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

• ClinicalTrials.gov Identifier: NCT03866577
• Recruitment Status: Recruiting
• First Posted: March 7, 2019
• Last Update Posted: December 16, 2020
• Sponsor: Momenta Pharmaceuticals, Inc.
• Information provided by (Responsible Party): Momenta Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: February 12, 2019
• First Posted Date: March 7, 2019
• Last Update Posted Date: December 16, 2020
• Actual Study Start Date: January 28, 2019
• Estimated Primary Completion Date: April 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 5, 2019)

• Number and Severity of Adverse Events (AEs) - Part A [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part A [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part A [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Vital Signs - Part A [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part A [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Electrocardiograms (ECGs) - Part A [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Electrocardiograms (ECGs) - Part A [ Time Frame: Up to approximately Day 29 ]
• Number and Severity of AEs - Part B [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part B [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part B [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Vital Signs - Part B [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part B [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in ECGs - Part B [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part B [ Time Frame: Up to approximately Day 29 ]
• Number and Severity of AEs - Part C [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part C [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part C [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in Vital Signs - Part C [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part C [ Time Frame: Up to approximately Day 29 ]
• Frequency of Clinically Significant Abnormalities in ECGs - Part C [ Time Frame: Up to approximately Day 29 ]
• Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part C [ Time Frame: Up to approximately Day 29 ]
• Measurement of Changes in Platelet Counts After M254 Administration Compared to IVIg - Part C [ Time Frame: Baseline to approximately Day 29 ]
• Number and Severity of AEs - Part D [ Time Frame: Up to approximately Day 71 ]
• Frequency of Clinically Significant Abnormalities in Clinical Safety Laboratory Values - Part D [ Time Frame: Up to approximately Day 71 ]
• Shift From Baseline in Clinically Significant Abnormalities for Clinical Safety Laboratory Values - Part D [ Time Frame: Up to approximately Day 71 ]
• Frequency of Clinically Significant Abnormalities in Vital Signs - Part D [ Time Frame: Up to approximately Day 71 ]
• Shift From Baseline in Clinically Significant Abnormalities for Vital Signs - Part D [ Time Frame: Up to approximately Day 71 ]
• Frequency of Clinically Significant Abnormalities in ECGs - Part D [ Time Frame: Up to approximately Day 71 ]
• Shift From Baseline in Clinically Significant Abnormalities for ECGs - Part D [ Time Frame: Up to approximately Day 71 ]

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: March 5, 2019)

• Maximum Plasma Concentration (Cmax) of M254 - Part A, B, and C [ Time Frame: Day 1 to Day 29 ]
• Time to Maximum Plasma Concentration (Tmax) of M254 - Part A, B, and C [ Time Frame: 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 [ Time Frame: Day 1 to Day 29 ]
• 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 ]
• Volume of Distribution (Vd) of M254 - Part A, B, and C [ Time Frame: Day 1 to Day 29 ]
• Clearance of Drug (CL) M254 - Part A, B, and C [ Time Frame: Day 1 to Day 29 ]
• Mean Residence Time (MRT) of M254 - Part A, B, and C [ Time Frame: Day 1 to Day 29 ]
• Apparent Terminal-phase Half-life (t½) of M254 - Part A, B, and C [ Time Frame: Day 1 to Day 29 ]
• Cmax of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• Tmax of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• AUC(0 last) of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• AUC(0 ∞) of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• Vd of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• CL of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• MRT of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• t½ of M254 - Part D [ Time Frame: Day 1 to Day 71 ]
• Measurements of Changes in Platelet Counts After M254 Administration - Part D [ Time Frame: Baseline to approximately Day 71 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• 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
• 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.
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Masking Description:

Part A: Double (Subject, Investigator);

Part B, C, and D: Open Label Investigations

Primary Purpose: Treatment

• Condition: Immune Thrombocytopenic Purpura (ITP)

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

Study Arms

• Experimental: Part A
  Healthy volunteers will receive a single ascending dose of M254 or placebo
  Interventions:

  • Biological: Biological: M254
  • Drug: Placebo
• Experimental: Part B
  Immune thrombocytopenic purpura (ITP) patients will receive a single ascending dose of M254 followed by IVIg
  Interventions:

  • Biological: Biological: M254
  • Biological: Intravenous immunoglobulin (IVIg)
• Experimental: Part C
  ITP patients will receive a single dose of M254 or IVIg, followed by a single dose of the other drug approximately 28 days later
  Interventions:

  • Biological: Biological: M254
  • Biological: Intravenous immunoglobulin (IVIg)
• Experimental: Part D
  ITP patients will receive repeated doses of M254
  Intervention: Biological: Biological: M254

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: March 5, 2019): 70
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 2022
• Estimated Primary Completion Date: April 2022 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Contacts

Contact: Momenta General Queries; +1 617-491-9700; ClinicalTrialInfo@momentapharma.com

• Listed Location Countries: Belgium, France, Germany, Hungary, Italy, Netherlands, Poland, Spain, United States

Administrative Information

• NCT Number: NCT03866577
• Other Study ID Numbers: MOM-M254-001; 2018-003534-32 ( EudraCT Number )
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: Momenta Pharmaceuticals, Inc.
• Study Sponsor: Momenta Pharmaceuticals, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Momenta General Queries; Momenta Pharmaceuticals, Inc.

• PRS Account: Momenta Pharmaceuticals, Inc.
• Verification Date: December 2020