Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study in Healthy Male and Female Subjects and Safety, Tolerability, Pharmacokinetics, and Pilot Efficacy Biomarkers in Subjects with Cold Agglutinin Disease

Phase 1

Recruiting

• Conditions: First in Man Study to Evaluate Initial Safety
• Interventions: Drug: GL-0719; Drug: Placebo

• Registration Number: NCT05318534
• Lead Sponsor: Gliknik Inc.

Brief Summary

The purpose of this first-in-human (FIH) study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of GL-0719 following single intravenous (IV) and subcutaneous injection (SC) doses in healthy adult male and female subjects.

In addition, safety, tolerability, PK, and pilot efficacy biomarkers will be evaluated in subjects with cold agglutinin disease (CAD).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-28
• Study First Submitted QC: 2022-04-07
• Study Start: 2022-04-08
• Study First Posted: 2022-04-08
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-29
• Last Update Posted: 2025-01-31
• Primary Completion: 2025-07-01
• Study Completion: 2025-07-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
GL-0719	GL-0719	Dose level cohorts randomized in a 3:1 ratio to GL-0719 or placebo treatment, respectively. The study will comprise a single-dose, sequential-group design. Single Ascending IV Dose Cohorts * Cohort 1: 4 subjects * Cohort 2: 8 subjects * Cohort 3: 8 subjects * Cohort 4: 8 subjects * Cohort 5: 8 subjects Subcutaneous Injection Cohort * Cohort 6: 8 subjects * Cohort 7: 8 subjects * Cohort 8 (Patient Arm): Up to 6 subjects with Cold Agglutinin Disease (CAD); Placebo is not applicable. * Cohort 9 (Patient Arm): Up to 12 subjects with Cold Agglutinin Disease (CAD); Placebo is not applicable.
Placebo	Placebo	Dose level cohorts randomized in a 3:1 ratio to GL-0719 or placebo treatment, respectively. The study will comprise a single-dose, sequential-group design. Single Ascending IV Dose Cohorts * Cohort 1: 4 subjects * Cohort 2: 8 subjects * Cohort 3: 8 subjects * Cohort 4: 8 subjects * Cohort 5: 8 subjects Subcutaneous Injection Cohort * Cohort 6: 8 subjects * Cohort 7: 8 subjects

Primary Outcome Measures

Name	Time	Method
Incidence of abnormal clinical laboratory findings in 12-lead ECG parameters, vital signs, physical examination and measurement of cytokines	Screening (Days -42 to -15) to Follow-up (Day 31±2)
Incidence and severity of adverse events (AEs)	Day 1 to Follow-up (Day 31±2)
Incidence of laboratory abnormalities, based on hematology, clinical chemistry, and urinalysis test results	Screening (Days -42 to -15) to Follow-up (Day 31±2)

Secondary Outcome Measures

Name	Time	Method
Time of the maximum observed concentration (tmax)	Day 1 to Follow-up (Day 31±2)
Maximum Observed Concentration (Cmax)	Day 1 to Follow-up (Day 31±2)
Apparent terminal elimination half-life (t1/2)	Day 1 to Follow-up (Day 31±2)
For Cohorts 1 to 7: The degree of complement classical pathway inhibition in study subjects over time as evaluated by the MicroVue CH50 Eq EIA assay	Day 1 to Follow-up (Day 31±2)
Incidence of anti-drug antibodies	Day -1, Day 15±1 and Follow-up (Day 31±2)
Area Under the Concentration time Curve from Time 0 Extrapolated to Infinity (AUC0-∞)	Day 1 to Follow-up (Day 31±2)
Area Under the Concentration time Curve from Time 0 to the Time of the Last (AUC0-tlast)	Day 1 to Follow-up (Day 31±2)

Trial Locations

Locations (1)

Fortrea Clinical Research Unit Ltd; 🇬🇧 Leeds, United Kingdom