COMPASS - Characterizing Organic acidemias: MMA & PA Across Stages and Symptoms
Sponsored by Moderna
Powered by PicnicHealth
About the COMPASS Study
This observational study is designed to better understand the impact of MMA and PA on families. By collecting medical records and brief survey responses, researchers can understand how the condition is managed, how symptoms evolve, and where support is most needed.
By capturing comprehensive medical records directly from patients across all care sites, this study builds a longitudinal view of disease course — not limited to a single clinical location.
That data will inform understanding of MDEs, HCRU, treatment patterns, and quality of life in these ultra-rare conditions.
- Genetically confirmed diagnosis of MMA MUT or PA
- Born on or after January 1, 2005
- US-based only
- Caregivers and legacy families of eligible patients
- Diagnoses of isolated MMA subtypes (cblA, cblB, cblD)
- Methylmalonyl-CoA epimerase deficiency
- Combined MMA with homocystinuria
About PicnicHealth
We are clinicians, scientists, and technologists modernizing clinical research by removing barriers for participants and researchers.
We believe every patient story matters — and through rigorous methods, we transform those stories into high-quality insights that advance patient care.
We are a full-service, end-to-end research partner, combining technology and services to manage every stage of a study - from design and regulatory approvals, to participant enrollment and engagement, data capture, curation, secure transfer, and analysis.
What’s provided for families?
Complete medical record access
Families can view and securely share medical records, imaging, and other health data with any provider — including you.
Easy research participation
We make it simple for families to participate in research from wherever they are, with minimal burden.
Meaningful patient input
Every six months, families can share insights on their quality of life, well-being, and dietary habits through surveys.
Fair compensation
Families receive $250 at enrollment and additional compensation for completing surveys, recognizing the value of their time and contributions.
How it works
01 Design
Built with metabolic experts to answer key questions on MMA MUT and PA progression, outcomes, and quality of life. Study design is reviewed and approved by a Central IRB.
02 Consent
Flexible, patient-centric consent approaches reduce participation barriers and meet families in their preferred settings.
03 Curate
Ongoing data capture combines longitudinal EHR data and ePROs captured every six months via self-report and parent/proxy reports.
04 Deliver
All data are unified into a single, anonymized schema and transformed into regulatory-aligned datasets, with deliverables including analytics and publications.
How we support your practice
Your role is simple and low-burden: identify and invite eligible patients to join the study.
Minimal time commitment
Your role is to identify eligible families and share referral materials. PicnicHealth handles all enrollment, data collection, IRB compliance, and regulatory oversight.
Easy-to-share materials
Download our patient brochure below to hand off or email directly to eligible families. No portal login required.
Fair compensation
Families receive $250 at enrollment and additional compensation for completing surveys, recognizing the value of their time and contributions.
Ready to refer a patient?
Share the study website link with families, or download the patient brochure to pass along in clinic. For direct referrals or questions, reach the COMPASS team at PicnicHealth.
Industry and academic partners
FAQs
Approximately 30 minutes to identify eligible families and share referral materials. PicnicHealth manages all operational aspects: consent, medical record retrieval, PCOA administration, and regulatory compliance. No site contracts or IRB submissions are needed on your end.
Yes. Deceased patients with a confirmed diagnosis and available medical records may be included. Legacy families will not be asked to complete PCOAs. Legacy families with a second living child with MMA MUT or PA are eligible for full study participation including PCOAs.
Not necessarily. Families can upload genetic test results to the platform, or PicnicHealth will retrieve them through medical record review. If genetic results are unavailable, physician attestation of diagnosis is sufficient for enrollment.