Refractory Chronic Inflammatory Demyelinating Polyneuropathy
Conditions
Brief summary
This multicenter, randomized, controlled trial aims to evaluate the efficacy and safety of Telitacicept in patients with refractory chronic inflammatory demyelinating polyneuropathy (CIDP). Eligible patients will be randomly assigned to receive either conventional therapy alone or Telitacicept plus conventional therapy for 24 weeks. Efficacy will be assessed using CIDP-related clinical and functional measures, including the Inflammatory Neuropathy Cause and Treatment (INCAT) disability score, Inflammatory Rasch-built Overall Disability Scale (I-RODS), Medical Research Council (MRC) sum score, grip strength, and the Timed Up and Go (TUG) test. Safety will be evaluated by monitoring adverse events, including their onset, duration, clinical manifestations, and management.
Interventions
DRUGConventional Therapy
Conventional therapy may include intravenous immunoglobulin or corticosteroids, with immunosuppressive agents permitted when clinically indicated.
DRUGTelitacicept
Telitacicept will be administered by subcutaneous injection in addition to conventional therapy for 24 weeks.
Sponsors
Sichuan Academy of Medical Sciences
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
(1) Male or female participants aged 18 to 65 years, inclusive. (2) Diagnosis of refractory chronic inflammatory demyelinating polyneuropathy (CIDP). Refractory CIDP is defined as the absence of evidence of clinical improvement (ECI) after at least 1 month of at least one first-line therapy, including intravenous immunoglobulin, corticosteroids, or plasma exchange, or a persistently elevated INCAT disability score of ≥2. ECI is defined as meeting at least one of the following criteria: a. A decrease of ≥1 point in the adjusted INCAT disability score; b. An increase of ≥4 points in the I-RODS total score; c. An increase of ≥3 points in the MRC sum score; d. An improvement of ≥8 kPa in grip strength. (3) INCAT disability score of 2 to 9. (4) Able to fully understand the study, willing to comply with study procedures, and able to provide written informed consent.
Exclusion criteria
1. Progressive neurological disease unrelated to CIDP. 2. Limb numbness or weakness caused by other etiologies, including but not limited to hereditary demyelinating neuropathy, neuropathy secondary to infection or systemic disease, diabetic neuropathy, drug- or toxin-induced neuropathy, multifocal motor neuropathy, polyneuropathy associated with IgM monoclonal gammopathy, POEMS syndrome, cerebral infarction, acute myelitis, multiple sclerosis, neuromyelitis optica spectrum disorder, or other conditions that may cause limb numbness or muscle weakness. 3. Active hepatitis or severe hepatic dysfunction, defined as liver function test values greater than two times the upper limit of normal. Participants who are positive for hepatitis B surface antigen (HBsAg) will be excluded. Participants who are positive only for hepatitis B core antibody (HBc-Ab) must undergo quantitative HBV-DNA testing and will not be excluded if the result is negative. 4. Severe renal impairment, including acute kidney injury or chronic kidney disease, or serum creatinine clearance \<60 mL/min calculated using the Cockcroft-Gault equation. 5. Current pregnancy, breastfeeding, or planned pregnancy within 48 weeks. 6. Participation in another interventional clinical trial within 28 days before enrollment or within five half-lives of the investigational drug, whichever is longer. 7. History of splenectomy. 8. History of allergic reactions to contrast agents or intravenously administered human-derived biological products. 9. Severe psychiatric symptoms that preclude cooperation with study procedures. 10. Treatment with B-cell-depleting agents, such as rituximab, within 6 months before enrollment, or failure of B-cell counts to recover to the normal range, whichever is longer. 11. Active tuberculosis. 12. Diabetes mellitus. 13. Failure to complete serum ganglioside antibody testing to exclude other diseases. 14. Any other condition that, in the investigator's judgment, makes the participant unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Confirmed Evidence of Clinical Improvement | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Percentage of participants with confirmed evidence of clinical improvement, defined according to prespecified improvements in CIDP-related clinical and functional assessments. |
| Change From Baseline in Adjusted INCAT Disability Score | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in the adjusted Inflammatory Neuropathy Cause and Treatment disability score. |
| Change From Baseline in MRC Sum Score | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in the Medical Research Council sum score. |
| Change From Baseline in I-RODS Score | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in the Inflammatory Rasch-built Overall Disability Scale score. |
| Change From Baseline in Timed Up and Go Test | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in the Timed Up and Go test. |
| Change From Baseline in Mean Grip Strength | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in mean grip strength. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Adverse Events | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Incidence of adverse events during the study period, including the time of onset, duration, clinical manifestations, severity, relationship to study treatment, and actions taken. |
| Change From Baseline in Serum IgG Levels | Baseline, Week 2, Week 4, Week 8, Week 16, and Week 24 | Change from baseline in serum immunoglobulin G levels during the study period. |
Countries
China
Outcome results
None listed