Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Official title

Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy [SUpplemental Patient dMd assessMents Investigating ouTcomes (SUMMIT)]

Overview

The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).

Detailed description

This is a multi-center, observational, prospective, longitudinal registry study, designed to collect safety data and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with DMD.

Primary Objective is to collect additional information on the safety of long-term treatment with AGAMREE® in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD) as determined by:

Growth parameters Body mass index (BMI) Musculoskeletal assessments Ophthalmological assessments Cardiovascular assessments Pubertal development assessments Adverse events (AEs)

Secondary Objective is to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires).

This registry will be conducted in the US, at approximately 40 sites known to treat and follow patients with DMD. The registry plans to enroll approximately 250 male patients aged 2 years and older with DMD.

Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 60 days) for assessments. Information on some historical and ongoing standard of care treatment and procedures for management of DMD will also be collected.

Interventions

• Drug Vamorolone
  Study treatment is AGAMREE®, which is commercially available as an oral suspension.

Primary outcome measures

• Change in Height z-score [Time frame: At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years).]
• Change in BMI z-score [Time frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years)]
• Change in North Star Ambulatory Assessment from baseline [Time frame: At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years).]
• Change in Performance of Upper Limb from baseline [Time frame: At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years).]
• Tanner stage documentation [Time frame: At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years).]
• Percentage of patients with cataract and/or glaucoma [Time frame: At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years).]
• Findings from lateral spine x-ray [Time frame: At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years).]
• Findings from anterior-posterior/posterior-anterior X-ray [Time frame: At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years).]
• Findings from hand-wrist X-ray [Time frame: At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).]
• Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry [Time frame: At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years).]

Secondary outcome measures (2)

• Changes in Duchenne Muscular Dystrophy Quality of Life questionnaire score [Time frame: At Enrollment Visit (baseline) and at each Yearly Follow-up Visit for up to 5 years.]
• Change in Patient Reported Outcome Measure for the Upper Limb score [Time frame: At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years).]

Eligibility criteria

Inclusion criteria

• Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
• Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
• Male patients at least 2 years old.
• Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
• Patient has a current, active prescription for, or is on, AGAMREE®.

Exclusion criteria

1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

United States · 31 centers

• Phoenix Children's Hospital — Phoenix
• Arkansas Childrens Hospital — Little Rock
• Loma Linda University Pediatric Subspecialty Clinics — Loma Linda
• Children's Hospital Los Angeles — Los Angeles
• Valley Children's Hospital — Madera
• Stanford University — Palo Alto
• University of California, Davis — Sacramento
• Children's National Health System — Washington D.C.
• … and 23 more centers

Puerto Rico · 2 centers

• San Jorge Children's Hospital — San Juan
• FDI Clinical Research — San Juan

Identifiers

NCT: NCT06564974 · DMD-001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗