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NCT06552052 · ClinicalTrials.gov registry record
A Retrospective and Prospective Natural History of Genetic Vasculopathies
A clinical trial of Multisystemic Smooth Muscle Dysfunction Syndrome and ACTA2, sponsored by Massachusetts General Hospital.
- Recruiting
- Registry status
- 50
- Enrollment target
- 1
- Study location
NCT06552052: Recruiting study of Multisystemic Smooth Muscle Dysfunction Syndrome and ACTA2, sponsored by Massachusetts General Hospital.
NCT06552052 is a study of Multisystemic Smooth Muscle Dysfunction Syndrome and ACTA2 that is actively recruiting participants, run by Massachusetts General Hospital. The registered enrollment target is 50 participants. The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06552052, a study of Multisystemic Smooth Muscle Dysfunction Syndrome and ACTA2, is actively recruiting participants, sponsored by Massachusetts General Hospital.
- RECRUITING
- Registry status
- 50 participants
- Enrollment target
- 1
- Study location
Study Summary
This study will combine retrospective review of medical records from patients with ACTA2 and ongoing collection of clinical data using standardized instruments and intervals on an observational basis from patients with ACTA2. Patients in cohorts 1-3 will be asked to attend clinic visits in person per the schedule of events. At minimum, the medical records of patients with ACTA2 will be reviewed to record data on aspects of the disease, including disease characteristics and developmental milestones. The study is planned to enroll a total of 100 patients: 7 in cohort 1, 7 in cohort 2, and the remaining in cohorts 3 and 4. This study is planned to study patients for at least 3 years with the option to continue as long as possible for assessment of disease progression. During their continued study participation, as patients age, they may move into the next cohort. Beyond 3 years the duration of the study with be determined by availability of funding from sponsors.
Primary Outcome
1.1 Develop patient surveys and identify retrospective/existing clinical data sources for aggregation, harmonization, and analyses of outcomes and biomarkers to be compared to existing published data. 1.2. Identify modifiers of symptom progression in patients with MSMDS. 1.3 Correlate the degree of disease progression by systems in 40 patients (cross-sectional study) and construct an MSMDS Rating Scale to evaluate disease severity and progression in children. 1.4 Create case report forms based o
Conditions Studied
Study Locations (1)
Massachusetts
- Massachusetts General Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 50 participants |
| Start Date | 2024-05-08 |
| Est. Completion | 2028-07 |
What NCT06552052 shows while recruiting
NCT06552052 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 50 participants, a relatively small participant target.
The record links to 2 conditions, with Multisystemic Smooth Muscle Dysfunction Syndrome appearing as the primary indexed condition, and to 0 interventions.
NCT06552052 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT06552052 about?
NCT06552052 is a clinical study titled "A Retrospective and Prospective Natural History of Genetic Vasculopathies". This study will combine retrospective review of medical records from patients with ACTA2 and ongoing collection of clinical data using standardized instruments and intervals on an observational basis from patients with ACTA2. Patients in cohorts 1-3 will be asked to attend clinic visits in person p...
What is the current status of trial NCT06552052?
This trial is currently recruiting. The enrollment target is 50 participants. The study started on 2024-05-08. Estimated completion is 2028-07.
What conditions does trial NCT06552052 study?
This clinical trial studies the following conditions: Multisystemic Smooth Muscle Dysfunction Syndrome, ACTA2.
Who is sponsoring clinical trial NCT06552052?
This trial is sponsored by Massachusetts General Hospital, which has 2,032 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06552052 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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