Condition (MeSH)
Noonan Syndrome
ClinicalTrials.gov lists 47 trials for Noonan Syndrome or a narrower condition beneath it. Novo Nordisk A/S has the most among industry and government lead sponsors, with 8.
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Trials per phase
47 of the 47 trials are tagged with Noonan Syndrome itself; the rest are tagged with a narrower condition beneath it.
When did Noonan Syndrome trials start? (18)
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 3
- Not applicable
- Observational
Top of scale: 5
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.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2025 | 3 |
| 2024 | 4 |
| 2023 | 3 |
| 2022 | 4 |
| 2021 | 4 |
| 2020 | 4 |
| 2019 | 4 |
| 2018 | 5 |
| 2017 | 4 |
Showing 10 of 18 earlier years.
No forecast is made for this condition: it averages 3.2 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 6 trials still running at the 2026-10-09 snapshot; registered median of 37 months across 27 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Noonan Syndrome trials in? (6)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 8 |
| Phase 1/2 | 3 |
| Phase 2 | 6 |
| Phase 3 | 9 |
| Not applicable | 7 |
| Observational | 14 |
How many Noonan Syndrome trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 2 |
| Recruiting | 11 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 4 |
| Completed | 19 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Noonan Syndrome 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.
| Sponsor | Trials |
|---|---|
| Novo Nordisk A/S | 8 |
| Jacobio Pharmaceuticals Co., Ltd. | 3 |
| Erasca, Inc. | 2 |
| National Cancer Institute (NCI) | 2 |
| Revolution Medicines, Inc. | 2 |
Showing 5 of 9 sponsors.
Which drugs are being tested for Noonan Syndrome? (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.
| Drug | Trials |
|---|---|
| GSK1120212 | 2 |
| Osimertinib | 2 |
| BMN 111 | 2 |
| Somapacitan | 2 |
| RMC-4630 | 2 |
Showing 5 of 7 drugs.
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| somatropin AQ | 7 |
| ERAS-601 | 2 |
| Lamotrigine use | 1 |
| Oral Glucose Tolerance Test | 1 |
| MAXOMAT ®, biosynthetic growth hormone | 1 |
Showing 5 of 8 interventions.
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 Noonan Syndrome trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| pneumonia | 6 | 1 |
| pneumonitis | 4 | 1 |
| gastrointestinal hemorrhage | 3 | 1 |
| pleural effusion | 3 | 1 |
| sepsis | 3 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Noonan Syndrome? (22)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Long-term efficacy and safety of two doses of Norditropin<sup>®</sup> (somatropin) in Noonan syndrome: a 4-year randomized, double-blind, multicenter trial in Japanese patients. | Endocrine journal | 2020 | Yes |
| Efficacy and safety of two doses of Norditropin<sup>®</sup> (somatropin) in short stature due to Noonan syndrome: a 2-year randomized, double-blind, multicenter trial in Japanese patients. | Endocrine journal | 2018 | Yes |
| Autosomal recessive Noonan syndrome associated with biallelic LZTR1 variants. | Genetics in medicine : official journal of the American College of Medical Genetics | 2018 | Yes |
| Autism spectrum disorder and other neurobehavioural comorbidities in rare disorders of the Ras/MAPK pathway. | Developmental medicine and child neurology | 2017 | Yes |
| Constitutional bone impairment in Noonan syndrome. | American journal of medical genetics. Part A | 2017 | Yes |
Showing 5 of 22 publications.
Prevalence (6)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Noonan syndrome | Prevalence at birth | 6-9 / 10 000 | United States | US | Unspecified |
| Noonan syndrome | Prevalence at birth | 6-9 / 10 000 | United States | US | Exact |
| Noonan syndrome | Prevalence at birth | 6-9 / 10 000 | United States | US | Exact |
| Noonan syndrome | Point prevalence | 1-5 / 10 000 | Worldwide | Not stated | Exact |
| Noonan syndrome | Point prevalence | 1-5 / 10 000 | Worldwide | Not stated | Exact |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Noonan syndrome | 648 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Noonan syndrome | 648 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.