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Condition (MeSH)

Purine-Pyrimidine Metabolism, Inborn Errors

ClinicalTrials.gov lists 340 trials for Purine-Pyrimidine Metabolism, Inborn Errors or a narrower condition beneath it. Ardea Biosciences, Inc. has the most among industry and government lead sponsors, with 20.

MeSH D011686 Congenital / Hereditary / Neonatal C16 C18 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 58 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 68 Phase 2/3 Phase 2/3 6 Phase 3 Phase 3 46 Phase 4 Phase 4 31 Not applicable Not applicable 63 Observational Observational 62 Phase 1 Phase 1 58 Phase 1/2 Phase 1/2 6 Phase 2 Phase 2 68 Phase 2/3 Phase 2/3 6 Phase 3 Phase 3 46 Phase 4 Phase 4 31 Not applicable Not applicable 63 Observational Observational 62

Trials per phase

4 of the 340 trials are tagged with Purine-Pyrimidine 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 Purine-Pyrimidine Metabolism, Inborn Errors trials start? (30)

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 2/3
  • Phase 3
  • Phase 4
  • Not applicable
  • Observational

Top of scale: 27

1992: 1 trials (Observational 1) 1996: 1 trials (Phase 2 1) 1998: 1 trials (Phase 4 1) 2000: 1 trials (Observational 1) 2001: 2 trials (Phase 2 2) 2002: 3 trials (Observational 2, Phase 3 1) 2003: 2 trials (Phase 3 2) 2004: 2 trials (Phase 2 2) 2005: 1 trials (Observational 1) 2006: 4 trials (Observational 1, Phase 3 3) 2007: 6 trials (Not applicable 1, Phase 4 2, Phase 3 2, Phase 2 1) 2008: 7 trials (Observational 1, Phase 3 2, Phase 2 3, Phase 1 1) 2009: 14 trials (Observational 1, Phase 4 1, Phase 3 5, Phase 2/3 1, Phase 2 4, Phase 1 2) 2010: 12 trials (Observational 1, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2 6, Phase 1 1) 2011: 18 trials (Observational 1, Not applicable 1, Phase 4 3, Phase 3 8, Phase 2 4, Phase 1 1) 2012: 16 trials (Observational 6, Not applicable 1, Phase 4 1, Phase 3 5, Phase 2 1, Phase 1 2) 2013: 12 trials (Observational 3, Not applicable 2, Phase 3 2, Phase 2 2, Phase 1 3) 2014: 20 trials (Observational 5, Phase 4 1, Phase 3 1, Phase 2 7, Phase 1/2 1, Phase 1 5) 2015: 19 trials (Observational 7, Phase 4 3, Phase 2 4, Phase 1 5) 2016: 7 trials (Observational 1, Not applicable 2, Phase 2/3 1, Phase 2 1, Phase 1 2) 2017: 15 trials (Observational 1, Not applicable 4, Phase 4 3, Phase 2 2, Phase 1 5) 2018: 10 trials (Observational 1, Not applicable 5, Phase 4 1, Phase 2 1, Phase 1 2) 2019: 19 trials (Observational 2, Not applicable 6, Phase 4 1, Phase 3 1, Phase 2 3, Phase 1/2 1, Phase 1 5) 2020: 16 trials (Observational 6, Not applicable 1, Phase 4 1, Phase 2 4, Phase 1 4) 2021: 27 trials (Observational 4, Not applicable 10, Phase 4 2, Phase 3 3, Phase 2 2, Phase 1 6) 2022: 18 trials (Observational 3, Not applicable 6, Phase 3 1, Phase 2 5, Phase 1/2 2, Phase 1 1) 2023: 21 trials (Observational 5, Not applicable 1, Phase 3 4, Phase 2/3 2, Phase 2 2, Phase 1/2 1, Phase 1 6) 2024: 22 trials (Observational 4, Not applicable 4, Phase 4 3, Phase 3 1, Phase 2/3 2, Phase 2 2, Phase 1/2 1, Phase 1 5) 2025: 24 trials (Observational 2, Not applicable 10, Phase 4 3, Phase 3 3, Phase 2 6)

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) 18
2025 24
2024 22
2023 21
2022 18
2021 27
2020 16
2019 19
2018 10
2017 15

Showing 10 of 30 earlier years.

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Forecast of the full-year count, planned starts included, not rows of this table: about 23 trials starting in 2026 and about 22 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 34 months from start to primary completion across 28 trials still running at the 2026-10-09 snapshot; registered median of 15 months across 234 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Purine-Pyrimidine Metabolism, Inborn Errors trials in? (8)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 58
Phase 1/2 6
Phase 2 68
Phase 2/3 6
Phase 3 46
Phase 4 31
Not applicable 63
Observational 62

How many Purine-Pyrimidine Metabolism, Inborn Errors trials are recruiting? (9)

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 14
Recruiting 33
Enrolling by invitation 3
Active, not recruiting 13
Completed 211

Showing 5 of 9 statuses.

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

Which lead sponsors run the most Purine-Pyrimidine Metabolism, Inborn Errors trials? (12)

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
Ardea Biosciences, Inc. 20
Takeda 15
LG Chem 10
Novartis Pharmaceuticals 10
Atom Therapeutics Co., Ltd 9

Showing 5 of 12 sponsors.

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Which drugs are being tested for Purine-Pyrimidine Metabolism, Inborn Errors? (6)

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
Verinurad 10
Pegloticase 10
Tigulixostat 8
FYU-981 6
Rilonacept 6

Showing 5 of 6 drugs.

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Interventions as named in the registry (9)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Febuxostat IR 42
Allopurinol Pill 39
Colchicines 26
Canakinumab, ACZ885 8
Lesinurad 8

Showing 5 of 9 interventions.

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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 Purine-Pyrimidine 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
Cardiac failure 46 7
Pneumonia 45 15
Acute kidney injury 37 4
Gout 37 17
Cellulitis 35 13

Showing 5 of 10 adverse events.

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Which publications cover Purine-Pyrimidine Metabolism, Inborn Errors? (7)

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

Publications
Publication Journal Year Major topic
Allopurinol Treatment Improves Cognitive Skills, Adaptive Behavior, and Biochemical Markers in Young Patients With Adenylosuccinate Lyase Deficiency. Journal of inherited metabolic disease 2025 Yes
Mitochondrial purine and pyrimidine metabolism and beyond. Nucleosides, nucleotides & nucleic acids 2016 Yes
Implementation of TPMT testing. British journal of clinical pharmacology 2014 Yes
Inborn errors of purine metabolism: clinical update and therapies. Journal of inherited metabolic disease 2014 Yes
Inborn errors of pyrimidine metabolism: clinical update and therapy. Journal of inherited metabolic disease 2014 Yes

Showing 5 of 7 publications.

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Rare-disease identity (2)

Shown in this order: exact, broader, narrower, other.

Rare-disease identity
Orphanet disease ORPHA code Relation Source
Disorder of purine or pyrimidine metabolism 79224 Exact Orphanet Rare Disease Ontology (ORDO)
Disorder of purine or pyrimidine metabolism 79224 Exact Orphadata Science knowledge files, Product 1

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.

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