Condition (MeSH)
Bardet-Biedl Syndrome
ClinicalTrials.gov lists 18 trials for Bardet-Biedl Syndrome or a narrower condition beneath it. Rhythm Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 4.
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Trials per phase
18 of the 18 trials are tagged with Bardet-Biedl Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Bardet-Biedl Syndrome trials start? (10)
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 2
- Phase 3
- Phase 4
- 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 |
| 2023 | 2 |
| 2022 | 2 |
| 2021 | 2 |
| 2020 | 1 |
| 2018 | 3 |
| 2014 | 2 |
| 2011 | 1 |
| 2004 | 1 |
| 2003 | 1 |
No forecast is made for this condition: it averages 1.6 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial 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 Bardet-Biedl Syndrome trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 2 | 1 |
| Phase 3 | 3 |
| Phase 4 | 1 |
| Not applicable | 2 |
| Observational | 10 |
| Expanded access | 1 |
How many Bardet-Biedl Syndrome trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 6 |
| Active, not recruiting | 1 |
| Completed | 7 |
| Approved for marketing | 1 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Bardet-Biedl Syndrome trials? (3)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Rhythm Pharmaceuticals, Inc. | 4 |
| National Human Genome Research Institute (NHGRI) | 1 |
| Rolfs Consulting und Verwaltungs-GmbH (RCV) | 1 |
Which drugs are being tested for Bardet-Biedl Syndrome? (1)
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 |
|---|---|
| Setmelanotide | 4 |
Interventions as named in the registry (4)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Setmelanotide, administered subcutaneously [SC], once daily. | 1 |
| BLOOD SAMPLE | 1 |
| 1% Metformin | 1 |
| Urine sample | 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.
Which serious adverse events do Bardet-Biedl Syndrome trials report? (4)
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 |
|---|---|---|
| Anaemia | 1 | 1 |
| Anaphylactic reaction | 1 | 1 |
| Blindness | 1 | 1 |
| Suicidal ideation | 0 | 1 |
Which publications cover Bardet-Biedl Syndrome? (22)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Healthcare needs, care use and health status outcomes in adults with Bardet-Biedl syndrome: a cross-sectional study in Norway. | BMJ open | 2025 | Yes |
| Bardet-Biedl syndrome improved diagnosis criteria and management: Inter European Reference Networks consensus statement and recommendations. | European journal of human genetics : EJHG | 2024 | Yes |
| Ultrasound evaluation of kidney and liver involvement in Bardet-Biedl syndrome. | Orphanet journal of rare diseases | 2024 | Yes |
| Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet-Biedl syndrome: phase 3 trial results. | Orphanet journal of rare diseases | 2023 | Yes |
| Interview-Based Patient- and Caregiver-Reported Experiences of Hunger and Improved Quality of Life with Setmelanotide Treatment in Bardet-Biedl Syndrome. | Advances in therapy | 2023 | Yes |
Showing 5 of 22 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Bardet-Biedl syndrome | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Bardet-Biedl syndrome | Point prevalence | 1-9 / 100 000 | United States | US | Exact |
| Bardet-Biedl syndrome | Point prevalence | 1-9 / 100 000 | United States | US | Unspecified |
| Bardet-Biedl syndrome | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Unspecified |
| Bardet-Biedl syndrome | Prevalence at birth | 1-9 / 1 000 000 | Europe | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Bardet-Biedl syndrome | 110 | Exact | Orphadata Science knowledge files, Product 1 |
| Bardet-Biedl syndrome | 110 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.