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B7 Coreceptor Molecules in Hyper IgD Syndrome Form of Mevalonate Kinase Deficiency (HIDS-MKD)

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2012 by Michigan Technological University
Sponsor:
Information provided by (Responsible Party):
Kenneth Michael Gibson, Michigan Technological University
ClinicalTrials.gov Identifier:
NCT01568736
First received: March 29, 2012
Last updated: March 30, 2012
Last verified: March 2012

March 29, 2012
March 30, 2012
March 2012
March 2016   (final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT01568736 on ClinicalTrials.gov Archive Site
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B7 Coreceptor Molecules in Hyper IgD Syndrome Form of Mevalonate Kinase Deficiency
B7 Coreceptor Molecules as Clinically-Relevant Surrogate Biomarkers in the Hyper IgD Syndrome (HIDS) Form of Mevalonate Kinase Deficiency (MKD)

The hyper IgD syndrome (HIDS) is an inflammatory disease caused by mevalonate kinase deficiency. There is no cure, and available treatments of HIDS febrile episodes have shown limited clinical efficacy. The development of effective interventions for HIDS is limited by our poor understanding of the disease. The goal of the study is to better characterize the inflammatory response during HIDS episodes and to determine the relationship between this response and blood and urine markers of mevalonate kinase deficiency. This knowledge will help us learn more about the cause of the disease and should lead to the identification of new disease biomarkers that can be used to evaluate clinical efficacy in future therapeutic trials.

The primary hypothesis is that the costimulatory B7 glycoprotein abnormalities identified in the murine MKD model will be recapitulated in sera obtained from human HIDS patients, either before, during or after febrile episodes. The secondary hypothesis is that B7 glycoprotein molecule levels will correlate with clinical symptomatic severity score, other known biomarkers of HIDS, markers of inflammation and or markers of isoprenoid metabolism.

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Observational
Observational Model: Case-Only
Time Perspective: Prospective
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Retention:   Samples With DNA
Description:

Blood Urine

Non-Probability Sample

Subjects with Hyper IgD Syndrome (HIDS)

  • Hyper IgD Syndrome
  • Mevalonate Kinase Deficiency
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
10
March 2016
March 2016   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • any race or ethnicity
  • diagnosed with HIDS

Exclusion Criteria:

  • parents' inability to donate blood
  • currently having cancer, renal failure, diabetes, liver disease, thyroid diseases, major infectious diseases or immunodeficiency
  • pregnancy
  • inability to provide consent
Both
18 Years to 89 Years
No
Contact: Anna Simon, MD, PhD +31 24 3618819 A.Simon@aig.umcn.nl
United States,   Netherlands
 
NCT01568736
STAIR 7003
Yes
Kenneth Michael Gibson, Michigan Technological University
Michigan Technological University
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Michigan Technological University
March 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP