Clinical trial NCT03866577: Safety and Tolerability of M254 in Healthy Volunteers and Immune Thrombocytopenic Purpura (ITP) Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Immune Thrombocytopenic Purpura (ITP)
Keywords: Immune thrombocytopenic purpura
Healthy subjects
M254
Intravenous immunoglobulin (IVIg)
Lead sponsor: Momenta Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2019-03-07
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.

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

Eligibility 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.

Primary outcomes:
measure: Parts A, B, and C: Number of Participants With Treatment-emergent Adverse Events (TEAEs) by Severity
timeFrame: From Day 1 up to Day 29
measure: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Clinical Laboratory Values
timeFrame: From Day 1 up to Day 29
measure: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Vital Signs
timeFrame: From Day 1 up to Day 29
measure: Parts A, B, and C: Number of Participants With Clinically Significant Abnormalities in Electrocardiograms (ECGs)
timeFrame: From Day 1 up to Day 29
measure: Part C: Maximum Observed Response of M254 (Rmax) on Platelet Count
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Part C: Change From Baseline in Rmax of M254 in Platelet Count
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 14 (AUEC[0-Day 14]) of M254
timeFrame: Predose (baseline) up to Day 14 post dose
measure: Part C: Area Under Effect Curve of the Change From Baseline in Platelet Count From Day 0 to Day 28 (AUEC[0-Day 28]) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
Secondary outcomes:
measure: Part C: Number of Participants With Overall Platelet Response After M254 Administration Compared to IVIg
timeFrame: Up to Day 29
measure: Parts A, B, and C: Maximum Observed Plasma Concentration (Cmax) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Time to Reach Maximum Observed Plasma Concentration (Tmax) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Infinite Time (AUC[0-Infinity]) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Area Under the Plasma Concentration-time Curve From Time Zero to Time of Last Quantifiable Concentration (AUC[0-Last]) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Apparent Terminal Phase Half-life (t1/2) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Volume of Distribution (Vz) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Clearance (CL) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Mean Residence Time (MRT) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
measure: Parts A, B, and C: Percentage of the Estimated Part for the Calculation of AUC(0-infinity) (%AUCextra) of M254
timeFrame: Predose (baseline) up to Day 29 post dose
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: University of Southern California
state: California
city: Whittier
country: United States
facility: Oncology Institute of Hope and Innovation
state: California
city: Miami Lakes
country: United States
facility: Lakes Research
state: Florida
city: Saint Petersburg
country: United States
facility: University of South Florida
state: Florida
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Taussig Cancer Insititute Cleveland Clinic
state: Ohio
city: Yvoir
country: Belgium
facility: Ucl de Mont-Godinne
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Kaposvar
country: Hungary
facility: Somogy Megyei Kaposi Mor Oktato Korhaz
city: Pecs
country: Hungary
facility: Pecsi Tudomanyegyetem
city: Meldola
country: Italy
facility: Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
city: Ravenna
country: Italy
facility: Azienda Unita Sanitaria Locale di Ravenna
city: Reggio Emilia
country: Italy
facility: Arcispedale Santa Maria Nuova - IRCCS
city: Roma
country: Italy
facility: Policlinico Universitario Agostino Gemelli
city: San Giovanni Rotondo
country: Italy
facility: Ospedale 'Casa Sollievo della Sofferenza' - U.O. Ematologia-
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: Groningen
country: Netherlands
facility: PRA Health Sciences
city: Chorzow
country: Poland
facility: Silesian Healthy Blood Clinic
city: Lublin
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny Nr 1
city: Opole
country: Poland
facility: Szpital Wojewodzki w Opolu
city: Poznan
country: Poland
facility: Samodzielny Publiczny Zaklad Opieki Zdrowotnej MSW w Poznaniu im prof Ludwika Bierkowskiego
city: Wroclaw
country: Poland
facility: Uniwersytecki Szpital Kliniczny im Jana Mikulicza Radeckiego we Wroclawiu
city: Barcelona
country: Spain
facility: Hosp Univ Vall D Hebron
city: Burgos
country: Spain
facility: Hosp. Univ. de Burgos
city: Malaga
country: Spain
facility: Hosp. Regional. Carlos Haya
city: Murcia
country: Spain
facility: Hosp. Gral. Univ. J.M. Morales Meseguer
city: Salamanca
country: Spain
facility: Hosp Clinico Univ de Salamanca
city: Valencia
country: Spain
facility: Hosp. Univ. Dr. Peset
Study officials:
affiliation: Momenta Pharmaceuticals, Inc.
name: Momenta General Queries
role: STUDY_DIRECTOR
References: