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

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

Sponsors

Lead Sponsor: Momenta Pharmaceuticals, Inc.

Source Momenta Pharmaceuticals, Inc.
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.

Overall Status Recruiting
Start Date January 28, 2019
Completion Date June 2022
Primary Completion Date April 2022
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Number and Severity of Adverse Events (AEs) - Part A Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part A Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part A Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Vital Signs - Part A Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part A Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Electrocardiograms (ECGs) - Part A Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Electrocardiograms (ECGs) - Part A Up to approximately Day 29
Number and Severity of AEs - Part B Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part B Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part B Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Vital Signs - Part B Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part B Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in ECGs - Part B Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part B Up to approximately Day 29
Number and Severity of AEs - Part C Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part C Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part C Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in Vital Signs - Part C Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part C Up to approximately Day 29
Frequency of Clinically Significant Abnormalities in ECGs - Part C Up to approximately Day 29
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part C Up to approximately Day 29
Measurement of Changes in Platelet Counts After M254 Administration Compared to IVIg - Part C Baseline to approximately Day 29
Number and Severity of AEs - Part D Up to approximately Day 71
Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part D Up to approximately Day 71
Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part D Up to approximately Day 71
Frequency of Clinically Significant Abnormalities in Vital Signs - Part D Up to approximately Day 71
Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part D Up to approximately Day 71
Frequency of Clinically Significant Abnormalities in ECGs - Part D Up to approximately Day 71
Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part D Up to approximately Day 71
Secondary Outcome
Measure Time Frame
Maximum Plasma Concentration (Cmax) of M254 - Part A, B, and C Day 1 to Day 29
Time to Maximum Plasma Concentration (Tmax) of M254 - Part A, B, and C Day 1 to Day 29
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
Area Under the Concentration-time Curve From Zero to Infinity [AUC(0 ∞)] of M254 - Part A, B, and C Day 1 to Day 29
Volume of Distribution (Vd) of M254 - Part A, B, and C Day 1 to Day 29
Clearance of Drug (CL) M254 - Part A, B, and C Day 1 to Day 29
Mean Residence Time (MRT) of M254 - Part A, B, and C Day 1 to Day 29
Apparent Terminal-phase Half-life (t½) of M254 - Part A, B, and C Day 1 to Day 29
Cmax of M254 - Part D Day 1 to Day 71
Tmax of M254 - Part D Day 1 to Day 71
AUC(0 last) of M254 - Part D Day 1 to Day 71
AUC(0 ∞) of M254 - Part D Day 1 to Day 71
Vd of M254 - Part D Day 1 to Day 71
CL of M254 - Part D Day 1 to Day 71
MRT of M254 - Part D Day 1 to Day 71
t½ of M254 - Part D Day 1 to Day 71
Measurements of Changes in Platelet Counts After M254 Administration - Part D Baseline to approximately Day 71
Enrollment 70
Condition
Intervention

Intervention Type: Biological

Intervention Name: Biological: M254

Description: M254 administered as intravenous infusion

Intervention Type: Drug

Intervention Name: Placebo

Description: Placebo administered as intravenous infusion

Arm Group Label: Part A

Intervention Type: Biological

Intervention Name: Intravenous immunoglobulin (IVIg)

Description: IVIg administered as intravenous infusion

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 between the ages of 18 to 64 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.

Gender: All

Minimum Age: 18 Years

Maximum Age: 64 Years

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
Momenta General Queries Study Director Momenta Pharmaceuticals, Inc.
Overall Contact

Last Name: Momenta General Queries

Phone: +1 617-491-9700

Email: [email protected]

Location
Facility: Status:
Momenta Investigational Site | Los Angeles, California, 90089, United States Not yet recruiting
Momenta Investigational Site | Saint Petersburg, Florida, 33613, United States Not yet recruiting
Momenta Investigational Site | Durham, North Carolina, 27705, United States Not yet recruiting
Momenta Investigational Site | Cleveland, Ohio, 44106, United States Recruiting
Momenta Investigational Site | Cleveland, Ohio, 44195, United States Not yet recruiting
Momenta Investigational Site | Yvoir, Namur, 5530, Belgium Recruiting
Momenta Investigational Site | Le Mans Cedex 9, Pays De La Loire, 72037, France Withdrawn
Momenta Investigational Site | Aschaffenburg, Bayern, 63739, Germany Withdrawn
Momenta Investigational Site | Pecs, Baranya, 7643, Hungary Recruiting
Momenta Investigational Site | Debrecen, Hajdu-Bihar, 4032, Hungary Recruiting
Momenta Investigational Site | Kaposvar, Somogy, 7400, Hungary Recruiting
Momenta Investigational Site | San Giovanni Rotondo, Foggia, 71013, Italy Not yet recruiting
Momenta Investigational Site | Meldola, Forli-Cesena, 47014, Italy Not yet recruiting
Momenta Investigational Site | Reggio Emilia, Reggio Nella Emilia, 42123, Italy Not yet recruiting
Momenta Investigational Site | Ravenna, 48121, Italy Not yet recruiting
Momenta Investigational Site | Roma, 86100, Italy Not yet recruiting
Momenta Investigational Site | Groningen, 9713 GZ, Netherlands Recruiting
Momenta Investigational Site | Groningen, 9728 NZ, Netherlands Completed
Momenta Investigational Site | Wroclaw, Dolnoslaskie, 50-367, Poland Recruiting
Momenta Investigational Site | Lublin, Lubelskie, 20-001, Poland Recruiting
Momenta Investigational Site | Chorzow, Slaskie, 41-503, Poland Recruiting
Momenta Investigational Site | Poznan, Wielkopolskie, 61-828, Poland Recruiting
Momenta Investigational Site | Opole, 45-061, Poland Recruiting
Momenta Investigational Site | Barcelona, 8035, Spain Recruiting
Momenta Investigational Site | Burgos, 09006, Spain Recruiting
Momenta Investigational Site | Malaga, 29010, Spain Recruiting
Momenta Investigational Site | Murcia, 30008, Spain Recruiting
Momenta Investigational Site | Salamanca, 37007, Spain Recruiting
Momenta Investigational Site | Valencia, 46017, Spain Recruiting
Location Countries

Belgium

France

Germany

Hungary

Italy

Netherlands

Poland

Spain

United States

Verification Date

July 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 4
Arm Group

Label: Part A

Type: Experimental

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

Label: Part B

Type: Experimental

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

Label: Part C

Type: Experimental

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

Label: Part D

Type: Experimental

Description: ITP patients will receive repeated doses of M254

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Sequential Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Masking Description: Part A: Double (Subject, Investigator); Part B, C, and D: Open Label Investigations

Source: ClinicalTrials.gov