Condition (MeSH)
Homocystinuria
ClinicalTrials.gov lists 20 trials for Homocystinuria or a narrower condition beneath it. Travere Therapeutics, Inc. has the most among industry and government lead sponsors, with 4.
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Trials per phase
20 of the 20 trials are tagged with Homocystinuria itself; the rest are tagged with a narrower condition beneath it.
When did Homocystinuria trials start? (12)
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 1/2
- Phase 2
- Phase 3
- Not applicable
- Observational
Top of scale: 4
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 |
|---|---|
| 2025 | 1 |
| 2024 | 4 |
| 2023 | 1 |
| 2022 | 4 |
| 2021 | 2 |
| 2019 | 2 |
| 2018 | 1 |
| 2017 | 1 |
| 2015 | 1 |
| 2010 | 1 |
Showing 10 of 12 earlier years.
No forecast is made for this condition: it averages 2.0 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 Homocystinuria trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 4 |
| Phase 2 | 2 |
| Phase 3 | 2 |
| Not applicable | 3 |
| Observational | 8 |
How many Homocystinuria trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 3 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 2 |
| Completed | 9 |
| Suspended | 1 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Homocystinuria trials? (6)
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 |
|---|---|
| Travere Therapeutics, Inc. | 4 |
| Aeglea Biotherapeutics | 1 |
| Hamad Medical Corporation | 1 |
| King Fahad Armed Forces Hospital | 1 |
| National Center for Research Resources (NCRR) | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Homocystinuria? (5)
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 |
|---|---|
| Pegtibatinase | 3 |
| Taurine | 1 |
| Betaine | 1 |
| cysteine hydrochloride | 1 |
| Creatine | 1 |
Interventions as named in the registry (5)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| N Acetylcysteine | 2 |
| Pegtarviliase IV | 1 |
| Pegtarviliase SC | 1 |
| SYNB1353 | 1 |
| A3: Acetaminophen (Tylenol caplets®, from Janssen-Cilag, S.A. de C.V.) | 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 publications cover Homocystinuria? (18)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Efficacy and pharmacokinetics of betaine in CBS and cblC deficiencies: a cross-over randomized controlled trial. | Orphanet journal of rare diseases | 2022 | Yes |
| The determinants of maternal homocysteine in pregnancy: findings from the Ottawa and Kingston Birth Cohort. | Public health nutrition | 2020 | Yes |
| Spectrum of MMACHC mutations in Italian and Portuguese patients with combined methylmalonic aciduria and homocystinuria, cblC type. | Molecular genetics and metabolism | 2008 | Yes |
| Assessment of visual function in children with methylmalonic aciduria and homocystinuria. | Neuropediatrics | 2005 | Yes |
| Cerebral edema associated with betaine treatment in classical homocystinuria. | The Journal of pediatrics | 2004 | Yes |
Showing 5 of 18 publications.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: serious adverse events in this condition's trials, prevalence, rare-disease identity, and sub-conditions in mesh.