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Condition (MeSH)

Lipid Metabolism, Inborn Errors

ClinicalTrials.gov lists 1,138 trials for Lipid Metabolism, Inborn Errors or a narrower condition beneath it. Genzyme, a Sanofi Company has the most among industry and government lead sponsors, with 36.

MeSH D008052 Congenital / Hereditary / Neonatal C16 C18
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Phase 1 Phase 1 135 Phase 1/2 Phase 1/2 72 Phase 2 Phase 2 125 Phase 2/3 Phase 2/3 23 Phase 3 Phase 3 161 Phase 4 Phase 4 47 Not applicable Not applicable 144 Observational Observational 411 Phase 1 Phase 1 135 Phase 1/2 Phase 1/2 72 Phase 2 Phase 2 125 Phase 2/3 Phase 2/3 23 Phase 3 Phase 3 161 Phase 4 Phase 4 47 Not applicable Not applicable 144 Observational Observational 411

Trials per phase

1 more section with rows

23 of the 1,138 trials are tagged with Lipid Metabolism, Inborn Errors 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 Lipid Metabolism, Inborn Errors trials start? (36)

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

Top of scale: 66

1991: 2 trials (Observational 2) 1992: 1 trials (Phase 1 1) 1993: 3 trials (Observational 1, Phase 2 1, Phase 1 1) 1994: 2 trials (Observational 1, Phase 2 1) 1995: 5 trials (Observational 2, Not applicable 1, Phase 2/3 1, Phase 2 1) 1996: 1 trials (Phase 2 1) 1997: 3 trials (Observational 2, Phase 4 1) 1998: 6 trials (Observational 1, Not applicable 1, Phase 2 2, Phase 1/2 1, Phase 1 1) 1999: 11 trials (Observational 3, Not applicable 1, Phase 4 1, Phase 3 1, Phase 2 1, Phase 1 4) 2000: 6 trials (Observational 3, Phase 3 1, Phase 2/3 1, Phase 2 1) 2001: 18 trials (Observational 9, Phase 4 4, Phase 3 2, Phase 2 1, Phase 1 2) 2002: 18 trials (Observational 3, Phase 3 3, Phase 2/3 1, Phase 2 6, Phase 1 5) 2003: 17 trials (Observational 3, Phase 4 2, Phase 3 3, Phase 2 7, Phase 1 2) 2004: 18 trials (Observational 5, Phase 4 3, Phase 3 3, Phase 2 3, Phase 1/2 1, Phase 1 3) 2005: 18 trials (Observational 5, Phase 4 2, Phase 3 6, Phase 2 1, Phase 1 4) 2006: 34 trials (Observational 6, Not applicable 1, Phase 4 3, Phase 3 8, Phase 2/3 1, Phase 2 11, Phase 1 4) 2007: 30 trials (Observational 8, Not applicable 3, Phase 4 3, Phase 3 4, Phase 2/3 2, Phase 2 3, Phase 1/2 2, Phase 1 5) 2008: 24 trials (Observational 5, Not applicable 1, Phase 3 10, Phase 2 5, Phase 1/2 1, Phase 1 2) 2009: 31 trials (Observational 8, Not applicable 4, Phase 4 1, Phase 3 5, Phase 2/3 1, Phase 2 5, Phase 1/2 3, Phase 1 4) 2010: 38 trials (Observational 22, Not applicable 5, Phase 4 1, Phase 3 3, Phase 2 1, Phase 1/2 3, Phase 1 3) 2011: 56 trials (Observational 17, Not applicable 4, Phase 3 11, Phase 2/3 1, Phase 2 10, Phase 1/2 2, Phase 1 11) 2012: 43 trials (Observational 7, Not applicable 7, Phase 4 2, Phase 3 2, Phase 2/3 2, Phase 2 4, Phase 1/2 5, Phase 1 14) 2013: 34 trials (Observational 10, Not applicable 6, Phase 3 5, Phase 2 3, Phase 1/2 4, Phase 1 6) 2014: 46 trials (Observational 20, Not applicable 4, Phase 4 1, Phase 3 3, Phase 2 7, Phase 1/2 4, Phase 1 7) 2015: 51 trials (Observational 19, Not applicable 4, Phase 4 2, Phase 3 6, Phase 2/3 4, Phase 2 7, Phase 1/2 3, Phase 1 6) 2016: 49 trials (Observational 21, Not applicable 9, Phase 4 2, Phase 3 6, Phase 2/3 1, Phase 2 5, Phase 1/2 2, Phase 1 3) 2017: 46 trials (Observational 23, Not applicable 6, Phase 3 4, Phase 2/3 1, Phase 2 5, Phase 1/2 2, Phase 1 5) 2018: 58 trials (Observational 23, Not applicable 9, Phase 4 4, Phase 3 12, Phase 2 5, Phase 1/2 3, Phase 1 2) 2019: 59 trials (Observational 23, Not applicable 10, Phase 4 4, Phase 3 5, Phase 2/3 3, Phase 2 5, Phase 1/2 5, Phase 1 4) 2020: 52 trials (Observational 24, Not applicable 10, Phase 4 3, Phase 3 6, Phase 2 3, Phase 1/2 4, Phase 1 2) 2021: 63 trials (Observational 25, Not applicable 12, Phase 4 3, Phase 3 7, Phase 2 1, Phase 1/2 7, Phase 1 8) 2022: 58 trials (Observational 21, Not applicable 9, Phase 4 1, Phase 3 10, Phase 2 5, Phase 1/2 7, Phase 1 5) 2023: 66 trials (Observational 29, Not applicable 13, Phase 4 1, Phase 3 7, Phase 2/3 2, Phase 2 4, Phase 1/2 5, Phase 1 5) 2024: 57 trials (Observational 22, Not applicable 11, Phase 3 8, Phase 2 3, Phase 1/2 3, Phase 1 10) 2025: 38 trials (Observational 18, Not applicable 6, Phase 4 1, Phase 3 7, Phase 2/3 1, Phase 2 2, Phase 1/2 2, Phase 1 1)

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) 43
2025 38
2024 57
2023 66
2022 58
2021 63
2020 52
2019 59
2018 58
2017 46

Forecast of the full-year count, planned starts included, not rows of this table: about 52 trials starting in 2026 and about 44 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 78 trials still running at the 2026-10-09 snapshot; registered median of 24 months across 659 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Lipid Metabolism, Inborn Errors trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 135
Phase 1/2 72
Phase 2 125
Phase 2/3 23
Phase 3 161
Phase 4 47
Not applicable 144
Observational 411
Expanded access 20

How many Lipid Metabolism, Inborn Errors 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 35
Recruiting 153
Enrolling by invitation 12
Active, not recruiting 54
Available 7

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

Which lead sponsors run the most Lipid Metabolism, Inborn Errors trials? (15)

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 36
Shire 34
Amicus Therapeutics 29
Sanofi 23
National Institute of Neurological Disorders and Stroke (NINDS) 18

Which drugs are being tested for Lipid Metabolism, Inborn Errors? (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
REPLAGAL 20
Migalastat 20
AGALSIDASE BETA (GZ419828) 20
ATORVASTATIN 14
Velaglucerase Alfa 13

Interventions as named in the registry (2)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Inclisiran (Leqvio®) 300mg 9
Mofetil Mycophenolate 9

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 Lipid Metabolism, Inborn Errors 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
Coronary artery disease 197 22
Angina pectoris 186 29
Pneumonia 179 39
Acute myocardial infarction 167 22
Angina unstable 152 19

Which publications cover Lipid Metabolism, Inborn Errors? (61)

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

Sub-conditions in MeSH (14)

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.

Browse all condition evidence pages