Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy.
NCT Number: NCT06564974
Status: RECRUITING
Study Type: OBSERVATIONAL
Conditions: Duchenne Muscular Dystrophy
Interventions: DRUG: Vamorolone
Sponsor: Catalyst Pharmaceuticals, Inc.
Sex: MALE
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 250
Study Design: Observational Model: |Time Perspective: p
Start Date: 2024-07-31
Completion Date: 2030-11
Locations: Arkansas Childrens Hospital, Little Rock, Arkansas, 72202, United States|University of Massachusetts Memorial Medical Center, North Worcester, Massachusetts, 01655, United States|Neurology Rare Disease Center - Neurology & Neuromuscular Care Center, Denton, Texas, 76208, United States

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.

Primary Outcome Measures: Change in BMI Z score at the End-of-Registry ( 5 years) or Early-Termination., BMI is in kg/m\^2, using Height in CM (centimeters) and Weight in Kgs(kilograms). Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Height Z-score at the End-of-Registry ( 5 years) or Early-Termination., Height is measured in CM (centimeters). Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Puberty development using the Tanner Stage at the End-of-Registry ( 5 years) or Early-Termination., The Tanner Stage measures pubertal development of male external genitalia and pubic hair. This is a uniformly accepted scale and can be conducted by the Investigator or an endocrinologist.

The scale is 1-5 per the following:

Male External Genitalia Scale Stage 1: Testicular volume \< 4 ml or long axis \< 2.5 cm Stage 2: 4 ml-8 ml (or 2.5 to 3.3 cm long), 1st pubertal sign in males Stage 3: 9 ml-12 ml (or 3.4 to 4.0 cm long) Stage 4: 15-20 ml (or 4.1 to 4.5 cm long) Stage 5: \> 20 ml (or \> 4.5 cm long)

• Pubic Hair Scale Stage 1: No hair Stage 2: Downy hair Stage 3: Scant terminal hair Stage 4: Terminal hair that fills the entire triangle overlying the pubic region Stage 5: Terminal hair that extends beyond the inguinal crease onto the thigh

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Number of Fractures based on Spine x-rays at the End-of-Registry ( 5 years) or Early-Termination., Bone evaluations will be performed through lateral spine x-ray, Anteroposterior (AP) spine x-ray, and dual-energy x-ray absorptiometry., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Presence or absence of Cataracts at the End-of-Registry ( 5 years) or Early-Termination., Cataracts will be assessed by an ophthalmologist based on a Comprehensive Eye Exam to include the Slit Lamp test., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Presence of Cardiomyopathy at the End-of-Registry ( 5 years) or Early-Termination., Cardiomyopathy will be assessed by echocardiography using transmural strain profile (TMSP) analysis., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Evaluation of Hormonal status based on Cortisol levels at enrollment., Morning cortisol ( before 10 AM) levels will be measured in ug/dl by a Central Lab. The normal range is 3.7-19.4 ug/dl., At enrollment|Presence or absence of Glaucoma at the End-of-Registry ( 5 years) or Early-Termination., Glaucoma will be assessed by an ophthalmologist based on a Comprehensive Eye Exam and testing intraocular pressure., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Number of patients experiencing any Adverse Events (AEs) or Serious Adverse Events (SAEs) after signing the Informed Consent Form., Adverse Events (AEs)/ Serious Adverse Events (SAEs) will be assessed by the current version of the CTCAE at the time the adverse event is identified, and will be summarized by MedDRA system organ class and preferred term using the current version of MedDRA., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Pediatric Quality of Life Inventory Duchenne Muscular Dystrophy (PedsQL DMD) Module at the End-of-Registry ( 5 years) or Early-Termination., PedsQL DMD is self-reported. It consists of 18 items in 4 domains. Daily Activities' (5 items),Treatment' (4 items), 'Worry' (6 items) and 'Communication' (3 items). 5-point response scale to indicate how much of a problem each item has been in the past month (0 = never a problem to 4 = almost always a problem). Items are reverse scored and linearly transformed to a 0-100 scale, so that higher scores indicate better QoL.

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Duchenne Muscular Dystrophy Quality of Life (DMD-QoL)at the End-of-Registry ( 5 years) or Early-Termination., The DMD-QoL is a 14-item questionnaire. The questionnaire is answered using a 4-point response scale (Never, Sometimes, A lot of the time, All of the time). The DMD-QoL has a hierarchical (or 'higher-order') factor structure, with 3 lower-order factors (physical, social, and psychological) and 1 higher-order factor (overall QoL, comprised of the 3 lower-order factors). Higher scores represent a more positive QoL (overall or within each subscale).

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Euro Qol 5 Dimensions (EQ-5D) at the End-of-Registry ( 5 years) or Early-Termination., The EQ-5D-5L (5-Levels) will be completed by patients aged 16 years and older or Parent/guardian. The EQ-5D-5L consists of a descriptive system of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression) and 5 levels (1, no problems; 2, slight problems; 3, moderate problems; 4, severe problems; 5, extreme problems), and a visual analog scale (VAS) from 0 (worst health) to 100 (best health). Patients aged 8 to 15 years will complete the EQ-5D-Y-3L (Youth) questionnaire. The EQ-5D-Y-3L consists of a descriptive system of 5 dimensions and 3 levels (no problems, some problems, and a lot of problems), and a visual analog scale. Parents/legal guardian of patients aged 4 to 7 years will complete the EQ-5D-Y-3L proxy version, which rates how the parent/legally authorized representative rates the health of the child.

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., t enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Patient Reported Outcome Measure for the Upper Limb (PROM UL) at the End-of-Registry ( 5 years) or Early-Termination., The PROM UL consists of 32 items covering four domains of activities of daily living: (1) food, (7 items, max score 14); (2) self-care, (8 items, max score 16); (3) household and environment, (6 items, max score 12); (4) leisure and communication, (11 items, max score 22). For each question, the patients and/or their parents/legal guardian are asked to report their perceived difficulty in performing the activity on a 3-level scale: Cannot do (0); Can do with difficulty (1); Can do easily (2). The maximum total score is 64.

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in North Star Ambulatory Assessment (NSAA) at the End-of-Registry ( 5 years) or Early-Termination., The NSAA is a clinical assessment scale specifically designed to measure functional ability in ambulant male patients. The NSAA consists of 17 scored items and 2 timed tests, including the Time to Run/Walk Test (TTRW) and the Time to Stand Test (TTSTAND).

The TTRW measures the time (in seconds) that it takes the patient to run or walk 10 meters.

The TTSTAND measures the time (in seconds) required for the patient to stand in an erect position from supine (floor).

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.|Change in Performance of Upper Limb (PUL) at the End-of-Registry ( 5 years) or Early-Termination., The PUL module is a tool designed for assessing upper limb function in ambulant and non-ambulant patients. The PUL module consists of 22 items with an entry item to define the starting functional level (which corresponds to the Brooke scale) and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal (i.e., wrist and fingers) level (8 items). For weaker patients, a low score on the entry item (i.e., less than 4 points) means that shoulder-level items do not need to be performed. Each dimension can be scored separately with a maximum score of 16 for the shoulder level, 34 for the elbow level, and 24 for the distal level. A total score can be achieved by adding the three-level scores, with a maximum global score of 74 points.

Change = End-of Registry ( 5 Years) or (early enrollment termination) minus (-) Enrollment., At enrollment, at each Yearly Follow-up Visit (+/- 30 days), and at the End-of-Registry ( 5 years) or Early-Termination.