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Retrospective Natural History Study of Infants and Toddlers With LAMA2-CMD

NCT04299321 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 75

Last updated 2022-02-07

No results posted yet for this study

Summary

This retrospective chart review study of 75-120 LAMA2-CMD patients will expand the investigators understanding of the natural history of this disease. Current and pending publications cover research performed only in ages 5-16 years; there is currently no documented natural history for patients ages 0-5 years. Data collected in this study has the potential to inform the design of future interventional studies that draw nearer to clinical trial readiness every day.

Conditions

  • Merosin Deficient Congenital Muscular Dystrophy

Sponsors & Collaborators

  • Cure CMD

    collaborator OTHER

  • The Bönnemann Laboratory, NINDS, National Institutes of Health

    collaborator UNKNOWN

  • Oscar H Mayer, MD, Children's Hospital of Philadelphia

    collaborator UNKNOWN

  • The Beggs Laboratory, Boston Children's Hospital

    collaborator UNKNOWN

  • Prothelia, Inc.

    lead INDUSTRY

Principal Investigators

  • Carsten Bönnemann, MD · NINDS/NIH

  • Reghan Foley, MD · NINDS/NIH

  • Oscar H Mayer, MD · Children's Hospital of Philadelphia

  • Alan Beggs, PhD · Boston Children's Hospital

  • Gustavo Dziewczapolski, PhD · Cure CMD

  • Rachel Alvarez, BS · Cure CMD

Eligibility

Min Age
2 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-04-24
Primary Completion
2021-12-31
Completion
2021-12-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04299321 on ClinicalTrials.gov