Economic evaluation of a therapy for children with Muscular Dystrophy.
Not Applicable
- Conditions
- Health Condition 1: G710- Muscular dystrophy
- Registration Number
- CTRI/2021/06/034274
- Lead Sponsor
- Dr Amarjeet Singh
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
Inclusion Criteria
(1)Families with affected boys with clinical symptoms suggestive of DMD and increased creatine kinase (CK) levels (CK >1500IU)
(2)Boys between 5-15 years of age.
(3)Laboratory confirmation:
oGenetic confirmation by Multiplex Ligation-Dependent Probe Amplification (MLPA)/ Polymerase Chain Reaction (PCR)/ Next-Generation Sequencing (NGS)/SANGER
or
oMuscle biopsy (showing absence of dystrophin expression genetically).
(4)Caregiver having smart phone or internet facility.
Exclusion Criteria
1.Subjects/families who are unwilling to participate or unable to comply with the protocol study procedures or visits
2.Consent or assent not given.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1.Change in quality of life <br/ ><br>2. Change in Out of Pocket expenditure incurredTimepoint: 6 months, 1year
- Secondary Outcome Measures
Name Time Method 1.Socio-economic determinants of the disease.Timepoint: Single (at the time of recruitment);2.Feasibility of tele follow-upTimepoint: 1year;3.Neuro-muscular quality of life in pediatric age group.Timepoint: three data points: <br/ ><br>1. Recruitment <br/ ><br>2.End of Phase-1 (6month) <br/ ><br>3.End of Phase-2 (1 year);4.Pattern of health care utilization for different stage of diseaseTimepoint: Single (at the time of recruitment)