Skip to content
Pharma Observatory a Scientist.com platform

Condition (MeSH)

Metal Metabolism, Inborn Errors

ClinicalTrials.gov lists 264 trials for Metal Metabolism, Inborn Errors or a narrower condition beneath it. Alexion Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 34.

MeSH D008664 Congenital / Hereditary / Neonatal C16 C18
Email me this page as a PDF

Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.

Phase 1 Phase 1 19 Phase 1/2 Phase 1/2 19 Phase 2 Phase 2 32 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 27 Phase 4 Phase 4 14 Not applicable Not applicable 37 Observational Observational 110 Phase 1 Phase 1 19 Phase 1/2 Phase 1/2 19 Phase 2 Phase 2 32 Phase 2/3 Phase 2/3 2 Phase 3 Phase 3 27 Phase 4 Phase 4 14 Not applicable Not applicable 37 Observational Observational 110

Trials per phase (log scale)

1 more section with rows

1 of the 264 trials are tagged with Metal Metabolism, Inborn Errors itself; the rest are tagged with a narrower condition beneath it.

Get the Metal Metabolism, Inborn Errors report as a PDF

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 Metal Metabolism, Inborn Errors trials start? (34)

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: 22

1990: 1 trials (Phase 1/2 1) 1992: 1 trials (Phase 3 1) 1993: 2 trials (Observational 1, Phase 4 1) 1994: 1 trials (Phase 3 1) 1995: 1 trials (Observational 1) 1997: 2 trials (Observational 2) 1999: 2 trials (Observational 2) 2000: 2 trials (Observational 1, Phase 3 1) 2001: 1 trials (Phase 2 1) 2002: 2 trials (Phase 4 1, Phase 3 1) 2003: 5 trials (Observational 3, Not applicable 1, Phase 2/3 1) 2004: 3 trials (Observational 1, Phase 3 1, Phase 1 1) 2005: 5 trials (Observational 2, Phase 3 1, Phase 2 1, Phase 1 1) 2006: 4 trials (Not applicable 1, Phase 3 1, Phase 1/2 2) 2007: 5 trials (Observational 1, Phase 3 2, Phase 2 1, Phase 1/2 1) 2008: 7 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 1/2 1, Phase 1 3) 2009: 6 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 2 3) 2010: 7 trials (Observational 2, Not applicable 2, Phase 2/3 1, Phase 2 2) 2011: 6 trials (Observational 1, Not applicable 3, Phase 2 1, Phase 1/2 1) 2012: 6 trials (Observational 2, Not applicable 3, Phase 1/2 1) 2013: 8 trials (Observational 2, Not applicable 2, Phase 3 2, Phase 2 1, Phase 1 1) 2014: 12 trials (Observational 6, Not applicable 1, Phase 2 4, Phase 1 1) 2015: 12 trials (Observational 4, Not applicable 1, Phase 4 1, Phase 3 2, Phase 2 4) 2016: 11 trials (Observational 5, Not applicable 2, Phase 4 1, Phase 3 1, Phase 2 2) 2017: 9 trials (Observational 6, Phase 4 1, Phase 3 1, Phase 2 1) 2018: 16 trials (Observational 8, Not applicable 3, Phase 4 1, Phase 3 2, Phase 2 2) 2019: 15 trials (Observational 9, Phase 4 2, Phase 3 1, Phase 1 3) 2020: 17 trials (Observational 6, Not applicable 1, Phase 4 1, Phase 2 5, Phase 1/2 1, Phase 1 3) 2021: 22 trials (Observational 12, Not applicable 1, Phase 4 2, Phase 3 3, Phase 1/2 3, Phase 1 1) 2022: 8 trials (Observational 4, Not applicable 2, Phase 1 2) 2023: 13 trials (Observational 7, Not applicable 3, Phase 4 2, Phase 1/2 1) 2024: 15 trials (Observational 5, Not applicable 4, Phase 3 3, Phase 1/2 1, Phase 1 2) 2025: 16 trials (Observational 9, Not applicable 3, Phase 2 1, 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) 14
2025 16
2024 15
2023 13
2022 8
2021 22
2020 17
2019 15
2018 16
2017 9

Forecast of the full-year count, planned starts included, not rows of this table: about 18 trials starting in 2026 and about 17 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 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. 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 23 trials still running at the 2026-10-09 snapshot; registered median of 24 months across 143 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Metal 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 19
Phase 1/2 19
Phase 2 32
Phase 2/3 2
Phase 3 27
Phase 4 14
Not applicable 37
Observational 110
Expanded access 4

How many Metal Metabolism, Inborn Errors trials are recruiting? (11)

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 11
Recruiting 41
Enrolling by invitation 4
Active, not recruiting 19
Available 1

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

Which lead sponsors run the most Metal Metabolism, Inborn Errors trials? (9)

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
Alexion Pharmaceuticals, Inc. 34
Kyowa Kirin Co., Ltd. 10
Fondation Ophtalmologique Adolphe de Rothschild 7
Kyowa Kirin, Inc. 6
Orphalan 6

Which drugs are being tested for Metal 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
Burosumab 19
Asfotase Alfa 16
ALXN1840 10
ALXN1850 4
Trientine 4

Interventions as named in the registry (9)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
theophylline SR 5
Deferoxamine mesylate only 5
Deferasirox FCT 4
Deferiprone pill 3
Liver biopsy 2

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 Metal 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
Craniosynostosis 24 4
Death 21 1
Sickle cell anaemia with crisis 21 1
Pneumonia 16 6
Pyrexia 16 4

Which publications cover Metal Metabolism, Inborn Errors? (2)

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

Sub-conditions in MeSH (7)

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