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

Primary Purpose

Immune Thrombocytopenic Purpura (ITP)

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Biological: M254
Placebo
Intravenous immunoglobulin (IVIg)
Sponsored by
Momenta Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Immune Thrombocytopenic Purpura (ITP) focused on measuring Immune thrombocytopenic purpura, Healthy subjects, M254, Intravenous immunoglobulin (IVIg)

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

  • University of Southern California
  • Oncology Institute of Hope and Innovation
  • Lakes Research
  • University of South Florida
  • Duke University Medical Center
  • Taussig Cancer Insititute - Cleveland Clinic
  • Ucl de Mont-Godinne
  • Debreceni Egyetem Klinikai Kozpont
  • Somogy Megyei Kaposi Mor Oktato Korhaz
  • Pecsi Tudomanyegyetem
  • Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
  • Azienda Unita Sanitaria Locale di Ravenna
  • Arcispedale Santa Maria Nuova - IRCCS
  • Policlinico Universitario Agostino Gemelli
  • Ospedale 'Casa Sollievo della Sofferenza' - U.O. Ematologia-
  • Universitair Medisch Centrum Groningen
  • PRA Health Sciences
  • Silesian Healthy Blood Clinic
  • Samodzielny Publiczny Szpital Kliniczny Nr 1
  • Szpital Wojewodzki w Opolu
  • Samodzielny Publiczny Zakład Opieki Zdrowotnej MSW w Poznaniu im. prof. Ludwika Bierkowskiego
  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
  • Hosp. Univ. Vall D Hebron
  • Hosp. Univ. de Burgos
  • Hosp. Regional. Carlos Haya
  • Hosp. Gral. Univ. J.M. Morales Meseguer
  • Hosp. Clinico Univ. de Salamanca
  • Hosp. Univ. Dr. Peset

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Part A

Part B

Part C

Part D

Arm Description

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

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

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

ITP patients will receive repeated doses of M254

Outcomes

Primary Outcome Measures

Number and Severity of Adverse Events (AEs) - Part A
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part A
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part A
Frequency of Clinically Significant Abnormalities in Vital Signs - Part A
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part A
Frequency of Clinically Significant Abnormalities in Electrocardiograms (ECGs) - Part A
Shift From Baseline in Clinically Significant Abnormalities for Electrocardiograms (ECGs) - Part A
Number and Severity of AEs - Part B
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part B
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part B
Frequency of Clinically Significant Abnormalities in Vital Signs - Part B
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part B
Frequency of Clinically Significant Abnormalities in ECGs - Part B
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part B
Number and Severity of AEs - Part C
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part C
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part C
Frequency of Clinically Significant Abnormalities in Vital Signs - Part C
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part C
Frequency of Clinically Significant Abnormalities in ECGs - Part C
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part C
Measurement of Changes in Platelet Counts After M254 Administration Compared to IVIg - Part C
Number and Severity of AEs - Part D
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part D
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part D
Frequency of Clinically Significant Abnormalities in Vital Signs - Part D
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part D
Frequency of Clinically Significant Abnormalities in ECGs - Part D
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part D

Secondary Outcome Measures

Maximum Plasma Concentration (Cmax) of M254 - Part A, B, and C
Time to Maximum Plasma Concentration (Tmax) of M254 - Part A, B, and C
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
Area Under the Concentration-time Curve From Zero to Infinity [AUC(0 ∞)] of M254 - Part A, B, and C
Volume of Distribution (Vd) of M254 - Part A, B, and C
Clearance of Drug (CL) M254 - Part A, B, and C
Mean Residence Time (MRT) of M254 - Part A, B, and C
Apparent Terminal-phase Half-life (t½) of M254 - Part A, B, and C
Cmax of M254 - Part D
Tmax of M254 - Part D
AUC(0 last) of M254 - Part D
AUC(0 ∞) of M254 - Part D
Vd of M254 - Part D
CL of M254 - Part D
MRT of M254 - Part D
t½ of M254 - Part D
Measurements of Changes in Platelet Counts After M254 Administration - Part D

Full Information

First Posted
February 12, 2019
Last Updated
June 30, 2023
Sponsor
Momenta Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03866577
Brief Title
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

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Terminated
Why Stopped
Study was terminated based on Sponsor decision.
Study Start Date
December 21, 2018 (Actual)
Primary Completion Date
June 9, 2021 (Actual)
Study Completion Date
June 9, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Momenta Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenic Purpura (ITP)
Keywords
Immune thrombocytopenic purpura, Healthy subjects, M254, Intravenous immunoglobulin (IVIg)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Masking Description
Part A: Double (Subject, Investigator); Part B, C, and D: Open Label Investigations
Allocation
Randomized
Enrollment
50 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Part A
Arm Type
Experimental
Arm Description
Healthy volunteers will receive a single ascending dose of M254 or placebo
Arm Title
Part B
Arm Type
Experimental
Arm Description
Immune thrombocytopenic purpura (ITP) patients will receive a single ascending dose of M254 followed by IVIg
Arm Title
Part C
Arm Type
Experimental
Arm 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
Arm Title
Part D
Arm Type
Experimental
Arm Description
ITP patients will receive repeated doses of M254
Intervention Type
Biological
Intervention Name(s)
Biological: M254
Intervention Description
M254 administered as intravenous infusion
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo administered as intravenous infusion
Intervention Type
Biological
Intervention Name(s)
Intravenous immunoglobulin (IVIg)
Intervention Description
IVIg administered as intravenous infusion
Primary Outcome Measure Information:
Title
Number and Severity of Adverse Events (AEs) - Part A
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part A
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part A
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Vital Signs - Part A
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part A
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Electrocardiograms (ECGs) - Part A
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Electrocardiograms (ECGs) - Part A
Time Frame
Up to approximately Day 29
Title
Number and Severity of AEs - Part B
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part B
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part B
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Vital Signs - Part B
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part B
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in ECGs - Part B
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part B
Time Frame
Up to approximately Day 29
Title
Number and Severity of AEs - Part C
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part C
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part C
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in Vital Signs - Part C
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part C
Time Frame
Up to approximately Day 29
Title
Frequency of Clinically Significant Abnormalities in ECGs - Part C
Time Frame
Up to approximately Day 29
Title
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part C
Time Frame
Up to approximately Day 29
Title
Measurement of Changes in Platelet Counts After M254 Administration Compared to IVIg - Part C
Time Frame
Baseline to approximately Day 29
Title
Number and Severity of AEs - Part D
Time Frame
Up to approximately Day 71
Title
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part D
Time Frame
Up to approximately Day 71
Title
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part D
Time Frame
Up to approximately Day 71
Title
Frequency of Clinically Significant Abnormalities in Vital Signs - Part D
Time Frame
Up to approximately Day 71
Title
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part D
Time Frame
Up to approximately Day 71
Title
Frequency of Clinically Significant Abnormalities in ECGs - Part D
Time Frame
Up to approximately Day 71
Title
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part D
Time Frame
Up to approximately Day 71
Secondary Outcome Measure Information:
Title
Maximum Plasma Concentration (Cmax) of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Time to Maximum Plasma Concentration (Tmax) of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
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
Time Frame
Day 1 to Day 29
Title
Area Under the Concentration-time Curve From Zero to Infinity [AUC(0 ∞)] of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Volume of Distribution (Vd) of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Clearance of Drug (CL) M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Mean Residence Time (MRT) of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Apparent Terminal-phase Half-life (t½) of M254 - Part A, B, and C
Time Frame
Day 1 to Day 29
Title
Cmax of M254 - Part D
Time Frame
Day 1 to Day 71
Title
Tmax of M254 - Part D
Time Frame
Day 1 to Day 71
Title
AUC(0 last) of M254 - Part D
Time Frame
Day 1 to Day 71
Title
AUC(0 ∞) of M254 - Part D
Time Frame
Day 1 to Day 71
Title
Vd of M254 - Part D
Time Frame
Day 1 to Day 71
Title
CL of M254 - Part D
Time Frame
Day 1 to Day 71
Title
MRT of M254 - Part D
Time Frame
Day 1 to Day 71
Title
t½ of M254 - Part D
Time Frame
Day 1 to Day 71
Title
Measurements of Changes in Platelet Counts After M254 Administration - Part D
Time Frame
Baseline to approximately Day 71

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Momenta General Queries
Organizational Affiliation
Momenta Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of Southern California
City
Los Angeles
State/Province
California
ZIP/Postal Code
90089
Country
United States
Facility Name
Oncology Institute of Hope and Innovation
City
Whittier
State/Province
California
ZIP/Postal Code
90603
Country
United States
Facility Name
Lakes Research
City
Miami Lakes
State/Province
Florida
ZIP/Postal Code
33014
Country
United States
Facility Name
University of South Florida
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Taussig Cancer Insititute - Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Ucl de Mont-Godinne
City
Yvoir
ZIP/Postal Code
5530
Country
Belgium
Facility Name
Debreceni Egyetem Klinikai Kozpont
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
Somogy Megyei Kaposi Mor Oktato Korhaz
City
Kaposvar
ZIP/Postal Code
7400
Country
Hungary
Facility Name
Pecsi Tudomanyegyetem
City
Pecs
ZIP/Postal Code
7624
Country
Hungary
Facility Name
Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
City
Meldola
ZIP/Postal Code
47014
Country
Italy
Facility Name
Azienda Unita Sanitaria Locale di Ravenna
City
Ravenna
ZIP/Postal Code
48100
Country
Italy
Facility Name
Arcispedale Santa Maria Nuova - IRCCS
City
Reggio Emilia
ZIP/Postal Code
42123
Country
Italy
Facility Name
Policlinico Universitario Agostino Gemelli
City
Roma
ZIP/Postal Code
168
Country
Italy
Facility Name
Ospedale 'Casa Sollievo della Sofferenza' - U.O. Ematologia-
City
San Giovanni Rotondo
ZIP/Postal Code
71013
Country
Italy
Facility Name
Universitair Medisch Centrum Groningen
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
PRA Health Sciences
City
Groningen
ZIP/Postal Code
NZ 9728
Country
Netherlands
Facility Name
Silesian Healthy Blood Clinic
City
Chorzow
ZIP/Postal Code
41-503
Country
Poland
Facility Name
Samodzielny Publiczny Szpital Kliniczny Nr 1
City
Lublin
ZIP/Postal Code
20-081
Country
Poland
Facility Name
Szpital Wojewodzki w Opolu
City
Opole
ZIP/Postal Code
45-061
Country
Poland
Facility Name
Samodzielny Publiczny Zakład Opieki Zdrowotnej MSW w Poznaniu im. prof. Ludwika Bierkowskiego
City
Poznan
ZIP/Postal Code
60-631
Country
Poland
Facility Name
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
City
Wroclaw
ZIP/Postal Code
50-367
Country
Poland
Facility Name
Hosp. Univ. Vall D Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hosp. Univ. de Burgos
City
Burgos
ZIP/Postal Code
09003
Country
Spain
Facility Name
Hosp. Regional. Carlos Haya
City
Malaga
ZIP/Postal Code
29010
Country
Spain
Facility Name
Hosp. Gral. Univ. J.M. Morales Meseguer
City
Murcia
ZIP/Postal Code
30008
Country
Spain
Facility Name
Hosp. Clinico Univ. de Salamanca
City
Salamanca
ZIP/Postal Code
37007
Country
Spain
Facility Name
Hosp. Univ. Dr. Peset
City
Valencia
ZIP/Postal Code
46017
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
No

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

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