Condition (MeSH)
Proteostasis Deficiencies
ClinicalTrials.gov lists 1,824 trials for Proteostasis Deficiencies or a narrower condition beneath it. Alnylam Pharmaceuticals has the most among industry and government lead sponsors, with 20.
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Trials per phase
2 more sections with rows
5 of the 1,824 trials are tagged with Proteostasis Deficiencies itself; the rest are tagged with a narrower condition beneath it.
When did Proteostasis Deficiencies trials start? (33)
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. 4 trials are registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
- Other (Expanded access, Unknown)
Top of scale: 146
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 |
|---|---|
| 2026 (partial year) | 134 |
| 2025 | 146 |
| 2024 | 137 |
| 2023 | 132 |
| 2022 | 103 |
| 2021 | 133 |
| 2020 | 107 |
| 2019 | 97 |
| 2018 | 84 |
| 2017 | 84 |
Showing 10 of 33 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 150 trials starting in 2026 and about 150 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 13 percent and ran about 12 percent low against the count later registered; forecasts made before the year began were off by about 19 percent and ran about 17 percent low against the count later registered, and had about the same error as repeating last year's count. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 36 months from start to primary completion across 248 trials still running at the 2026-10-09 snapshot; registered median of 28 months across 938 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Proteostasis Deficiencies trials in? (10)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 214 |
| Phase 1/2 | 112 |
| Phase 2 | 312 |
| Phase 2/3 | 45 |
| Phase 3 | 126 |
| Phase 4 | 28 |
| Not applicable | 411 |
| Observational | 556 |
| Expanded access | 19 |
| Unknown | 1 |
How many Proteostasis Deficiencies trials are recruiting? (13)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 93 |
| Recruiting | 363 |
| Enrolling by invitation | 38 |
| Active, not recruiting | 99 |
| Available | 4 |
Showing 5 of 13 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Proteostasis Deficiencies trials? (4)
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 |
|---|---|
| Alnylam Pharmaceuticals | 20 |
| National Institute of Neurological Disorders and Stroke (NINDS) | 15 |
| GlaxoSmithKline | 12 |
| Pfizer | 12 |
Which drugs are being tested for Proteostasis Deficiencies? (7)
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 |
|---|---|
| Melphalan | 32 |
| Cytoxan | 31 |
| Riluzole | 25 |
| THALIDOMIDE | 10 |
| POMALIDOMIDE | 10 |
Showing 5 of 7 drugs.
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Dexamethasone/Dexamethasone acetate | 59 |
| Bortezomib SC | 28 |
| Filgrastime (G-CSF) | 19 |
| Peripheral blood stem cell transplantation | 16 |
| Daratumumab SC | 16 |
Showing 5 of 8 interventions.
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 Proteostasis Deficiencies trials report? (10)
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 |
|---|---|---|
| Cardiac failure | 812 | 28 |
| Pneumonia | 558 | 79 |
| Respiratory failure | 408 | 58 |
| Overnight hospitalization | 334 | 1 |
| Cardiac failure congestive | 304 | 21 |
Showing 5 of 10 adverse events.
Which publications cover Proteostasis Deficiencies? (12)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Rescue by 4-phenylbutyrate of several misfolded creatine transporter-1 variants linked to the creatine transporter deficiency syndrome. | Neuropharmacology | 2019 | Yes |
| X-Linked Spinal and Bulbar Muscular Atrophy: From Clinical Genetic Features and Molecular Pathology to Mechanisms Underlying Disease Toxicity. | Advances in experimental medicine and biology | 2018 | Yes |
| In vitro studies in VCP-associated multisystem proteinopathy suggest altered mitochondrial bioenergetics. | Mitochondrion | 2015 | Yes |
| Non-enzymatic post-translational protein modifications and proteostasis network deregulation in carcinogenesis. | Journal of proteomics | 2013 | Yes |
| Disorders of protein misfolding: alpha-1-antitrypsin deficiency as prototype. | The Journal of pediatrics | 2013 | Yes |
Showing 5 of 12 publications.
Sub-conditions in MeSH (3)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.