Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04828863 · ClinicalTrials.gov registry record
Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)
A clinical trial, sponsored by Children's Hospital of Philadelphia.
- Completed
- Registry status
- 44
- Enrollment target
NCT04828863: Completed study, sponsored by Children's Hospital of Philadelphia.
NCT04828863 is a clinical trial that has completed, run by Children's Hospital of Philadelphia. The registered enrollment target is 44 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04828863 has completed, sponsored by Children's Hospital of Philadelphia.
- COMPLETED
- Registry status
- 44 participants
- Enrollment target
Study Summary
Maple Syrup Urine Disease (MSUD) is a disorder of protein metabolism that leads to neurological differences. It is an exciting time where people diagnosed with MSUD are living longer. The investigators want to learn about how adults with MSUD think, feel, and live. The purpose of this research study is to 1) look at thinking skills, behavior skills, life skills, and quality of life in adults with MSUD and 2) measure how medical and personal factors impact these areas. The results of the study will be used to learn how to best help adults with MSUD and how to prepare for their success. In this study, the participants will answer questions on thinking, life skills, behavior skills, and quality of life. These questions will be completed on the internet. The participants will have a formal testing of their thinking and behavior completed virtually. The participants may also have a 1-2-hour telephone conversation about their life. All tests will occur virtually at home. The investigators will send the participants a letter with the results of thinking and behavior tests.
Primary Outcome
The Behavior Rating Inventory of Executive Function for Adults will be completed, scored, and standardized to age-matched norms.
Interventions
- BEHAVIORAL Neurocognitive testing
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 44 participants |
| Start Date | 2021-06-12 |
| Est. Completion | 2023-11-30 |
What the finished NCT04828863 record still lists
NCT04828863 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 44 participants, a relatively small participant target.
The record links to 0 conditions, and to 1 intervention - of which Neurocognitive testing is the first listed.
NCT04828863 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04828863 about?
NCT04828863 is a clinical study titled "Neurocognitive Outcomes and Quality of Life in Adults With Maple Syrup Urine Disease (MSUD)". Maple Syrup Urine Disease (MSUD) is a disorder of protein metabolism that leads to neurological differences. It is an exciting time where people diagnosed with MSUD are living longer. The investigators want to learn about how adults with MSUD think, feel, and live. The purpose of this research study...
What is the current status of trial NCT04828863?
This trial is currently completed. The enrollment target is 44 participants. The study started on 2021-06-12. Estimated completion is 2023-11-30.
What interventions are being tested in trial NCT04828863?
The interventions under investigation include: Neurocognitive testing (BEHAVIORAL).
Who is sponsoring clinical trial NCT04828863?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02323945 · 44 participants · NA
Effects of Breathing Mild Bouts of Low Oxygen on Limb Mobility After Spinal Injury
- NCT03093909 · 44 participants · Phase 1
Study of Aerosol Gemcitabine in Patients With Solid Tumors and Pulmonary Metastases
- NCT03104491 · 44 participants · Phase 1
Inotuzumab Ozogamicin Post-Transplant For Acute Lymphocytic Leukemia
- NCT03781765 · 44 participants · Phase 4
Stimulant vs. Non-stimulant Treatments and Reward Processing in Drug-naive Youth at SUD Risk
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI