Combined Malonic and Methylmalonic Aciduria (CMAMMA): Gene Identification and Outcome Study

NCT ID: NCT01289158

Last Updated: 2011-02-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

6 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-02-28

Study Completion Date

2012-02-29

Brief Summary

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The investigators are interested in learning more about the changes found in the condition called "Combined elevation of Malonic and MethylMalonic Acid, or CMAMMA. " Malonic, or MA and MethylMalonic, or MMA, are acids formed from the breakdown of protein under normal conditions. However, in the condition called CMAMMA there is an increase of these acids in the blood and urine, which is not normal.

Some people with high MA and MMA in their blood and urine have a serious disease, starting as a baby or young child that includes heart disease and problems in learning. These people have changes in a special enzyme called Malonyl CoA Decarboxylase (MCD). Other people who have a high level of MA and MMA do not have any obvious illness. The investigators are not sure why they have high levels of MA and MMA and why they are not sick.

The goal of this study is to learn more about why some people have a high level of MA and MMA and to make sure there are no medical problems as a result of these high levels. The investigators also want to find out which gene and enzyme cause the high levels of MA and MMA.

Detailed Description

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Conditions

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Malonic Aciduria Methylmalonic Acidemia

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

CROSS_SECTIONAL

Study Groups

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non-classical CMAMMA, classical CMAMMA

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

1. Elevated Malonic and Methylmalonic Acid in blood and urine
2. Any age
3. Any sex
4. Asymptomatic

Exclusion Criteria

1. Defect in malonyl-coenzyme A decarboxylase (MCD) enzyme
2. History of metabolic acidosis, developmental delay and seizures
Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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McGill University Health Centre/Research Institute of the McGill University Health Centre

OTHER

Sponsor Role lead

Responsible Party

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The Research Institute of the MUHC | McGill University Health Centre

Locations

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McGill University Health Center

Montreal, Quebec, Canada

Site Status RECRUITING

Countries

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Canada

Central Contacts

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Ahmed Alfares, M.B.B.S

Role: CONTACT

5144124427

Facility Contacts

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Ahmed Alfares, M.B.B.S

Role: primary

5144124427

Related Links

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http://muhc.ca/research/dashboard

The Research Institute of the McGill University Health Centre

Other Identifiers

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10-131-PED

Identifier Type: -

Identifier Source: org_study_id

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