To determine the presence ofneutralizing activity and antibodies against AdenoAssociated Virus Vector serotypes in Patients ofDuchenne Muscular Dystrophy and Wilson’sdisease.
- Conditions
- Health Condition 1: Q688- Other specified congenital musculoskeletal deformities
- Registration Number
- CTRI/2023/09/057504
- Lead Sponsor
- Reliance life sciences
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Yet Recruiting
- Sex
- Not specified
- Target Recruitment
- 0
1. Patients with known diagnosis of Duchenne Muscular Dystrophy (DMD) or Wilson’s
disease
2. Patients who are = 5 years of age
3. Able to understand the study procedures, the risks involved, willing to provide
written Informed Consent. For patients <18 years of age, parents should be willing
to provide written informed consent and subject should be willing to give assent for
the study.
4. Inability to comply to the protocol
5. Patients previously treated with any gene therapy for their disease.
6. Patients lacking mental capacity to consent to the trial
7. Seropositive for HIV/HBsAg / HCV (as per previous reports or test conducted prior
to study enrolment is accepted)
8. Advanced stage of DMD or Wilson’s disease and / or other comorbidities that are
likely influence the interpretation of the current study parameter.
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To investigate the seroprevalence of AAV neutralizing factors and antibodies in <br/ ><br>patients of Duchenne Muscular Dystrophy and Wilsons disease.Timepoint: Day 0
- Secondary Outcome Measures
Name Time Method To collect the subject samples for biobanking & utilize these samples for the various <br/ ><br>evaluations / tests related to diagnosis or therapy of DMD & Wilson’s disease.Timepoint: Day 0 & future research.;To describe demographic & disease profile of patients of DMD & Wilson’s disease <br/ ><br>who are having preexisting anti AAV AntibodiesTimepoint: Day 0