Study Stopped
Company Decision
Company Decision
Sponsor study identifier
observational
0
0 countries
N/A
Study Start
June 2018
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Primary Completion
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not function properly). WHIM is an acronym for some of the symptoms of the disorder - Warts, Hypogammaglobulinemia (low levels of certain antibodies), Infections and Myelokathexis (too many white blood cells in the bone marrow).This study includes 10-year retrospective (Retrospective Phase) and up to 5-year prospective (Prospective Phase) components.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
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Some registry-reported names shown below are not yet linked in this graph.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
March 10, 2017
Actual (reported)March 22, 2017
Actual (reported)June 2018
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Incidence of infections
Infections assessed by hospitalizations (including intensive care), antibiotic use, outpatient medical appointments and missed days of school/work.
Up to five years, from time of enrollment through study completion or early termination
Severity of infections
Up to five years, from time of enrollment through study completion or early termination
Incidence of warts
Up to five years, from time of enrollment through study completion or early termination
Severity of warts
Warts assessed by number and size of lesions, need for surgical, systemic or topical treatment and complications.
Up to five years, from time of enrollment through study completion or early termination
No intervention
Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.
Patients diagnoses with WHIM syndrome.
Blood or buccal swab for the purposes of identifying a CXCR4 mutation.
Sudha Parasuraman, MD
X4 Pharmaceuticals, Inc.
0 participants (actual enrollment reported by the registry).
First Submitted
March 10, 2017
First Posted
March 22, 2017
Study Start
June 2018
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Project to Assess Feasibility of a Rapid Access Metastatic Bone Disease Program
Change in quality of life over time
Quality of life as assessed by the quality of life instrument the 36-Item Short Form Survey (SF-36)
Up to five years, from time of enrollment through study completion or early termination
Change in quality of life over time
Quality of life as assessed by the quality of life instrument the Pediatric Quality of Life Inventory (Peds-QL)
Up to five years, from time of enrollment through study completion or early termination
Change in quality of life over time
Quality of life as assessed by the quality of life instrument the Life Quality Index (LQI)
Up to five years, from time of enrollment through study completion or early termination
Change in quality of life over time
Quality of life as assessed by the quality of life instrument the HPV Impact Profile (HIP)
Up to five years, from time of enrollment through study completion or early termination
Change in medical resource utilization
Up to five years, from time of enrollment through study completion or early termination
Change in absolute neutrophil count (ANC) over time
Up to five years, from time of enrollment through study completion or early termination
Change in absolute lymphocyte count (ALC) over time
Up to five years, from time of enrollment through study completion or early termination
Change in serum immunoglobulin over time
Up to five years, from time of enrollment through study completion or early termination
Changes in anti-vaccine antibodies over time
Up to five years, from time of enrollment through study completion or early termination
Contact the study team to confirm eligibility.
Primary Completion
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Study Completion
March 2022
Estimated (date passed)This estimate has passed. The registry has not confirmed an actual date for this milestone.
Last Updated
December 7, 2018
Record last verified: 2018-12
Download the complete ClinicalTrials.gov record imported by Syfrah, including fields not shown in this page. The registry may contain a newer revision.
Source: ClinicalTrials.gov.
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