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Condition (MeSH)

Nervous System Malformations

ClinicalTrials.gov lists 853 trials for Nervous System Malformations or a narrower condition beneath it. Novartis Pharmaceuticals has the most among industry and government lead sponsors, with 18.

MeSH D009421 Nervous System Diseases C10 C16 Listed by Orphanet as a rare disease
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Phase 1 Phase 1 56 Phase 1/2 Phase 1/2 43 Phase 2 Phase 2 98 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 45 Phase 4 Phase 4 18 Not applicable Not applicable 248 Observational Observational 328 Phase 1 Phase 1 56 Phase 1/2 Phase 1/2 43 Phase 2 Phase 2 98 Phase 2/3 Phase 2/3 15 Phase 3 Phase 3 45 Phase 4 Phase 4 18 Not applicable Not applicable 248 Observational Observational 328

Trials per phase (log scale)

1 more section with rows

10 of the 853 trials are tagged with Nervous System Malformations itself; the rest are tagged with a narrower condition beneath it.

Get the Nervous System Malformations 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 Nervous System Malformations 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. 1 trial is 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: 64

1992: 1 trials (Observational 1) 1993: 1 trials (Observational 1) 1994: 1 trials (Observational 1) 1995: 1 trials (Observational 1) 1996: 3 trials (Observational 3) 1998: 2 trials (Observational 2) 1999: 2 trials (Observational 2) 2000: 6 trials (Observational 5, Not applicable 1) 2001: 3 trials (Observational 3) 2002: 3 trials (Observational 1, Phase 3 1, Phase 2 1) 2003: 4 trials (Observational 1, Not applicable 1, Phase 3 1, Phase 1/2 1) 2004: 5 trials (Observational 4, Phase 4 1) 2005: 6 trials (Observational 1, Not applicable 1, Phase 4 1, Phase 2 2, Phase 1/2 1) 2006: 10 trials (Observational 3, Not applicable 3, Phase 3 1, Phase 2 3) 2007: 10 trials (Observational 3, Not applicable 2, Phase 3 1, Phase 2/3 1, Phase 1/2 3) 2008: 15 trials (Observational 4, Not applicable 3, Phase 3 1, Phase 2 3, Phase 1/2 1, Phase 1 3) 2009: 24 trials (Observational 9, Not applicable 2, Phase 4 1, Phase 3 6, Phase 2 1, Phase 1 5) 2010: 23 trials (Observational 14, Phase 4 1, Phase 2 3, Phase 1/2 3, Phase 1 2) 2011: 14 trials (Observational 5, Not applicable 1, Phase 2/3 1, Phase 2 6, Phase 1 1) 2012: 31 trials (Observational 14, Not applicable 4, Phase 3 1, Phase 2/3 2, Phase 2 6, Phase 1/2 1, Phase 1 3) 2013: 31 trials (Observational 11, Not applicable 6, Phase 4 2, Phase 3 6, Phase 2 2, Phase 1/2 2, Phase 1 2) 2014: 23 trials (Observational 6, Not applicable 9, Phase 3 4, Phase 2 2, Phase 1/2 1, Phase 1 1) 2015: 43 trials (Observational 17, Not applicable 12, Phase 3 3, Phase 2 7, Phase 1 4) 2016: 51 trials (Observational 23, Not applicable 13, Phase 4 1, Phase 3 3, Phase 2/3 1, Phase 2 6, Phase 1/2 1, Phase 1 3) 2017: 51 trials (Observational 22, Not applicable 16, Phase 4 1, Phase 3 3, Phase 2/3 1, Phase 2 6, Phase 1/2 1, Phase 1 1) 2018: 52 trials (Observational 17, Not applicable 17, Phase 4 3, Phase 3 2, Phase 2 7, Phase 1/2 1, Phase 1 5) 2019: 44 trials (Observational 21, Not applicable 15, Phase 3 1, Phase 2/3 1, Phase 2 5, Phase 1 1) 2020: 45 trials (Observational 16, Not applicable 16, Phase 2/3 1, Phase 2 7, Phase 1/2 3, Phase 1 2) 2021: 51 trials (Observational 19, Not applicable 13, Phase 4 1, Phase 3 2, Phase 2/3 1, Phase 2 8, Phase 1/2 5, Phase 1 2) 2022: 64 trials (Observational 22, Not applicable 24, Phase 4 1, Phase 3 3, Phase 2/3 1, Phase 2 6, Phase 1/2 4, Phase 1 3) 2023: 57 trials (Observational 18, Not applicable 24, Phase 4 3, Phase 3 1, Phase 2/3 1, Phase 2 3, Phase 1/2 3, Phase 1 4) 2024: 64 trials (Observational 23, Not applicable 23, Phase 4 2, Phase 3 1, Phase 2/3 2, Phase 2 6, Phase 1/2 5, Phase 1 2) 2025: 56 trials (Observational 19, Not applicable 21, Phase 3 1, Phase 2 4, Phase 1/2 3, Phase 1 8)

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) 50
2025 56
2024 64
2023 57
2022 64
2021 51
2020 45
2019 44
2018 52
2017 51

Forecast of the full-year count, planned starts included, not rows of this table: about 63 trials starting in 2026 and about 58 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 89 trials still running at the 2026-10-09 snapshot; registered median of 29 months across 464 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.

What phase are Nervous System Malformations trials in? (9)

Shown in this order: phase order.

Show the trials by phase as a table
Trials by phase
Phase Trials
Phase 1 56
Phase 1/2 43
Phase 2 98
Phase 2/3 15
Phase 3 45
Phase 4 18
Not applicable 248
Observational 328
Expanded access 2

How many Nervous System Malformations 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 34
Recruiting 164
Enrolling by invitation 10
Active, not recruiting 53
Available 1

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

Which lead sponsors run the most Nervous System Malformations trials? (6)

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
Novartis Pharmaceuticals 18
National Institute of Neurological Disorders and Stroke (NINDS) 9
Pfizer 9
AstraZeneca 7
National Heart, Lung, and Blood Institute (NHLBI) 7

Which drugs are being tested for Nervous System Malformations? (7)

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
EVEROLIMUS 18
Sirolimus 15
FULVESTRANT 8
Cannabidiol 6
CETUXIMAB 5

Interventions as named in the registry (8)

Shown in this order: most trials first.

Interventions as named in the registry
Drug Trials
Alpelisib Pill 8
Tanezumab SC 7
MRI 5
Letrozol (Femara, Novartis) 4
Palbociclib Pill 3

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 Nervous System Malformations 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
Pneumonia 148 30
Diarrhoea 88 20
Dyspnoea 71 16
Seizure 68 19
Neutropenia 62 6

Which publications cover Nervous System Malformations? (45)

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

Sub-conditions in MeSH (9)

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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