Condition (MeSH)
Dysautonomia, Familial
ClinicalTrials.gov lists 14 trials for Dysautonomia, Familial or a narrower condition beneath it.
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Trials per phase
14 of the 14 trials are tagged with Dysautonomia, Familial itself; the rest are tagged with a narrower condition beneath it.
When did Dysautonomia, Familial trials start? (9)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 2
- Phase 3
- Not applicable
- Observational
Top of scale: 3
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2024 | 2 |
| 2019 | 1 |
| 2017 | 1 |
| 2016 | 2 |
| 2015 | 2 |
| 2014 | 1 |
| 2013 | 1 |
| 2011 | 1 |
| 2009 | 3 |
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 2 trials still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Dysautonomia, Familial trials in? (5)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 2 | 5 |
| Phase 3 | 2 |
| Not applicable | 4 |
| Observational | 2 |
How many Dysautonomia, Familial trials are recruiting? (5)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 2 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 1 |
| Completed | 8 |
| Withdrawn | 2 |
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which drugs are being tested for Dysautonomia, Familial? (4)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| DEXMEDETOMIDINE | 1 |
| Dronabinol | 1 |
| Carbidopa | 1 |
| Albuterol | 1 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Ipratropium HFA | 1 |
| Dexmedetomidine Sublingual | 1 |
| Carbidopa High-Dose | 1 |
| Carbidopa Low-Dose | 1 |
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
How do Dysautonomia, Familial trials deliver against plan? (1)
Shown in this order: one row, this condition's own record. Enrollment hit target: the share of trials mapped to this condition whose actual enrollment reached the planned enrollment. On time: the share whose actual primary completion date was no later than the planned date. Each share counts only trials where we observed an estimated figure before the actual one, and n is that number of trials. Below 5 trials the count is shown as a fraction, not a percentage.
| Enrollment | Timeline |
|---|---|
| 29% hit target (n=7); 7 of 14 trials not graded | 50% on time (n=6); 8 of 14 trials not graded |
Delivery grades from ClinicalTrials.gov / AACT (CTTI) planned-vs-actual history, public domain.
Which serious adverse events do Dysautonomia, Familial trials report? (2)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Hospitaliztion | 1 | 1 |
| lightheadedness / faint | 1 | 1 |
Which publications cover Dysautonomia, Familial? (10)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Current treatments in familial dysautonomia. | Expert opinion on pharmacotherapy | 2014 | Yes |
| Developmental abnormalities, blood pressure variability and renal disease in Riley Day syndrome. | Journal of human hypertension | 2013 | Yes |
| Cyclic vomiting associated with excessive dopamine in Riley-day syndrome. | Journal of clinical gastroenterology | 2013 | Yes |
| Hyperdopaminergic crises in familial dysautonomia: a randomized trial of carbidopa. | Neurology | 2013 | Yes |
| Can loss of muscle spindle afferents explain the ataxic gait in Riley-Day syndrome? | Brain : a journal of neurology | 2011 | Yes |
Showing 5 of 10 publications.
Prevalence (6)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Familial dysautonomia | Point prevalence | <1 / 1 000 000 | Europe | Not stated | Exact |
| Familial dysautonomia | Point prevalence | <1 / 1 000 000 | Europe | Not stated | Exact |
| Familial dysautonomia | Prevalence at birth | 1-5 / 10 000 | Specific population | Not stated | Exact |
| Familial dysautonomia | Prevalence at birth | 1-5 / 10 000 | Specific population | Not stated | Unspecified |
| Familial dysautonomia | Point prevalence | <1 / 1 000 000 | Europe | Not stated | Unspecified |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Familial dysautonomia | 1764 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Familial dysautonomia | 1764 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Checked, with nothing recorded for this condition: lead sponsors and sub-conditions in mesh.