Study Assessing Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of MGTA-145 in Healthy Volunteers as a Single Agent or in Combination With Plerixafor

A Randomized, Placebo-Controlled, Ascending Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Parameters of MGTA-145 in Healthy Subjects Administered as a Single Agent, as Well as in Combination With Plerixafor

To investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of MGTA-145 in healthy volunteers as a single agent and in combination with plerixafor.

Study Overview

• Status: Completed
• Conditions: Healthy Volunteers
• Intervention / Treatment: Biological: MGTA-145; Biological: Placebo; Biological: plerixafor

Detailed Description

The study consists of up to four parts: Part A, to investigate the safety and tolerability of MGTA-145; Part B, to investigate the safety and tolerability of MGTA-145 when administered in combination with plerixafor; Part C, to investigate the safety and tolerability of two sequential days of dosing MGTA-145 in combination with plerixafor; and Part D, to investigate the safety, tolerability, and measure by apheresis, the total number of CD34+ cells mobilized after a dose of MGTA-145 administered in combination with plerixafor.

• Study Type: Interventional
• Enrollment (Actual): 107
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States, 45227
      • Medpace CPU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

1. Age from 18 to 60 years
2. Body weight ≥50 kg and body mass index 19 to 33 kg/m2
3. No clinically significant abnormalities on physical examination at Screening
4. Non-smoker for at least 2 years
5. No clinically significant lab abnormalities for renal, hepatic or hematologic parameters
6. No clinically significant abnormalities on ECG
7. Female subjects must be of non-childbearing potential
8. Male subjects who are sexually abstinent or surgically sterilized (vasectomy), or those who are sexually active with a female partner(s) and agree to use an acceptable method of contraception
9. No contraindications for apheresis

Exclusion Criteria:

1. Any clinically significant laboratory value outside the normal range at screening
2. Donation of more than 500 mL of blood or plasma within 12 weeks prior to dosing
3. History of alcoholism or drug abuse within the past 3 years
4. Subject has used any prescription drugs within 14 days prior to dosing or any dietary supplements or non-prescription drugs within 7 days prior to dosing
5. Acute illness, infection (requiring medical treatment [eg, antibiotics]), or surgery within 4 weeks of dosing
6. Seropositive for hepatitis B surface antigen, hepatitis C virus antibody, or human immunodeficiency virus
7. Subject has received another investigational drug or participated in an investigational drug or device study within 12 weeks prior to dosing
8. History of anaphylaxis or clinically important reaction to any drug including plerixafor
9. Any clinically significant hematologic, cardiovascular, pulmonary, central nervous system, metabolic, renal, hepatic, or gastrointestinal conditions

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Single Ascending Dose of MGTA-145 or placebo; MGTA-145 or placebo dose escalation as single agent, single dose	Biological: MGTA-145; MGTA-145 will be given in various doses intravenously; Biological: Placebo; Placebo will be given in various doses intravenously
Placebo Comparator: Single Dose MGTA-145 or placebo plus plerixafor; MGTA-145 or placebo in combination with plerixafor, single dose	Biological: MGTA-145; MGTA-145 will be given in various doses intravenously; Biological: Placebo; Placebo will be given in various doses intravenously; Biological: plerixafor; 240 µg/kg subcutaneously; Other Names: Mozobil
Experimental: Single dose MGTA-145 plus plerixafor for 2 sequential d; MGTA-145 in combination with plerixafor on two consecutive days; single dose per day	Biological: MGTA-145; MGTA-145 will be given in various doses intravenously; Biological: plerixafor; 240 µg/kg subcutaneously; Other Names: Mozobil
Experimental: Single dose MGTA-145 plus plerixafor followed by apheresis; MGTA-145 in combination with plerixafor followed by apheresis	Biological: MGTA-145; MGTA-145 will be given in various doses intravenously; Biological: plerixafor; 240 µg/kg subcutaneously; Other Names: Mozobil

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety as measured by incidence of treatment-emergent adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs).	Investigate the safety and tolerability of MGTA-145 following intravenous (IV) administration as monotherapy or in combination with plerixafor in healthy subjects (e.g. adverse events, clinical laboratory tests, vital signs, ECGs)	28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics Biomarkers	Investigate area under the curve (AUC) of MGTA-145	15 days
Pharmacokinetics Biomarkers	Investigate maximum plasma concentration (Cmax) of MGTA-145	15 days
Pharmacokinetic Biomarkers	Investigate clearance (CL) of MGTA-145	15 days
Pharmacokinetic Biomarkers	Investigate the volume of distribution at steady state (Vdss) of MGTA-145	15 days
Pharmacokinetic Biomarkers	Investigate the half-life of MGTA-145	15 days
Pharmacodynamic Biomarkers	Assess CD34+ cells per uL of blood by flow cytometry	15 days
Pharmacodynamic Biomarkers	Assess stem cell progenitors (colony forming units)	15 days

Collaborators and Investigators

• Sponsor: Ensoma

Study record dates

Study Major Dates

• Study Start (Actual): April 22, 2019
• Primary Completion (Actual): February 25, 2020
• Study Completion (Actual): February 25, 2020

Study Registration Dates

• First Submitted: April 22, 2019
• First Submitted That Met QC Criteria: April 27, 2019
• First Posted (Actual): May 1, 2019

Study Record Updates

• Last Update Posted (Estimated): January 11, 2024
• Last Update Submitted That Met QC Criteria: January 9, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Anti-Infective Agents; Antiviral Agents; Anti-HIV Agents; Anti-Retroviral Agents; Plerixafor
• Other Study ID Numbers: 145-HV-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No