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NCT03233841 · ClinicalTrials.gov registry record
Farber Disease Natural History Study
A clinical trial, sponsored by Sumitomo Pharma Switzerland.
- Completed
- Registry status
- 45
- Enrollment target
NCT03233841: Completed study, sponsored by Sumitomo Pharma Switzerland.
NCT03233841 is a clinical trial that has completed, run by Sumitomo Pharma Switzerland. The registered enrollment target is 45 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03233841 has completed, sponsored by Sumitomo Pharma Switzerland.
- COMPLETED
- Registry status
- 45 participants
- Enrollment target
Study Summary
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber disease. All patients diagnosed with Farber disease are eligible, including both those who have and have not undergone hematopoietic stem cell transplantation (HSCT). Additionally, data and records from deceased patients will provide valuable retrospective data for this study. The secondary objective of the study is to establish a set of clinical data, laboratory data (biomarkers), and functional data potentially useful for: * Assessing the efficacy of HSCT and the efficacy of potential future therapies (for example with RVT-801, recombinant human acid ceramidase) in Farber disease * Characterizing changes in symptoms of patients over time * Characterizing distinct groups (phenotypes) within the patient population * Documenting the disease histories of individual patients to serve as intra-subject control data for those who may enroll in any future clinical studies with therapies for Farber disease The exploratory objectives of the study are: * To explore the relationship between patient disease activity or phenotype and specific ceramide levels or specific immunologic markers (cytokines/chemokines) in blood * To evaluate a standardized tool, the Farber Disease Natural History Instrument (FDNI), to be used for the collection of patient history information, data from clinical, laboratory, genetic, and functional studies, and data from review of medical records
Primary Outcome
Collection of information for all subjects will include data from: * Medical history * Farber disease diagnosis, presentation, treatments and symptom progression Collection of information from living subjects will include: * Medical examination * Disease-specific data (Farber Disease Natural History Instrument - FDNI) * Laboratory tests (laboratory assessments and inflammatory markers) * Functional tests * Six-minute walk test (6MWT) * Pulmonary function testing * Additional assessments
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 45 participants |
| Start Date | 2017-11-22 |
| Est. Completion | 2019-12-09 |
What the finished NCT03233841 record still lists
NCT03233841 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 45 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT03233841 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03233841 about?
NCT03233841 is a clinical study titled "Farber Disease Natural History Study". The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber disease. All patients diagnosed with Farber disease are eligible, includi...
What is the current status of trial NCT03233841?
This trial is currently completed. The enrollment target is 45 participants. The study started on 2017-11-22. Estimated completion is 2019-12-09.
Who is sponsoring clinical trial NCT03233841?
This trial is sponsored by Sumitomo Pharma Switzerland, which has 13 total clinical trials registered on ClinicalTrials.gov.
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