A First-in-Patient Clinical Trial of KINE-101 in Patients With Corticosteroid-Refractory CIDP

Kine Sciences Co., Ltd.

Status and phase: Completed; Phase 1

Conditions: CIDP - Chronic Inflammatory Demyelinating Polyneuropathy

Treatments: Drug: KINE-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT07343310

CIDP101-CR1-001P

Details and patient eligibility

About

This is a multicenter, open-label, single-dose, dose-escalation study evaluating the safety and tolerability of intravenous (IV) KINE-101 in patients with corticosteroid-refractory chronic inflammatory demyelinating polyneuropathy (CIDP). On Day 1, subjects receive a single IV dose of KINE-101 at the assigned cohort level and are discharged on Day 3, approximately 48 hours after investigational product (IP) administration, once all required in-clinic assessments have been completed. Safety assessments (including dose-limiting toxicities [DLTs], adverse events, clinical laboratory tests, vital signs, physical examinations, and 12-lead ECGs), exploratory efficacy evaluations, and PK/PD assessments are conducted through Day 28 in accordance with the schedule of assessments.

Exploratory efficacy assessments through Day 28 include changes from baseline in the following clinical measures: Inflammatory Neuropathy Cause and Treatment (INCAT) disability score, Medical Research Council (MRC) total sum score, Inflammatory Rasch-Built Overall Disability Scale (I-RODS), Timed Up-and-Go (TUG) test, mean grip strength, and the Overall Neuropathy Limitations Scale (ONLS).

Pharmacodynamic (PD) assessments include immunophenotyping of CD4+ T-cell subsets (CD4, CD25, FOXP3, CD39, CD69, CTLA-4, LAG-3, TNFR2, TIGIT, CCR5, and CXCR3); measurement of serum cytokines and immunoglobulins (IgM, IgG, IL-2, IL-6, IL-10, IL-17, IFN-γ, MCP-1, and TGF-β); evaluation of autoantibody and complement markers (antinuclear antibodies, anti-SM, anti-RNP, anti-SSA, anti-double-stranded DNA antibodies, and complement C4); and additional laboratory parameters related to systemic inflammation.

Dose escalation follows a standard 3+3 design based on review of safety, including DLTs, in the preceding cohort. Three KINE-101 dose cohorts are planned: Cohort 1 (120 mg), Cohort 2 (240 mg), and Cohort 3 (360 mg). If safety and tolerability are deemed acceptable in a given cohort, enrollment proceeds sequentially to the next higher dose level.

Enrollment: 9 patients

Sex: All

Ages: 19+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Adults aged ≥19 years at informed consent.

• Diagnosed with CIDP and refractory to corticosteroid treatment for ≥3 months prior to enrollment, or corticosteroid treatment deemed inappropriate or cannot be continued for safety reasons.

• Meets EAN/PNS 2021 criteria for typical CIDP:

  • Progressive or relapsing symmetrical motor weakness in arms and legs with sensory involvement in ≥2 limbs
  • Symptom duration ≥8 weeks
  • Reduced or absent tendon reflexes in all extremities

• INCAT disability score ≥2 at screening (score of 2 must result solely from leg disability).

• CIDP Disease Activity Status (CDAS) score ≥3 at screening.

• Received IVIg ≥2 months prior to IP administration.

• If the subject is of childbearing potential, agrees to use highly effective contraception for ≥28 days after IP administration.

• Adequate venous access for IV administration and blood sampling.

• Willing and able to comply with all study procedures.

Exclusion criteria

• Polyneuropathy due to other causes (e.g., MMN, MGUS with anti-MAG antibodies, hereditary neuropathies, POEMS syndrome, diabetic or systemic disease-related neuropathy, drug/toxin-induced neuropathy).
• History of myelopathy or confirmed central demyelination.
• Known allergy or hypersensitivity to the investigational product or its excipients.
• Uncontrolled severe hepatic disease, CNS disorders, alcoholism, substance abuse, or psychiatric disorders.
• Other medical conditions that better explain symptoms (e.g., stroke, CNS trauma, connective tissue disease).
• Malignancy within 5 years, except adequately treated low-risk cancers.
• Moderate to severe heart failure or severe cardiovascular disease (e.g., MI, ischemic stroke).
• Moderate to severe substance or alcohol use disorder within 1 year.
• Positive pregnancy test or planning pregnancy, breastfeeding, or gamete donation within 28 days post-IP.
• Use of systemic immunosuppressants or immunostimulants within 5 half-lives prior to IP administration.
• Plasma exchange within 8 weeks prior to IP administration.
• Chronic infections or expected need for anti-infective treatment during the study.
• Active hepatitis B or C infection or HIV positive.
• Abnormal labs at screening: AST/ALT >3×ULN, Hb <9 g/dL, ANC <1,500/μL, Platelets <100×10³/μL.
• Major surgery within 3 months or planned during study participation.
• Investigator deems subject unsuitable for any reason.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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9 participants in 3 patient groups

KINE-101 120mg

Experimental group

Description:

Subjects received a single intravenous dose of KINE-101 120 mg.

Treatment:

Drug: KINE-101

KINE-101 240 mg

Experimental group

Description:

Subjects received a single intravenous dose of KINE-101 240 mg.

Treatment:

Drug: KINE-101

KINE-101 360 mg

Experimental group

Description:

Subjects received a single intravenous dose of KINE-101 360 mg.

Treatment:

Drug: KINE-101