Individualized trajectories of upper arm disease progression in Duchenne muscular dystrophy patients

Individualized trajectories of upper arm disease progression in Duchenne muscular dystrophy patients - Individualized disease trajectories in DMD

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON52311

Enrollment

Registered

2022-08-19

Start date

2022-10-18

Completion date

Unknown

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne Duchenne muscular dystrophy

Interventions

None listed

Eligibility

Age

2 Years to 17 Years

Inclusion criteria

Inclusion criteria: - Confirmed diagnosis of DMD through genetic testing; - Age five years and older.

Exclusion criteria

Exclusion criteria: - Lack of confirmed mutation in the dystrophin gene or patients with a secondary (neuro)muscular or metabolic disease that affect muscle function; - Intake of investigational medications or exposure to an investigational drug within 6 months prior to the start of the study; - Cognitive problems that would make it difficult to follow directions and participate in testing; - Contraindications to MRI exposure (such as a metal implant).

Design outcomes

Primary

Measure	Time frame
The main study parameters at the four different timepoints (0, 6, 12 and 18 months) are the mFF of the upper limb flexor muscles, measured by qMRI, the muscle force of the upper limb (elbow flexion strength) and upper arm function as measured with the PUL2.0. These datapoints will be used to construct the individual disease trajectories.	—

Secondary

Measure	Time frame
Secondary endpoints are: - The clinical elbow flexion endpoint, defined as the loss of the ability to bring the hand to the mouth with a 200g weighing cup (supporting elbow on table is allowed). - Upper limb strength: shoulder abduction, elbow flexion and extension strength and wrist flexion- and extension strength measured using hand held dynamometry (HHD; N); - Absolute grip strength (kg), pinch strength (kg), measured using MyoGrip and MyoPinch; - The DMD Upper Limb patient*reported outcome measure (PROM; score 0-64); - The PedsQL3.0 Neuromuscular questionnaire (score 0-100);	—

Measure

Time frame

Secondary endpoints are: - The clinical elbow flexion endpoint, defined as the loss of the ability to bring the hand to the mouth with a 200g weighing cup (supporting elbow on table is allowed). - Upper limb strength: shoulder abduction, elbow flexion and extension strength and wrist flexion- and extension strength measured using hand held dynamometry (HHD; N); - Absolute grip strength (kg), pinch strength (kg), measured using MyoGrip and MyoPinch; - The DMD Upper Limb patient*reported outcome measure (PROM; score 0-64); - The PedsQL3.0 Neuromuscular questionnaire (score 0-100);

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Countries

Netherlands

Outcome results