Nemaline Myopathy Clinical Research Network (NM-CTRN)

Part of paid clinical trials in Palo Alto, California.

Sponsor
Stanford University
Study ID
NCT06774703
Status
Not Yet Recruiting

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Conditions

  • Nemaline Myopathy

Eligibility Criteria

Sex
ALL
Age
0 Years - 18 Years
Healthy Volunteers
Not accepted

Study Details

The goal of this study is to establish a research network to help define the natural disease history and clinical outcome measures for Nemaline Myopathy (NM).

Key Dates

Start date
Jan 31, 2025
Status verified
Jan 2025
Primary completion
Dec 31, 2029
Completion
Dec 31, 2029

Study Design

Enrollment
50 participants (estimated)

Arms

  • Arm: Individuals with Nemaline Myopathy
    All participants in the study will have a diagnosis of Nemaline Myopathy, with either a pathogenic or likely pathogenic mutation in ACTA1 (AD) or NEB (AR). Participants can be either ambulatory or non-ambulatory and must be between the ages of 0-18.

Primary Outcome Measure

Validate the change over 36 months using the Alberta Infant Motor Scale (AIMS) Score [ Time Frame: 36 months ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
Stanford University/Lucile Packard Children's HospitalPalo AltoCalifornia94304
Sarah Ismail, BSc
[email protected]
650-460-4596
Carolina Tesi-Rocha, MD
Tina Duong, PhD, PT
John W Day, MD, PhD
National Institute of HealthBethesdaMaryland20892
Christopher Mendoza
[email protected]
Carsten Bonnemann, MD
Boston Children's HospitalBostonMassachusetts02115
Aleah Pagan, BA
[email protected]
617-919-7498
Alan Beggs, PhD
Leslie Hayes, MD
St Jude Children's Research HospitalMemphisTennessee38105
Jean Laboe, MSN, RN
[email protected]
901-595-1693
Richard Finkel, MD
UT Southwestern Medical Centre/Children's Health DallasDallasTexas75207
Holly Lawrence, CCRC
[email protected]
214-456-9561
Kaitlin Batley, MD

Find similar trials in Palo Alto, CA

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Stanford University/Lucile Packard Children's Hospital· Palo Alto, CANational Institute of Health· Bethesda, MDBoston Children's Hospital· Boston, MASt Jude Children's Research Hospital· Memphis, TNUT Southwestern Medical Centre/Children's Health Dallas· Dallas, TX

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