NCT01224483

Brief Summary

Paroxysmal nocturnal hemoglobinuria (PNH) is a hematopoietic stem cell disorder in which unregulated activation of the complement system leads to significant ischemic morbidities with shortened lifespan. Life-threatening thromboembolism (TE) is the most feared complication of PNH, accounting for up to 45% of patient deaths. It is estimated that 40% of PNH patients experience a clinically evident TE and 60% of patients without clinically diagnosed TE demonstrate TE by high-sensitivity MRI, indicating the ongoing thrombotic risk in most patients with PNH. Much of these data come from PNH patients from European descent. To understand the impact of TE in patients with PNH from non-European regions, we performed this study to evaluate the clinical characteristics of Korean patients with PNH.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
350

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Aug 2010

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2010

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

October 18, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 20, 2010

Completed
12 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2010

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2010

Completed
Last Updated

October 20, 2010

Status Verified

October 1, 2010

Enrollment Period

3 months

First QC Date

October 18, 2010

Last Update Submit

October 19, 2010

Conditions

Eligibility Criteria

Age1 Year+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Korean PNH patients: diagnosis confirmed by Ham's test or Flow cytometry

You may qualify if:

  • Diagnosis confirmed by Ham's test or Flow cytometry Patients have any Flow cytometry data

You may not qualify if:

  • Patients have no Flow cytometry data

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Division of Hematology Oncology, Samsung Medical Center

Seoul, 135-710, South Korea

RECRUITING

Related Publications (3)

  • Jang JH, Kim JS, Yoon SS, Lee JH, Kim YK, Jo DY, Chung J, Sohn SK, Lee JW. Predictive Factors of Mortality in Population of Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH): Results from a Korean PNH Registry. J Korean Med Sci. 2016 Feb;31(2):214-21. doi: 10.3346/jkms.2016.31.2.214. Epub 2016 Jan 26.

  • Kim JS, Jang JH, Yoon SS, Lee JH, Kim YK, Jo DY, Chung JS, Sohn SK, Lee JW. Distinct subgroups of paroxysmal nocturnal hemoglobinuria (PNH) with cytopenia: results from South Korean National PNH Registry. Ann Hematol. 2016 Jan;95(1):125-133. doi: 10.1007/s00277-015-2511-z. Epub 2015 Sep 29.

  • Lee JW, Jang JH, Kim JS, Yoon SS, Lee JH, Kim YK, Jo DY, Chung J, Sohn SK. Clinical signs and symptoms associated with increased risk for thrombosis in patients with paroxysmal nocturnal hemoglobinuria from a Korean Registry. Int J Hematol. 2013 Jun;97(6):749-57. doi: 10.1007/s12185-013-1346-4. Epub 2013 May 1.

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesMyelodysplastic SyndromesBone Marrow Diseases

Study Officials

  • Jun Ho Jang, M.D.

    Samsung Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 18, 2010

First Posted

October 20, 2010

Study Start

August 1, 2010

Primary Completion

November 1, 2010

Study Completion

December 1, 2010

Last Updated

October 20, 2010

Record last verified: 2010-10

Locations