The participant has a documented diagnosis of a CIDP variant per EAN/PNS 2021 criteria.
* The participant has any neuropathy of other causes, including the following:

  1. Hereditary demyelinating neuropathies, such as hereditary sensory and motor neuropathy, Charcot-Marie-Tooth disease, and hereditary sensory and autonomic neuropathies.
  2. Neuropathies secondary to infections, disorders, or systemic diseases such as Borrelia burgdorferi infection (Lyme disease), diphtheria, systemic lupus erythematosus, POEMS (polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes) syndrome, osteosclerotic myeloma, diabetic and nondiabetic lumbosacral radiculoplexus neuropathy, lymphoma, amyloidosis.
  3. Multifocal motor neuropathy.
  4. Drug-, biologic-, chemotherapy-, or toxin-induced peripheral neuropathy.
  5. Diabetic peripheral neuropathy.
* The participant has any chronic or debilitating disease, or central nervous disorder that causes neurological symptoms or that may interfere with assessment of CIDP or outcome measures, including (but not limited to) multiple sclerosis, arthritis, stroke, and Parkinson's disease.
* The participant is required to take or has taken immunomodulatory/immunosuppressive agents (except IGIV) that include, but are not limited to, complement inhibitors, rituximab, efgartigimod, and chemotherapeutic drugs, within 6 months of screening.
* The participant has undergone plasma exchange within 3 months of screening.
* The participant has a history of malignancy with less than 2 years of complete remission before screening, or active malignancy requiring chemotherapy and/or radiotherapy.

Note: Participants with adequately treated basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, or stable prostate cancer not requiring treatment are eligible.

* The participant has experienced deep vein thrombosis or arterial thromboembolic events (example, cerebrovascular accident, pulmonary embolism) within 12 months of screening.
* The participant has any medical condition, laboratory finding, or physical examination finding that precludes participation or with clinical evidence of any significant acute or chronic disease that, in the opinion of the investigator, may interfere with successful completion of the study or place the participant at undue medical risk.
* The participant has participated in another clinical study involving an IP or investigational device within 30 days before screening or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.

Primary outcomes:
measure: Number of Participants With Improvement in Functional Ability at Week 24
timeFrame: At Week 24
Secondary outcomes:
measure: Number of Participants With Improvement in Functional Ability at Weeks 12 and 54
timeFrame: At 12 and 54 weeks
measure: Change From Baseline in Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Score
timeFrame: Baseline (last assessment prior first dose on Day 1), at 12, 24 and 54 weeks
measure: Change From Screening in the Adjusted INCAT Score
timeFrame: Screening, at 12, 24 and 54 weeks
measure: Number of Participants With Improvement in Functional Ability on Inflammatory Rasch-built Overall Disability Scale (I-RODS) Score
timeFrame: At 12, 24 and 54 weeks
measure: Change From Baseline in I-RODS Score
timeFrame: Baseline (last assessment prior first dose on Day 1), at 12, 24 and 54 weeks
measure: Change From Screening in I-RODS Score
timeFrame: Screening, at 12, 24 and 54 weeks
measure: Change From Baseline in Medical Research Council Sum Score (MRC-SS)
timeFrame: Baseline (last assessment prior first dose on Day 1), at 12, 24 and 54 weeks
measure: Change From Screening in MRC-SS
timeFrame: Screening, at 12, 24 and 54 weeks
measure: Change From Baseline in Bilateral Hand Grip Strength
timeFrame: Baseline (last assessment prior first dose on Day 1), at 12, 24 and 54 weeks
measure: Change From Screening in Bilateral Grip Strength
timeFrame: Screening, at 12, 24 and 54 weeks
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: From start of study drug administration up to 54 weeks
measure: Number of Participants With Infusion Discontinuations, Interruptions, and Infusion Rate Reductions due to TAK-411 Related TEAEs
timeFrame: From start of study drug administration up to 54 weeks
measure: Number of Participants With Antibodies Against Host Cell Proteins (HCP), Beta-1,4-galactosyltransferase (B4GalT1) and Alpha-2,6-sialyltransferase (St6Gal1)
timeFrame: Up to 54 weeks
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Perelman Center for Advanced Medicine, University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: