Safety and Tolerability of M254 in Healthy Volunteers and Immune Thrombocytopenic Purpura (ITP) Patients

Momenta Pharmaceuticals

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Immune Thrombocytopenic Purpura (ITP)

Treatments: Biological: Biological: M254; Biological: Intravenous immunoglobulin (IVIg); Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03866577

2018-003534-32 (EudraCT Number)

CR108979

MOM-M254-001

Details and patient eligibility

About

The purpose of this study is to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of M254 after administration of a single ascending dose and repeat doses in healthy volunteers and immune thrombocytopenic purpura (ITP) patients. The pharmacodynamics of the drug will be measured as platelet response in patients with ITP.

Full description

The Part A of the study is currently not accepting healthy volunteers as the recruitment for the part A has completed.

Enrollment: 50 patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Key Criteria for Healthy Volunteers: Subject must be between the ages of 18 and 55 years; healthy as indicated by medical history, physical examination, vital signs, clinical laboratory tests, and 12-lead electrocardiogram, and all abnormal findings are assessed as not clinically significant by the Investigator; not pregnant or breastfeeding; and no other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate.

Key Criteria for Immune Thrombocytopenic Purpura (ITP) Patients: Patient must be aged ≥18 years and diagnosed with ITP at least 3 months prior to screening, stable maintenance therapy for at least 4 weeks prior to the first study visit, not pregnant or breastfeeding, and no other clinically relevant abnormalities currently or in their history that the Investigator would deem them ineligible to participate.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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50 participants in 4 patient groups

Part A

Experimental group

Description:

Healthy volunteers will receive a single ascending dose of M254 or placebo

Treatment:

Drug: Placebo

Biological: Biological: M254

Part B

Experimental group

Description:

Immune thrombocytopenic purpura (ITP) patients will receive a single ascending dose of M254 followed by IVIg

Treatment:

Biological: Intravenous immunoglobulin (IVIg)

Biological: Biological: M254

Part C

Experimental group

Description:

ITP patients will receive a single dose of M254 or IVIg, followed by a single dose of the other drug approximately 28 days later

Treatment:

Biological: Intravenous immunoglobulin (IVIg)

Biological: Biological: M254

Part D

Experimental group

Description:

ITP patients will receive repeated doses of M254

Treatment:

Biological: Biological: M254