"The MaP Study": Mapping the Patient Journey in MMA and PA
NCT ID: NCT03484767
Last Updated: 2021-08-02
Study Results
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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COMPLETED
97 participants
OBSERVATIONAL
2018-03-20
2021-05-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Methylmalonic Acidemia Participants
Individuals with isolated MMA (mut0 and mut-)
No interventions assigned to this group
Propionic Acidemia Participants
Individuals with isolated PA
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Elevated plasma/serum/DBS or urine methylmalonic acid levels
* Presence of normal serum/plasma vitamin B12 and plasma homocysteine levels
* Confirmed by molecular genetic testing. Genetic testing can be performed after the administration of informed consent if not available, however, molecular genetic results must be confirmed before the second study visit
PA Only
• Patient has a confirmed diagnosis of isolated PA based on the following criteria:
* Elevated plasma/DBS/urine 2-MC and/or 3-HP
* Elevated plasma/serum/DBS propionylcarnitine (C3)
* Confirmed by genetic testing for mutations of the PCCA or PCCB genes. Genetic testing can be performed after the administration of informed consent if not available, however, molecular genetic results must be confirmed before the second study visit
Both MMA and PA
* Patient (and/or legally authorized representative as applicable to local regulations) is willing and able to comply with study-related assessments and activities
* Patient or legally authorized representative is willing and able to provide informed consent and/or assent as mandated by local regulation
Exclusion Criteria
* The patient is pregnant or lactating at the time of screening. (Note: Patients who become pregnant during the study may remain in the study) MMA Only
* Patients diagnosed with isolated MMA cblA, cblB, or cblD enzymatic subtypes or methylmalonyl-CoA epimerase deficiency or combined MMA with homocystinuria PA Only
* Patient has a confirmed diagnosis of multiple carboxylase deficiency
ALL
No
Sponsors
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ModernaTX, Inc.
INDUSTRY
Responsible Party
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Locations
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Stanford Health Services
Stanford, California, United States
Emory Univeristy
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Icahn School of Medicine at Mount Sinai
New York, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Seattle Children's Hospital
Seattle, Washington, United States
Hôpital de la Timone
Marseille, Bouches-du-Rhône, France
Hôpital Necker - Enfants Malades
Paris, Paris, France
Hospital de Cruces
Barakaldo, Vizcaya, Spain
Great Ormond Street Hospital
Bloomsbury, Greater London, United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
Manchester University Hospitals
Manchester, , United Kingdom
Countries
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Other Identifiers
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mRNA-3704-P001
Identifier Type: -
Identifier Source: org_study_id
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