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Condition (MeSH)

Brain Diseases, Metabolic

ClinicalTrials.gov lists 1,515 trials for Brain Diseases, Metabolic or a narrower condition beneath it. Genzyme, a Sanofi Company has the most among industry and government lead sponsors, with 67.

MeSH D001928 Nervous System Diseases C10 C18
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Phase 1 Phase 1 126 Phase 1/2 Phase 1/2 107 Phase 2 Phase 2 171 Phase 2/3 Phase 2/3 30 Phase 3 Phase 3 138 Phase 4 Phase 4 98 Not applicable Not applicable 255 Observational Observational 565 Phase 1 Phase 1 126 Phase 1/2 Phase 1/2 107 Phase 2 Phase 2 171 Phase 2/3 Phase 2/3 30 Phase 3 Phase 3 138 Phase 4 Phase 4 98 Not applicable Not applicable 255 Observational Observational 565

Trials per phase

1 more section with rows

19 of the 1,515 trials are tagged with Brain Diseases, Metabolic itself; the rest are tagged with a narrower condition beneath it.

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The PDF carries up to 50 rows of each section, where the page shows the first few, with the sources listed. Enter a work email and confirm it from the message we send, and the report follows by email.

When did Brain Diseases, Metabolic trials start? (37)

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. 8 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)

Top of scale: 101

1990: 1 trials (Phase 1/2 1) 1991: 2 trials (Observational 2) 1992: 1 trials (Phase 3 1) 1993: 4 trials (Observational 1, Phase 4 1, Phase 2 1, Phase 1 1) 1994: 4 trials (Observational 1, Phase 3 1, Phase 2 2) 1995: 7 trials (Observational 3, Not applicable 2, Phase 2/3 1, Phase 1 1) 1996: 1 trials (Phase 2 1) 1997: 2 trials (Observational 1, Phase 4 1) 1998: 4 trials (Not applicable 1, Phase 2 2, Phase 1 1) 1999: 7 trials (Observational 2, Phase 3 1, Phase 2 2, Phase 1 2) 2000: 5 trials (Observational 2, Not applicable 1, Phase 3 1, Phase 2 1) 2001: 10 trials (Observational 6, Phase 4 3, Phase 2 1) 2002: 12 trials (Observational 3, Not applicable 2, Phase 3 1, Phase 2 5, Phase 1 1) 2003: 13 trials (Other 1, Observational 2, Phase 4 1, Phase 3 1, Phase 2/3 1, Phase 2 5, Phase 1/2 1, Phase 1 1) 2004: 16 trials (Observational 6, Not applicable 1, Phase 4 3, Phase 3 1, Phase 2 4, Phase 1/2 1) 2005: 23 trials (Observational 6, Not applicable 2, Phase 4 2, Phase 3 7, Phase 2/3 1, Phase 2 3, Phase 1 2) 2006: 26 trials (Observational 6, Not applicable 2, Phase 4 2, Phase 3 4, Phase 2 9, Phase 1 3) 2007: 37 trials (Observational 12, Not applicable 7, Phase 4 2, Phase 3 4, Phase 2/3 1, Phase 2 8, Phase 1/2 2, Phase 1 1) 2008: 42 trials (Observational 11, Not applicable 3, Phase 4 7, Phase 3 8, Phase 2 8, Phase 1/2 1, Phase 1 4) 2009: 48 trials (Observational 15, Not applicable 6, Phase 4 3, Phase 3 7, Phase 2/3 2, Phase 2 7, Phase 1/2 2, Phase 1 6) 2010: 51 trials (Observational 23, Not applicable 8, Phase 4 6, Phase 3 3, Phase 2/3 1, Phase 2 4, Phase 1/2 3, Phase 1 3) 2011: 59 trials (Observational 21, Not applicable 6, Phase 4 2, Phase 3 10, Phase 2/3 1, Phase 2 10, Phase 1/2 3, Phase 1 6) 2012: 56 trials (Observational 21, Not applicable 8, Phase 4 7, Phase 3 3, Phase 2 8, Phase 1/2 6, Phase 1 3) 2013: 54 trials (Observational 12, Not applicable 15, Phase 4 7, Phase 3 6, Phase 2/3 2, Phase 2 2, Phase 1/2 3, Phase 1 7) 2014: 58 trials (Observational 21, Not applicable 9, Phase 4 4, Phase 3 2, Phase 2/3 1, Phase 2 12, Phase 1/2 6, Phase 1 3) 2015: 65 trials (Observational 29, Not applicable 7, Phase 4 7, Phase 3 3, Phase 2/3 3, Phase 2 4, Phase 1/2 2, Phase 1 10) 2016: 50 trials (Observational 24, Not applicable 8, Phase 4 3, Phase 3 1, Phase 2/3 4, Phase 2 2, Phase 1/2 3, Phase 1 5) 2017: 57 trials (Observational 21, Not applicable 16, Phase 4 1, Phase 3 4, Phase 2/3 1, Phase 2 3, Phase 1/2 5, Phase 1 6) 2018: 76 trials (Observational 32, Not applicable 12, Phase 4 5, Phase 3 12, Phase 2 8, Phase 1/2 4, Phase 1 3) 2019: 97 trials (Observational 41, Not applicable 21, Phase 4 2, Phase 3 5, Phase 2/3 1, Phase 2 8, Phase 1/2 11, Phase 1 8) 2020: 74 trials (Observational 29, Not applicable 12, Phase 4 6, Phase 3 4, Phase 2/3 2, Phase 2 6, Phase 1/2 7, Phase 1 8) 2021: 96 trials (Observational 39, Not applicable 17, Phase 4 4, Phase 3 9, Phase 2/3 3, Phase 2 8, Phase 1/2 10, Phase 1 6) 2022: 88 trials (Observational 33, Not applicable 18, Phase 4 4, Phase 3 14, Phase 2/3 1, Phase 2 4, Phase 1/2 9, Phase 1 5) 2023: 101 trials (Observational 47, Not applicable 17, Phase 4 6, Phase 3 8, Phase 2/3 2, Phase 2 9, Phase 1/2 5, Phase 1 7) 2024: 79 trials (Observational 31, Not applicable 19, Phase 4 1, Phase 3 6, Phase 2 7, Phase 1/2 7, Phase 1 8) 2025: 77 trials (Observational 35, Not applicable 17, Phase 4 5, Phase 3 5, Phase 2/3 1, Phase 2 8, Phase 1/2 3, Phase 1 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.

Trials started per year
Year Trials started
2026 (partial year) 73
2025 77
2024 79
2023 101
2022 88
2021 96
2020 74
2019 97
2018 76
2017 57

Showing 10 of 37 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 90 trials starting in 2026 and about 80 in 2027, the average of the 24 months through September, counted as registered and carried forward, with about 0.7 times the starts already registered for the rest of 2026 added. 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 15 percent and ran about 1 percent high against the count later registered; forecasts made before the year began were off by about 26 percent and ran about 20 percent low against the count later registered. The part-way figures are from forecasts that used a multiple fitted only on earlier years. 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 109 trials still running at the 2026-10-09 snapshot; registered median of 28 months across 838 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Brain Diseases, Metabolic trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 126
Phase 1/2 107
Phase 2 171
Phase 2/3 30
Phase 3 138
Phase 4 98
Not applicable 255
Observational 565
Expanded access 25

How many Brain Diseases, Metabolic trials are recruiting? (12)

Shown in this order: the order a trial moves through, from not yet recruiting to unknown.

Trials by recruitment status
Status Trials
Not yet recruiting 68
Recruiting 226
Enrolling by invitation 19
Active, not recruiting 81
Available 7

Showing 5 of 12 statuses.

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Status as registered on ClinicalTrials.gov. Each trial is counted once.

Which lead sponsors run the most Brain Diseases, Metabolic trials? (10)

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.

Lead sponsors
Sponsor Trials
Genzyme, a Sanofi Company 67
Amicus Therapeutics 38
BioMarin Pharmaceutical 38
Shire 34
Sanofi 27

Showing 5 of 10 sponsors.

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Which drugs are being tested for Brain Diseases, Metabolic? (13)

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.

Drugs tested
Drug Trials
Lactulose 33
ALGLUCOSIDASE ALFA 28
REPLAGAL 20
Migalastat 20
AGALSIDASE BETA (GZ419828) 20

Showing 5 of 13 drugs.

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Interventions as named in the registry (2)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
saproterin dihydrochloride 38
Rifaximine 21

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 Brain Diseases, Metabolic trials report? (10)

Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.

Serious adverse events in this condition's trials
Adverse event Subjects affected Trials
Hepatic encephalopathy 200 7
Pneumonia 154 43
Hyperammonaemia 96 9
Pyrexia 95 35
Death 94 12

Showing 5 of 10 adverse events.

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Which publications cover Brain Diseases, Metabolic? (27)

Shown in this order: major-topic papers first, then newest, then most linked to trials.

Publications
Publication Journal Year Major topic
Symptom Prevalence and Genotype-Phenotype Correlations in Patients With TANGO2-Related Metabolic Encephalopathy and Arrhythmias (TRMEA). Pediatric neurology 2021 Yes
Neuroimaging Spectrum of Inherited Neurotransmitter Disorders. Neuropediatrics 2020 Yes
Inherited paediatric neurometabolic disorders, can brain magnetic resonance imaging predict? Neurosciences (Riyadh, Saudi Arabia) 2020 Yes
Single Lysis-Salting Out Method of Genomic DNA Extraction From Dried Blood Spots. Journal of clinical laboratory analysis 2016 Yes
Hyponatremia following mild/moderate subarachnoid hemorrhage is due to SIAD and glucocorticoid deficiency and not cerebral salt wasting. The Journal of clinical endocrinology and metabolism 2014 Yes

Showing 5 of 27 publications.

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Sub-conditions in MeSH (6)

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.

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