"The MaP Study": Mapping the Patient Journey in MMA and PA

• ClinicalTrials.gov Identifier: NCT03484767
• Recruitment Status: Completed
• First Posted: April 2, 2018
• Last Update Posted: August 2, 2021
• Sponsor: ModernaTX, Inc.
• Information provided by (Responsible Party): ModernaTX, Inc.

Tracking Information

• First Submitted Date: March 13, 2018
• First Posted Date: April 2, 2018
• Last Update Posted Date: August 2, 2021
• Actual Study Start Date: March 20, 2018
• Actual Primary Completion Date: May 29, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: June 7, 2018)

• Change in plasma methylmalonic acid levels (MMA only) [ Time Frame: Baseline through 12 months ]
• Frequency of disease related clinical events in enrolled participants (mut0 and mut- MMA patients) [ Time Frame: Baseline through 12 months ]
• Changes in plasma 2-MC levels (PA only) [ Time Frame: Baseline through 12 months ]
• Changes in plasma 3-HP levels (PA only) [ Time Frame: Baseline through 12 months ]
• Frequency of disease related clinical events in enrolled participants (PA patients) [ Time Frame: Baseline through 12 months ]

Original Primary Outcome Measures (submitted: March 29, 2018)

• Change in plasma methylmalonic acid levels [ Time Frame: Baseline through 36 months ]
• Number of participants (mut0 and mut- MMA patients) with disease-related clinical events as assessed by DAIDS 2017, corrected version 2.1 [ Time Frame: Baseline through 36 months ]

• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: "The MaP Study": Mapping the Patient Journey in MMA and PA
• Official Title: A Longitudinal, Exploratory, Natural History Study to Further Characterize and Describe the Signs and Symptoms of Patients With Organic Acidemias
• Brief Summary: Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to their disease.
• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Individuals of any age with confirmed diagnosis of isolated MMA due to MUT deficiency (mut0 or mut-) or isolated PA

Condition

• Methylmalonic Acidemia
• Propionic Acidemia

• Intervention: Not Provided

Study Groups/Cohorts

• Methylmalonic Acidemia Participants
  Individuals with isolated MMA (mut0 and mut-)
• Propionic Acidemia Participants
  Individuals with isolated PA

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: November 23, 2020): 97
• Original Estimated Enrollment (submitted: March 29, 2018): 60
• Actual Study Completion Date: May 29, 2021
• Actual Primary Completion Date: May 29, 2021 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

MMA Only • Patient has a confirmed diagnosis of isolated MMA due to MUT deficiency (mut0 or mut-) based on the following 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:

• Estimated GFR <30 mL/min/1.73m2 based on age appropriate equations or patients who undergo chronic dialysis
• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: Child, Adult, Older Adult
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France, Spain, United Kingdom, United States
• Removed Location Countries: Italy

Administrative Information

• NCT Number: NCT03484767
• Other Study ID Numbers: mRNA-3704-P001
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: ModernaTX, Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: ModernaTX, Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: ModernaTX, Inc.
• Verification Date: July 2021