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

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ClinicalTrials.gov Identifier: NCT03484767
Recruitment Status : Recruiting
First Posted : April 2, 2018
Last Update Posted : August 21, 2019
Sponsor:
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 21, 2019
Actual Study Start Date March 20, 2018
Estimated Primary Completion Date March 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 ]
Change History Complete list of historical versions of study NCT03484767 on ClinicalTrials.gov Archive Site
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

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: June 7, 2018)
120
Original Estimated Enrollment
 (submitted: March 29, 2018)
60
Estimated Study Completion Date March 2021
Estimated Primary Completion Date March 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: Moderna Clinical Trials 855-663-6762 clinicaltrials@modernatx.com
Listed Location Countries France,   Italy,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
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
Responsible Party ModernaTX, Inc.
Study Sponsor ModernaTX, Inc.
Collaborators Not Provided
Investigators Not Provided
PRS Account ModernaTX, Inc.
Verification Date August 2019