Ignite Creation Date:
2024-05-06 @ 1:54 PM
Last Modification Date:
2024-10-26 @ 1:21 PM
Study NCT ID:
NCT04154488
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-07
First Post:
2019-11-04
Brief Title:
A Study of Mavorixafor in Participants With Congenital Neutropenia and Chronic Idiopathic Neutropenia Disorders
Sponsor:
X4 Pharmaceuticals
Organization:
X4 Pharmaceuticals
Study Overview
Official Title:
A Phase 1b2 Open-Label Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia The main goal of Part 1 Phase 1b is to help researchers learn more about how the investigational medicine mavorixafor impacts people living with chronic neutropenia including congenital idiopathic and cyclic In Part 2 Phase 2 the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia
Detailed Description:
Part 1 Participants will receive one oral dose of mavorixafor and be monitored for 8 hours to see if neutrophil cell counts increase Participants screened after implementation of Protocol Version 80 do not need to enter Part 1 and can start with the Part 2 Screening visit Day -28 to Day -1
Part 2 For participants enrolled in the study before implementation of Protocol Version 80 Part 2 will include those who completed Part 1 and exhibited a response to treatment Eligible participants from Part 1 enrolled in the study before implementation of Protocol Version 80 can directly roll-over to the Baseline Day -3 to Day -1 visit in Part 2 Participants will receive once daily oral dosing of mavorixafor for 6 months and be monitored throughout to see if neutrophil cell counts increase
Study visits can be conducted at-home or at one of many study clinic locations depending on the participants preference
Part 2 For participants enrolled in the study before implementation of Protocol Version 80 Part 2 will include those who completed Part 1 and exhibited a response to treatment Eligible participants from Part 1 enrolled in the study before implementation of Protocol Version 80 can directly roll-over to the Baseline Day -3 to Day -1 visit in Part 2 Participants will receive once daily oral dosing of mavorixafor for 6 months and be monitored throughout to see if neutrophil cell counts increase
Study visits can be conducted at-home or at one of many study clinic locations depending on the participants preference
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None