A Study Comparing Once-Daily vs. Twice-Daily Dosing of X4P-001 in Healthy Volunteers

NCT02680782 | Terminated Phase 1 FDA Drug

• 1 other identifier: X4P-001-REGA
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

This study will evaluate the safety, tolerability and pharmacokinetics of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily. This study will also assess the pharmacodynamic effects of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily on levels of circulating white blood cells (total and by cell type).

Conditions

Healthy

Keywords

Healthy Volunteers; Comparison; Once-Daily; Twice-Daily; X4P-001

Outcome Measures

Primary Outcomes (4)

• Incidence of treatment emergent adverse events (safety and tolerability) in subjects administered X4P-001 200 mg twice daily compared with 400 mg once daily.

  Safety assessments include ongoing monitoring of adverse events, ongoing monitoring of concomitant medications and regulatory scheduled vital signs, physical examinations and laboratory tests (hematology, clinical chemistry, urinalysis and coagulation).

  Up to 14 to 21 days post-last dose

• Maximum Plasma Concentration (Cmax) of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily.

  Cmax data will be collected to determine the pharmacokinetics of X4P-001 administered as 200 mg twice daily and 400 mg once daily.

  Up to 48 hours post-dose

• Area under the curve (AUC) of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily.

  AUC data will be collected to determine the pharmacokinetics of X4P-001 administered as 200 mg twice daily and 400 mg once daily.

  Up to 48 hours post-dose

• Minimum Plasma Concentration (Cmin) of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily.

  Amin data will be collected to determine the pharmacokinetics of X4P-001 administered as 200 mg twice daily and 400 mg once daily.

  Up to 48 hours post-dose

Secondary Outcomes (2)

• To assess the pharmacodynamic effects of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily on levels of circulating white blood cells (total and by cell type).

  Up to 48 hours post-dose

• To assess the pharmacodynamic effects of X4P-001 administered as 200 mg twice daily compared with 400 mg once daily on levels of circulating mononuclear cell phenotypes by cell surface markers.

  Up to 48 hours post-dose

Study Arms (2)

X4P-001 QD

EXPERIMENTAL

Subjects will receive study drug in two dosing periods. In this arm subjects will receive drug once daily in the first period, followed by twice daily in the second dosing period.

Drug: X4P-001

X4P-001 BID

EXPERIMENTAL

Subjects will receive study drug in two dosing periods. In this arm subjects will receive drug twice daily in the first period, followed by once daily in the second dosing period.

Drug: X4P-001

Interventions

X4P-001 DRUG

100 mg capsules, administered orally for 10 days either as 200 mg BID or 400 mg QD. Subjects will be randomized to determine if they will receive the drug QD or BID in the first dosing period.

Also known as: AMD11070

X4P-001 BID; X4P-001 QD

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Between 18 and 65 years of age, inclusive.
• Have signed the current approved informed consent form.
• For women of childbearing potential, (a) agree to use effective contraceptive methods from screening, through the study, and for at least 4 weeks after the last dose of study drug; and (b) have a negative pregnancy test (serum or urine) at screening and on Day -1 prior to each Dosing period.
• For men, agree both to (a) use effective contraceptive methods and (b) abstain from donating sperm, from admission to the in-residence unit prior to the first Dosing Period, through the study, and for at least 4 weeks after the last dose of study drug.
• Be willing and able to comply with this protocol.

You may not qualify if:

• Is an employee of the Phase 1 unit or an immediate family member of an employee.
• Has a BMI \<18.0 or \>30.0.
• Has a history of hypersensitivity or allergy to any drug compound, food or other substance assessed as significant by the Investigator.
• Has a history or presence of any medical condition capable of altering absorption, metabolism or elimination of drugs (history of routine cholecystectomy is permitted).
• Has alcohol intake exceeding 21 units per week for males or 14 units per week for females, where 1 unit = 12 oz (360 mL) beer, 5 oz (150 mL) wine, or 1.5 oz (45 mL) distilled spirits.
• Has within the past 12 months used illicit drugs.
• Has within the past 6 months been a smoker or used tobacco or nicotine replacement products.
• Is within 6 months post-partum or termination of a pregnancy.
• Has within the past 30 days or 5 half-lives, whichever is longer, participated in any other clinical trial involving an investigational treatment.
• Has within the past 30 days had an acute medical illness, including an active infection.
• Has within the past 30 days donated more than 500 mL of blood.
• Has within the past 30 days, or is scheduled to have while participating in the study, surgery requiring general anesthesia.
• Has within the past 30 days, or is scheduled to have while participating in the study, any immunizations.
• Has within the past 14 days been nursing.
• Has within the past 14 days donated plasma.

Sponsors & Collaborators

• X4 Pharmaceuticals: lead
• Covance: collaborator

Study Sites (1)

Covance CRU, Inc.

Daytona Beach, Florida, 32117, United States

Location

MeSH Terms

Interventions

mavorixafor

Study Officials

• Lu Gan, MD, PhD

  X4 Pharmaceuticals, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2016
• First Posted: February 12, 2016
• Study Start: January 12, 2016
• Primary Completion: February 28, 2016
• Study Completion: February 28, 2016
• Last Updated: December 7, 2018

Record last verified: 2018-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)