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

Immune Thrombocytopenic Purpura (ITP) Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03866577
• Other study ID #: CR108979
• Secondary ID: 2018-003534-32MO
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: December 21, 2018
• Est. completion date: June 9, 2021

Study information

• Verified date: June 2023
• Source: Momenta Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 50
• Est. completion date: June 9, 2021
• Est. primary completion date: June 9, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenic Purpura (ITP)
• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic

Intervention

Biological:
• Biological: M254
M254 administered as intravenous infusion

Drug:
• Placebo
Placebo administered as intravenous infusion

Biological:
• Intravenous immunoglobulin (IVIg)
IVIg administered as intravenous infusion

Locations

Country	Name	City	State
Belgium	Ucl de Mont-Godinne	Yvoir
Hungary	Debreceni Egyetem Klinikai Kozpont	Debrecen
Hungary	Somogy Megyei Kaposi Mor Oktato Korhaz	Kaposvar
Hungary	Pecsi Tudomanyegyetem	Pecs
Italy	Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori	Meldola
Italy	Azienda Unita Sanitaria Locale di Ravenna	Ravenna
Italy	Arcispedale Santa Maria Nuova - IRCCS	Reggio Emilia
Italy	Policlinico Universitario Agostino Gemelli	Roma
Italy	Ospedale 'Casa Sollievo della Sofferenza' - U.O. Ematologia-	San Giovanni Rotondo
Netherlands	PRA Health Sciences	Groningen
Netherlands	Universitair Medisch Centrum Groningen	Groningen
Poland	Silesian Healthy Blood Clinic	Chorzow
Poland	Samodzielny Publiczny Szpital Kliniczny Nr 1	Lublin
Poland	Szpital Wojewodzki w Opolu	Opole
Poland	Samodzielny Publiczny Zaklad Opieki Zdrowotnej MSW w Poznaniu im. prof. Ludwika Bierkowskiego	Poznan
Poland	Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu	Wroclaw
Spain	Hosp. Univ. Vall D Hebron	Barcelona
Spain	Hosp. Univ. de Burgos	Burgos
Spain	Hosp. Regional. Carlos Haya	Malaga
Spain	Hosp. Gral. Univ. J.M. Morales Meseguer	Murcia
Spain	Hosp. Clinico Univ. de Salamanca	Salamanca
Spain	Hosp. Univ. Dr. Peset	Valencia
United States	Taussig Cancer Insititute - Cleveland Clinic	Cleveland	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	University of Southern California	Los Angeles	California
United States	Lakes Research	Miami Lakes	Florida
United States	University of South Florida	Saint Petersburg	Florida
United States	Oncology Institute of Hope and Innovation	Whittier	California

Sponsors (1)

Lead Sponsor	Collaborator
Momenta Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Belgium,  Hungary,  Italy,  Netherlands,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number and Severity of Adverse Events (AEs) - Part A		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part A		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part A		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Vital Signs - Part A		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part A		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Electrocardiograms (ECGs) - Part A		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Electrocardiograms (ECGs) - Part A		Up to approximately Day 29
Primary	Number and Severity of AEs - Part B		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part B		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part B		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Vital Signs - Part B		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part B		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in ECGs - Part B		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part B		Up to approximately Day 29
Primary	Number and Severity of AEs - Part C		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part C		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part C		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in Vital Signs - Part C		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part C		Up to approximately Day 29
Primary	Frequency of Clinically Significant Abnormalities in ECGs - Part C		Up to approximately Day 29
Primary	Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part C		Up to approximately Day 29
Primary	Measurement of Changes in Platelet Counts After M254 Administration Compared to IVIg - Part C		Baseline to approximately Day 29
Primary	Number and Severity of AEs - Part D		Up to approximately Day 71
Primary	Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part D		Up to approximately Day 71
Primary	Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part D		Up to approximately Day 71
Primary	Frequency of Clinically Significant Abnormalities in Vital Signs - Part D		Up to approximately Day 71
Primary	Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part D		Up to approximately Day 71
Primary	Frequency of Clinically Significant Abnormalities in ECGs - Part D		Up to approximately Day 71
Primary	Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part D		Up to approximately Day 71
Secondary	Maximum Plasma Concentration (Cmax) of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Time to Maximum Plasma Concentration (Tmax) of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Area Under the Concentration-time Curve From Zero to Time of Last Measurable Concentration Area [AUC(0 last)] of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Area Under the Concentration-time Curve From Zero to Infinity [AUC(0 8)] of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Volume of Distribution (Vd) of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Clearance of Drug (CL) M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Mean Residence Time (MRT) of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Apparent Terminal-phase Half-life (t½) of M254 - Part A, B, and C		Day 1 to Day 29
Secondary	Cmax of M254 - Part D		Day 1 to Day 71
Secondary	Tmax of M254 - Part D		Day 1 to Day 71
Secondary	AUC(0 last) of M254 - Part D		Day 1 to Day 71
Secondary	AUC(0 8) of M254 - Part D		Day 1 to Day 71
Secondary	Vd of M254 - Part D		Day 1 to Day 71
Secondary	CL of M254 - Part D		Day 1 to Day 71
Secondary	MRT of M254 - Part D		Day 1 to Day 71
Secondary	t½ of M254 - Part D		Day 1 to Day 71
Secondary	Measurements of Changes in Platelet Counts After M254 Administration - Part D		Baseline to approximately Day 71