Clinical trial NCT06564974: Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.
Official title: Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
Neuromuscular
AGAMREE®
Lead sponsor: Catalyst Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2024-08-21
Enrollment: 250
Sex: MALE
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Male subjects 2 years of age and older will be selected from DMD centers and Institutions across the US. Subjects will be recruited from the sites respective database.

Brief summary: The goal of this observational study is to follow patients being treated with the FDA approved drug AGAMREE® in male patients 2 years of age or older with Duchenne's Muscular Dystrophy for long term safety and quality of life.

Detailed description: This is a multi-center, observational, prospective, longitudinal registry designed to collect data in male patients aged 2 years and older with DMD treated with AGAMREE®.

This registry will be conducted in the US, at approximately 25 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.

Evaluations will include:

* Growth parameters
* Body mass index (BMI)
* Vital Signs
* Physical Exam
* Laboratory (Chemistry and Hematology)
* North Star Ambulatory Assessment (NSAA)
* Performance of Upper Limb (PUL)
* Cardiovascular status
* Fractures
* Bone density
* Puberty
* Quality of life (QoL)
* Adverse events (AEs)

Patients will be followed for approximately 5 years in the registry and will return to the site for Yearly Follow-up Visits (+/- 30 days) for registry assessments. Information on standard of care treatment and procedures for management of DMD will also be collected. Patients and/or their parents/legal guardians will be asked to complete paper QoL questionnaires at enrollment and at each Yearly Follow-up Visit (+/- 30 days).

Eligibility criteria: Inclusion Criteria:

1. Patient or parent/guardian willing and able to provide written informed consent after the nature of the registry has been explained and before the start of any registry-related procedures.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to antidystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
5. Currently on treatment with 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.

Primary outcomes:
measure: Change in BMI Z score at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Height Z-score at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Puberty development using the Tanner Stage at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Number of Fractures based on Spine x-rays at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Presence or absence of Cataracts at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Presence of Cardiomyopathy at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Evaluation of Hormonal status based on Cortisol levels at enrollment.
timeFrame: At enrollment
measure: Presence or absence of Glaucoma at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Number of patients experiencing any Adverse Events (AEs) or Serious Adverse Events (SAEs) after signing the Informed Consent Form.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Pediatric Quality of Life Inventory Duchenne Muscular Dystrophy (PedsQL DMD) Module at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Duchenne Muscular Dystrophy Quality of Life (DMD-QoL)at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Euro Qol 5 Dimensions (EQ-5D) at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: t enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Patient Reported Outcome Measure for the Upper Limb (PROM UL) at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in North Star Ambulatory Assessment (NSAA) at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
measure: Change in Performance of Upper Limb (PUL) at the End-of-Registry ( 5 years) or Early-Termination.
timeFrame: At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.
Secondary outcomes:
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Little Rock
country: United States
facility: Arkansas Childrens Hospital
state: Arkansas
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical and Translational Science Institue
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Chicago
country: United States
facility: Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Health - Riley Hospital for Children
state: Indiana
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: North Worcester
country: United States
facility: University of Massachusetts Memorial Medical Center
state: Massachusetts
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital
state: Michigan
city: Charlotte
country: United States
facility: Atrium Health Neurosciences Institute
state: North Carolina
city: Durham
country: United States
facility: Duke University Medical Center and Childrens Health Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute
state: Pennsylvania
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia (CHOP)
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Denton
country: United States
facility: Neurology Rare Disease Center - Neurology & Neuromuscular Care Center
state: Texas
city: San Antonio
country: United States
facility: The University of Texas Health Science Center at San Antonio
state: Texas
city: Charlottesville
country: United States
facility: University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center
state: Virginia
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: San Juan
country: Puerto Rico
facility: FDI Clinical Research
Study officials:
affiliation: Catalyst Pharmaceuticals
name: Gary Ingenito, MD, PhD
role: STUDY_DIRECTOR
affiliation: Arkansas Childrens Hospital
name: Aravindham Veerapandiyan, MD
role: PRINCIPAL_INVESTIGATOR
References: