- Conditions
- Hematopoietic and Stem Cell Transplant
- Interventions
- General Health Module-Baseline Adolescent-Self Report (13-18 years), General Health Module-Follow Up Adolescent-Self Report (13-18 years), General Health Module-Baseline Parent Report-Adolescent (13-18 years), General Health Module-Follow Up Parent Report-Adolescent (13-18 years), General Health Module-Baseline Parent Report-School Age (5-12 years), General Health Module-Follow Up Parent Report-School Age (5-12 years), Demographic Data Form, SCT Daily Activity Log, The Functional Independence Measure for Children, Manual Muscle Testing, 3-Minute Step Test, HSCT Module-Follow Up Adolescent-Self Report (13-18 years), HSCT Module-Follow Child-Self Report (5-12 years), HSCT Module-Follow Up Parent Report-adolescent (13-18 years), HSCT Module-Follow Up Parent Report-School Age (5-12 years)
- Other
- Lead sponsor
- Washington University School of Medicine
- Other
- Eligibility
- 7 Years to 17 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
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Showing 1–4
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matching trials from the live ClinicalTrials.gov search.
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- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Interventions
- AAV9-GLB1, Abdominal ultrasound, Rituximab, Sirolimus, Methylprednisolone, Prednisone, Audiology assessment with ABR, Bone density scan (DEXA), Electrocardiogram (EKG), Echocardiogram, Electroencephalogram (EEG) awake and extended overnight, Laboratory tests, Lumbar puncture, Brain MRI/MRS/fMRI, Neurocognitive testing, Neurology exam, PICC or other Central line placement, Skeletal survey, Skin biopsy, Speech and modified barium swallow study, Ophthalmology exam
- Biological · Procedure · Drug + 3 more
- Lead sponsor
- National Human Genome Research Institute (NHGRI)
- NIH
- Eligibility
- 6 Months to 12 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2028
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- Conditions
- Aggression, Self-Injurious Behavior
- Interventions
- Functional Communication Training
- Behavioral
- Lead sponsor
- University of Nebraska
- Other
- Eligibility
- 3 Years to 18 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Omaha, Nebraska
- Conditions
- Spina Bifida
- Interventions
- Altimate Medical EasyStand Bantam
- Device
- Lead sponsor
- University of St. Augustine for Health Sciences
- Other
- Eligibility
- 5 Years to 12 Years
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- San Diego, California