Studies
| Study First Submitted Date | 2023-01-04 |
| Study First Posted Date | 2023-01-13 |
| Last Update Posted Date | 2023-01-13 |
| Start Month Year | December 20, 2022 |
| Primary Completion Month Year | December 2024 |
| Verification Month Year | January 2023 |
| Verification Date | 2023-01-31 |
| Last Update Posted Date | 2023-01-13 |
Detailed Descriptions
| Sequence: | 20696664 |
| Description | This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD). Information collected in this study may be used to identify potential participants for DMD investigational gene therapy clinical trials. This study consists of: A phone/video interview to provide e-consent and medical history A single home health visit to collect blood sample for antibody testing A phone/video call for communication of AAV8 antibody test results |
Facilities
| Sequence: | 199783312 |
| Status | Recruiting |
| Name | Rare Disease Research |
| City | Atlanta |
| State | Georgia |
| Zip | 30329 |
| Country | United States |
Facility Contacts
| Sequence: | 28074790 | Sequence: | 28074791 |
| Facility Id | 199783312 | Facility Id | 199783312 |
| Contact Type | primary | Contact Type | backup |
| Name | Mandy Fruscione, PharmD | Name | Marcial Almaraz |
| [email protected] | [email protected] | ||
| Phone | 678-883-6897 | ||
Facility Investigators
| Sequence: | 18311120 |
| Facility Id | 199783312 |
| Role | Principal Investigator |
| Name | Han Phan, MD |
Conditions
| Sequence: | 52103700 |
| Name | Duchenne Muscular Dystrophy |
| Downcase Name | duchenne muscular dystrophy |
Id Information
| Sequence: | 40105753 |
| Id Source | org_study_id |
| Id Value | RGX-202-0101 |
Countries
| Sequence: | 42508278 |
| Name | United States |
| Removed | False |
Interventions
| Sequence: | 52417714 |
| Intervention Type | Diagnostic Test |
| Name | AAV8 DetectCDx |
| Description | An in vitro diagnostic assay to detect antibodies to AAV8 in human serum specimens. |
Keywords
| Sequence: | 79760819 | Sequence: | 79760820 | Sequence: | 79760821 |
| Name | DMD | Name | Duchenne Muscular Dystrophy | Name | Duchenne |
| Downcase Name | dmd | Downcase Name | duchenne muscular dystrophy | Downcase Name | duchenne |
Design Outcomes
| Sequence: | 177144453 | Sequence: | 177144454 |
| Outcome Type | primary | Outcome Type | secondary |
| Measure | Prevalence of anti-AAV8 antibodies in patients with DMD | Measure | Prevalence of anti-AAV9 antibodies in patients with DMD |
| Time Frame | 90 days | Time Frame | 90 days |
| Description | To evaluate the prevalence of AAV8 antibodies in patients with DMD To identify participants who may be eligible for investigational gene therapy clinical trials in males with DMD | Description | • To evaluate the prevalence of AAV9 antibodies in patients with DMD |
Browse Conditions
| Sequence: | 193221857 | Sequence: | 193221858 | Sequence: | 193221859 | Sequence: | 193221860 | Sequence: | 193221861 | Sequence: | 193221862 | Sequence: | 193221863 | Sequence: | 193221864 | Sequence: | 193221865 |
| Mesh Term | Muscular Dystrophies | Mesh Term | Muscular Dystrophy, Duchenne | Mesh Term | Muscular Disorders, Atrophic | Mesh Term | Muscular Diseases | Mesh Term | Musculoskeletal Diseases | Mesh Term | Neuromuscular Diseases | Mesh Term | Nervous System Diseases | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Genetic Diseases, X-Linked |
| Downcase Mesh Term | muscular dystrophies | Downcase Mesh Term | muscular dystrophy, duchenne | Downcase Mesh Term | muscular disorders, atrophic | Downcase Mesh Term | muscular diseases | Downcase Mesh Term | musculoskeletal diseases | Downcase Mesh Term | neuromuscular diseases | Downcase Mesh Term | nervous system diseases | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | genetic diseases, x-linked |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48257442 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | REGENXBIO Inc. |
Central Contacts
| Sequence: | 11994274 |
| Contact Type | primary |
| Name | Patient Advocacy |
| Phone | 866-860-0117 |
| [email protected] | |
| Role | Contact |
Eligibilities
| Sequence: | 30726792 |
| Sampling Method | Probability Sample |
| Gender | Male |
| Minimum Age | 0 Years |
| Maximum Age | 11 Years |
| Healthy Volunteers | No |
| Population | Primary care clinic, Musculoskeletal care, Neuromusculoskeletal care |
| Criteria | Inclusion Criteria: Males at least 0 to <12 years of age Diagnosis of DMD Provision of signed and dated informed consent form (ICF) and assent as required per local regulations or requirements Exclusion Criteria: Prior participation in a gene therapy trial OR recipient of a gene therapy drug Other inclusion/exclusion criteria apply |
| Gender Based | True |
| Adult | False |
| Child | True |
| Older Adult | False |
Calculated Values
| Sequence: | 253978116 |
| Number Of Facilities | 1 |
| Registered In Calendar Year | 2023 |
| Were Results Reported | False |
| Has Us Facility | True |
| Has Single Facility | True |
| Minimum Age Num | 0 |
| Maximum Age Num | 11 |
| Minimum Age Unit | Years |
| Maximum Age Unit | Years |
| Number Of Primary Outcomes To Measure | 1 |
| Number Of Secondary Outcomes To Measure | 1 |
Designs
| Sequence: | 30473224 |
| Observational Model | Cohort |
| Time Perspective | Prospective |
Responsible Parties
| Sequence: | 28839645 |
| Responsible Party Type | Sponsor |