A 4-part Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 in healthy volunteers and in patients with immune thrombocytopenic purpura.

A 4-part Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 in healthy volunteers and in patients with immune thrombocytopenic purpura. - Safety and tolerability of M254 in HV and ITP patients.

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON49624

Enrollment

Registered

2018-12-05

Start date

2019-01-04

Completion date

Unknown

Last updated

2025-09-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

immune thrombocytopenia

Interventions

Part A: Group 1

Day 1

M254 3 mg / kg or placebo

once

Intravenous infusion Group 2

M254 10 mg / kg or placebo, once, intravenous infusion Group 3

M254 30 mg / kg or placebo, once, intravenous infusion Group 4

M254 60 mg / kg or placebo, once, intravenous infusion Group 5

M254 120 mg / kg or placebo, once

intravenous infusion Group 6

M254 250 mg / kg or placebo, once

intravenous infusion Group 7c

M254 NTB mg / kg or placebo, once

intravenous infusion Group 8c

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: healthy male or female subjects 18 - 55 years of age Weight: body weight must be within a range that allows for the planned M254 infusion time to be completed in * 4 hours. BMI 18.5 - 30 kilograms/meter2 Patients: male or female diagnosed with ITP for at least 3 months 18 years and older

Exclusion criteria

Exclusion criteria: Suffering from hepatitis B, hepatitis C, cancer or HIV/AIDS. In case of participation in another drug study within 90 days before the start of this study or being a blood donor within 30 days from the start of the study. In case of donating more than 1.5 liters of blood in the 10 months prior the start of this study.

Design outcomes

Primary

Measure	Time frame
Part A: To assess the safety and tolerability of a single ascending dose of intravenous administration of M254 in healthy volunteers. Part B: To assess the safety and tolerability of a single intravenous administration of M254 in immune thrombocytopenia patients compared to 1000 mg/kg intravenous Privigen Part C: To assess the safety of a single intravenous administration of M254 compared to 1000 mg/kg of Privigen To characterize the PD of single intravenous administration of M254 compared to 1000 mg/kg Privigen Part D: To assess the safety and tolerability of repeated intravenous administration of M254 in ITP patients.	—

Measure

Time frame

Part A: To assess the safety and tolerability of a single ascending dose of intravenous administration of M254 in healthy volunteers. Part B: To assess the safety and tolerability of a single intravenous administration of M254 in immune thrombocytopenia patients compared to 1000 mg/kg intravenous Privigen Part C: To assess the safety of a single intravenous administration of M254 compared to 1000 mg/kg of Privigen To characterize the PD of single intravenous administration of M254 compared to 1000 mg/kg Privigen Part D: To assess the safety and tolerability of repeated intravenous administration of M254 in ITP patients.

—

Secondary

Measure	Time frame
Part A: To characterize the pharmacokinetics (PK) of a single intravenous administration of M254 at different doses in healthy volunteers Part B: To characterize the PK of a single intravenous administration of M254 at different doses in ITP patients Part C: To characterize the PK of a single intravenous administration of M254 at different doses. Part D: To characterize the PK of repeated intravenous doses of M254 in ITP patients and to assess PD of repeated intravenous doses of M254 in ITP patients	—

Measure

Time frame

Part A: To characterize the pharmacokinetics (PK) of a single intravenous administration of M254 at different doses in healthy volunteers Part B: To characterize the PK of a single intravenous administration of M254 at different doses in ITP patients Part C: To characterize the PK of a single intravenous administration of M254 at different doses. Part D: To characterize the PK of repeated intravenous doses of M254 in ITP patients and to assess PD of repeated intravenous doses of M254 in ITP patients

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)