Skip to content
Pharma Observatory a Scientist.com platform

Condition (MeSH)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ClinicalTrials.gov lists 24,677 trials for Congenital, Hereditary, and Neonatal Diseases and Abnormalities or a narrower condition beneath it. National Cancer Institute (NCI) has the most among industry and government lead sponsors, with 203.

MeSH D009358 Congenital / Hereditary / Neonatal C16
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 1,991 Phase 1/2 Phase 1/2 1,133 Phase 2 Phase 2 2,956 Phase 2/3 Phase 2/3 441 Phase 3 Phase 3 2,128 Phase 4 Phase 4 1,094 Not applicable Not applicable 6,694 Observational Observational 8,109 Phase 1 Phase 1 1,991 Phase 1/2 Phase 1/2 1,133 Phase 2 Phase 2 2,956 Phase 2/3 Phase 2/3 441 Phase 3 Phase 3 2,128 Phase 4 Phase 4 1,094 Not applicable Not applicable 6,694 Observational Observational 8,109

Trials per phase

2 more sections with rows

108 of the 24,677 trials are tagged with Congenital, Hereditary, and Neonatal Diseases and Abnormalities itself; the rest are tagged with a narrower condition beneath it.

Get the Congenital, Hereditary, and Neonatal Diseases and Abnormalities 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 Congenital, Hereditary, and Neonatal Diseases and Abnormalities 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. 60 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: 1,554

1990: 16 trials (Observational 10, Not applicable 2, Phase 3 1, Phase 2 2, Phase 1/2 1) 1991: 15 trials (Observational 7, Not applicable 1, Phase 3 2, Phase 2/3 2, Phase 2 3) 1992: 30 trials (Observational 11, Not applicable 2, Phase 4 3, Phase 3 5, Phase 2 5, Phase 1/2 2, Phase 1 2) 1993: 38 trials (Observational 15, Not applicable 1, Phase 4 2, Phase 3 9, Phase 2 5, Phase 1/2 1, Phase 1 5) 1994: 36 trials (Observational 18, Not applicable 4, Phase 3 3, Phase 2/3 1, Phase 2 7, Phase 1 3) 1995: 57 trials (Observational 25, Not applicable 8, Phase 3 7, Phase 2/3 1, Phase 2 7, Phase 1/2 1, Phase 1 8) 1996: 50 trials (Observational 22, Not applicable 7, Phase 4 1, Phase 3 7, Phase 2 11, Phase 1/2 2) 1997: 48 trials (Observational 19, Not applicable 7, Phase 4 3, Phase 3 7, Phase 2/3 2, Phase 2 6, Phase 1/2 1, Phase 1 3) 1998: 69 trials (Observational 22, Not applicable 10, Phase 4 1, Phase 3 9, Phase 2/3 1, Phase 2 10, Phase 1/2 1, Phase 1 15) 1999: 110 trials (Observational 38, Not applicable 15, Phase 4 4, Phase 3 9, Phase 2/3 1, Phase 2 26, Phase 1/2 5, Phase 1 12) 2000: 138 trials (Observational 45, Not applicable 16, Phase 4 4, Phase 3 22, Phase 2/3 8, Phase 2 22, Phase 1/2 10, Phase 1 11) 2001: 173 trials (Observational 62, Not applicable 15, Phase 4 13, Phase 3 17, Phase 2/3 5, Phase 2 34, Phase 1/2 11, Phase 1 16) 2002: 191 trials (Observational 63, Not applicable 25, Phase 4 8, Phase 3 25, Phase 2/3 4, Phase 2 37, Phase 1/2 8, Phase 1 21) 2003: 205 trials (Other 1, Observational 49, Not applicable 30, Phase 4 15, Phase 3 25, Phase 2/3 11, Phase 2 42, Phase 1/2 13, Phase 1 19) 2004: 322 trials (Observational 98, Not applicable 37, Phase 4 30, Phase 3 40, Phase 2/3 9, Phase 2 55, Phase 1/2 12, Phase 1 41) 2005: 408 trials (Observational 119, Not applicable 58, Phase 4 37, Phase 3 60, Phase 2/3 8, Phase 2 66, Phase 1/2 15, Phase 1 45) 2006: 495 trials (Other 1, Observational 141, Not applicable 82, Phase 4 38, Phase 3 69, Phase 2/3 14, Phase 2 88, Phase 1/2 21, Phase 1 41) 2007: 513 trials (Other 1, Observational 170, Not applicable 80, Phase 4 43, Phase 3 61, Phase 2/3 14, Phase 2 72, Phase 1/2 37, Phase 1 35) 2008: 570 trials (Observational 149, Not applicable 101, Phase 4 40, Phase 3 68, Phase 2/3 16, Phase 2 98, Phase 1/2 35, Phase 1 63) 2009: 672 trials (Other 1, Observational 186, Not applicable 118, Phase 4 51, Phase 3 75, Phase 2/3 20, Phase 2 112, Phase 1/2 29, Phase 1 80) 2010: 696 trials (Observational 228, Not applicable 129, Phase 4 40, Phase 3 71, Phase 2/3 11, Phase 2 117, Phase 1/2 38, Phase 1 62) 2011: 749 trials (Observational 219, Not applicable 158, Phase 4 40, Phase 3 89, Phase 2/3 17, Phase 2 116, Phase 1/2 41, Phase 1 69) 2012: 854 trials (Observational 286, Not applicable 208, Phase 4 43, Phase 3 68, Phase 2/3 22, Phase 2 110, Phase 1/2 31, Phase 1 86) 2013: 841 trials (Observational 289, Not applicable 199, Phase 4 41, Phase 3 76, Phase 2/3 11, Phase 2 112, Phase 1/2 50, Phase 1 63) 2014: 936 trials (Observational 304, Not applicable 228, Phase 4 38, Phase 3 73, Phase 2/3 20, Phase 2 138, Phase 1/2 43, Phase 1 92) 2015: 1,054 trials (Observational 338, Not applicable 274, Phase 4 50, Phase 3 82, Phase 2/3 18, Phase 2 134, Phase 1/2 48, Phase 1 110) 2016: 1,073 trials (Observational 342, Not applicable 330, Phase 4 47, Phase 3 80, Phase 2/3 14, Phase 2 145, Phase 1/2 41, Phase 1 74) 2017: 1,126 trials (Other 1, Observational 372, Not applicable 308, Phase 4 50, Phase 3 81, Phase 2/3 16, Phase 2 149, Phase 1/2 55, Phase 1 94) 2018: 1,367 trials (Observational 452, Not applicable 416, Phase 4 53, Phase 3 116, Phase 2/3 19, Phase 2 161, Phase 1/2 55, Phase 1 95) 2019: 1,367 trials (Observational 500, Not applicable 406, Phase 4 35, Phase 3 101, Phase 2/3 25, Phase 2 156, Phase 1/2 56, Phase 1 88) 2020: 1,314 trials (Observational 502, Not applicable 380, Phase 4 52, Phase 3 94, Phase 2/3 19, Phase 2 138, Phase 1/2 52, Phase 1 77) 2021: 1,511 trials (Observational 583, Not applicable 456, Phase 4 52, Phase 3 103, Phase 2/3 22, Phase 2 125, Phase 1/2 65, Phase 1 105) 2022: 1,480 trials (Observational 508, Not applicable 494, Phase 4 51, Phase 3 112, Phase 2/3 22, Phase 2 123, Phase 1/2 66, Phase 1 104) 2023: 1,483 trials (Observational 498, Not applicable 521, Phase 4 56, Phase 3 101, Phase 2/3 28, Phase 2 117, Phase 1/2 66, Phase 1 96) 2024: 1,554 trials (Observational 496, Not applicable 551, Phase 4 45, Phase 3 121, Phase 2/3 19, Phase 2 119, Phase 1/2 82, Phase 1 121) 2025: 1,434 trials (Observational 460, Not applicable 516, Phase 4 46, Phase 3 103, Phase 2/3 20, Phase 2 129, Phase 1/2 56, Phase 1 104)

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) 1,285
2025 1,434
2024 1,554
2023 1,483
2022 1,480
2021 1,511
2020 1,314
2019 1,367
2018 1,367
2017 1,126

Showing 10 of 37 earlier years.

Start your 14-day trial Email me all 37 earlier years as a PDF

Forecast of the full-year count, planned starts included, not rows of this table: about 1,600 trials starting in 2026 and about 1,500 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 10 percent and ran about 4 percent high against the count later registered; forecasts made before the year began were off by about 25 percent and ran about 15 percent low against the count later registered, and had about 7 percent more error than repeating last year's count. The part-way figures are from forecasts that used a multiple fitted only on earlier years. A stricter check that trains only on older years did not support adding planned starts for conditions of this size: it ran about 6 percent high 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 1,998 trials still running at the 2026-10-09 snapshot; registered median of 24 months across 14,661 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials in? (10)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 1,991
Phase 1/2 1,133
Phase 2 2,956
Phase 2/3 441
Phase 3 2,128
Phase 4 1,094
Not applicable 6,694
Observational 8,109
Expanded access 129
Unknown 2

How many Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials are recruiting? (13)

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 1,065
Recruiting 3,190
Enrolling by invitation 307
Active, not recruiting 1,144
Available 40

Showing 5 of 13 statuses.

Start your 14-day trial Email me all 13 statuses as a PDF

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

Which lead sponsors run the most Congenital, Hereditary, and Neonatal Diseases and Abnormalities trials? (7)

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
National Cancer Institute (NCI) 203
Pfizer 200
Novartis Pharmaceuticals 196
National Heart, Lung, and Blood Institute (NHLBI) 153
National Institute of Allergy and Infectious Diseases (NIAID) 148

Showing 5 of 7 sponsors.

Start your 14-day trial Email me all 7 sponsors as a PDF

Which drugs are being tested for Congenital, Hereditary, and Neonatal Diseases and Abnormalities? (3)

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
Cytoxan 158
Busulfan 127
Sirolimus 104

Interventions as named in the registry (12)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
SQ Fludarabine 170
Cyclosporine 0.05% 100
Cytarabine IT 97
IR Tacrolimus 91
Mofetil Mycophenolate 90

Showing 5 of 12 interventions.

Start your 14-day trial Email me all 12 interventions as a PDF

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 Congenital, Hereditary, and Neonatal Diseases and Abnormalities 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
Infective pulmonary exacerbation of cystic fibrosis 2,530 96
Pneumonia 2,454 560
Neutrophil count decreased 1,588 38
Platelet count decreased 1,588 42
Anemia 1,558 52

Showing 5 of 10 adverse events.

Start your 14-day trial Email me all 10 adverse events as a PDF

Sub-conditions in MeSH (4)

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: publications, prevalence, and rare-disease identity.

Browse all condition evidence pages