Trials Sponsors Conditions Drugs Pricing

Conditions

Accumulation of Mucopolysaccharides Morquio B Disease Morquio Syndrome Morquio Syndrome A

Keywords

BiomarkerMorquio Disease

Outcome Measures

Primary Outcomes (1)

Development of a new MS-based biomarker for the early and sensitive diagnosis of Morquio disease from blood (plasma)
New methods, like mass-spectrometry give a good chance to characterize specific metabolic alterations in the blood of affected patients that allow diagnosing in the future the disease earlier, with a higher sensitivity and specificity.
24 month

Secondary Outcomes (1)

Testing for clinical robustness, specificity and long-term stability of the biomarker
36 months

Study Arms (1)

Observation

Patients with Morquio disease

Eligibility Criteria

Age12 Months+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodProbability Sample

Study Population

Patients with Morquio disease

You may qualify if:

Informed consent will be obtained from the patient or the parents before any study related procedures

Sponsors & Collaborators

CENTOGENE GmbH Rostock
Industrylead

Study Sites (5)

Children's Hospital, Faculty of Medicine, Ain Shams University

Cairo, 89075, Egypt

Centogene AG

Rostock, 18055, Germany

Amrita Institute of Medical Sciences & Research Centre

Kochi, Kerala, 682041, India

Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN)

Mumbai, 400705, India

Lady Ridgeway Hospital for Children

Colombo, 00800c, Sri Lanka

Biospecimen

Retention: SAMPLES WITH DNA

For the development of the new biomarkers using the technique of mass-spectometry 10 ml EDTA blood and a dry blood spot filter card are used. To prove the correct Morquio diagnosis in those patients where up to the enrollment in the study no genetic testing has been done, sequencing of Morquio will be done as routine diagnostic sequencing. The analyses will done in the Centogene AG Am Strande 7 18055 Rostock Germany

MeSH Terms

Conditions

Mucopolysaccharidosis IV

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

Peter Bauer, Prof.
Centogene GmbH
STUDY CHAIR

Enrollment

0 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: PROSPECTIVE
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

October 21, 2011

First Posted

October 24, 2011

Study Start

August 20, 2018

Actual (reported)

Study Stopped

Biomarker for Morquio Disease (BioMorquio)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

Observation

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (5)

Related Links

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (1)

Study Arms (1)

Observation

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (5)

Related Links

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations