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

NCT07343310 | Completed Phase 1

• 1 other identifier: CIDP101-CR1-001P
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

CIDP - Chronic Inflammatory Demyelinating Polyneuropathy

Outcome Measures

Primary Outcomes (7)

• 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 Outcomes (11)

• Change From Baseline in Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score

  Day 1 (predose), Day 14, and Day 28

• Change From Baseline in Medical Research Council (MRC) Sum Score

  Day 1 (predose), Day 14, and Day 28

• Change From Baseline in Inflammatory Rasch-Built Overall Disability Scale (I-RODS)

  Day 1 (predose), Day 14, and Day 28

Study Arms (3)

KINE-101 120mg

EXPERIMENTAL

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

Drug: KINE-101

KINE-101 240 mg

EXPERIMENTAL

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

Drug: KINE-101

KINE-101 360 mg

EXPERIMENTAL

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

Drug: KINE-101

Interventions

KINE-101 DRUG

KINE-101 injection, 12.5 mg/mL, administered once intravenously on Day 1.

KINE-101 120mg; KINE-101 240 mg; KINE-101 360 mg

Eligibility Criteria

• Age: 19 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Kine Sciences Co., Ltd.: lead

Study Sites (2)

Kangbuk Samsung Hospital

Seoul, Seoul, 03181, South Korea

Location

Samsung Medical Center

Seoul, Seoul, 06351, South Korea

Location

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Hanna Park

  Kine Sciences Co., Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Sequential dose escalation in single experimental arm with cohorts receiving 120 mg, 240 mg, or 360 mg.

Study Record Dates

• First Submitted: December 10, 2025
• First Posted: January 15, 2026
• Study Start: August 27, 2024
• Primary Completion: April 14, 2025
• Study Completion: April 14, 2025
• Last Updated: January 20, 2026

Record last verified: 2026-01

Locations

KR (2)