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

Multi-Center, Open-label, Single Dosing, Dose-Ascending, Phase 1 Study to Evaluate the Safety and Tolerability of KINE-101 in Patients With CIDP (Chronic Inflammatory Demyelinating Polyneuropathy) Refractory to Corticosteroid Treatment

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.

Study Overview

• Status: Completed
• Conditions: CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
• Intervention / Treatment: Drug: KINE-101

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 1

Contacts and Locations

Study Locations

• South Korea
  • Seoul
    • Seoul, Seoul, South Korea, 06351
      • Samsung Medical Center
    • Seoul, Seoul, South Korea, 03181
      • Kangbuk Samsung Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: KINE-101 120mg; Subjects received a single intravenous dose of KINE-101 120 mg.	Drug: KINE-101; KINE-101 injection, 12.5 mg/mL, administered once intravenously on Day 1.
Experimental: KINE-101 240 mg; Subjects received a single intravenous dose of KINE-101 240 mg.	Drug: KINE-101; KINE-101 injection, 12.5 mg/mL, administered once intravenously on Day 1.
Experimental: KINE-101 360 mg; Subjects received a single intravenous dose of KINE-101 360 mg.	Drug: KINE-101; KINE-101 injection, 12.5 mg/mL, administered once intravenously on Day 1.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of Adverse Events (AEs)	Incidence, severity, and relationship of treatment-emergent adverse events following a single intravenous dose of KINE-101.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Hematology	Count of participants with clinically significant abnormal hematology findings (e.g., white blood cell count, hemoglobin, platelets, absolute neutrophil count), as defined per protocol.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Clinical Chemistry	Count of participants with clinically significant abnormal clinical chemistry findings (e.g., ALT, AST, creatinine), as defined per protocol.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Urinalysis	Count of participants with clinically significant abnormal urinalysis findings (e.g., protein/albumin, glucose, pH), as defined per protocol.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Vital Signs	Count of participants with clinically significant abnormal vital sign findings (e.g., systolic or diastolic blood pressure, pulse rate, body temperature, respiratory rate), as defined per protocol.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Physical Examination	Count of participants with clinically significant abnormal physical examination findings, as defined per protocol.	Day 1 to Day 28
Number of Participants with Clinically Significant Abnormal Electrocardiogram	Count of participants with clinically significant abnormal electrocardiogram findings (e.g., PR interval, QRS duration, QT interval), as defined per protocol.	Day 1 to Day 28

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score	Change from baseline in the INCAT disability score (range 0-10; higher scores indicate worse disability).	Day 1 (predose), Day 14, and Day 28
Change From Baseline in Medical Research Council (MRC) Sum Score	Change from baseline in the MRC sum score (range 0-60; higher scores indicate better muscle strength).	Day 1 (predose), Day 14, and Day 28
Change From Baseline in Inflammatory Rasch-Built Overall Disability Scale (I-RODS)	Change from baseline in the I-RODS score (range 0-48; higher scores indicate better function).	Day 1 (predose), Day 14, and Day 28
Change From Baseline in Timed Up-and-Go (TUG) Test	Change from baseline in the Timed Up-and-Go test, measured in seconds (lower values indicate better performance).	Day 1 (predose), Day 14, and Day 28
Change From Baseline in Mean Grip Strength	Change from baseline in mean grip strength (higher values indicate greater muscle strength).	Day 1 (predose), Day 14, and Day 28
Change From Baseline in Overall Neuropathy Limitations Scale (ONLS)	Change from baseline in the ONLS score (range 0-12; higher scores indicate worse disability).	Day 1 (predose), Day 14, and Day 28
Peak Plasma Concentration of KINE-101 (Cmax)	Maximum observed plasma concentration of KINE-101.	Day 1
Area Under the Plasma Concentration-Time Curve of KINE-101 (AUC)	Area under the plasma concentration-time curve of KINE-101 (AUClast and AUCinf).	Day 1
Change From Baseline in Immunophenotyping markers following KINE-101 administration	The change from baseline in immunophenotyping markers following intravenous administration of KINE-101 including CD4, CD25, FoxP3, CD39, CD69, CTLA4, LAG-3, TNFR2, TIGIT, CCR5, and CXCR3.	Day 1 to Day 28
Change From Baseline in Serum biomarkers following KINE-101 administration	The change from baseline in serum biomarkers following intravenous administration of KINE-101 including IgG, IgM, IL-2, IL-6, IL-10, IL-17, IFN-gamma, MCP-1, and TGF-beta.	Day 1 to Day 28
Changes From Baseline in Autoantibodies and Inflammatory Laboratory markers following KINE-101 administration	Changes from baseline in serum autoantibodies and inflammatory laboratory markers.	Day 1 to Day 28

Collaborators and Investigators

• Sponsor: Kine Sciences Co., Ltd.
• Investigators: Study Director: Hanna Park, Kine Sciences Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): August 27, 2024
• Primary Completion (Actual): April 14, 2025
• Study Completion (Actual): April 14, 2025

Study Registration Dates

• First Submitted: December 10, 2025
• First Submitted That Met QC Criteria: January 6, 2026
• First Posted (Estimated): January 15, 2026

Study Record Updates

• Last Update Posted (Actual): January 20, 2026
• Last Update Submitted That Met QC Criteria: January 15, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Polyneuropathies; Polyradiculoneuropathy; Pathological Conditions, Signs and Symptoms; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
• Other Study ID Numbers: CIDP101-CR1-001P

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No