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ORPHA:238624

Idiopathic intracranial hypertension

medium confidenceDisorder

Also known as: Benign intracranial hypertension · IIH · Pseudotumor cerebri

Publications

12,386

95.7th percentile

Trials

37

Interventional, condition-specific

Researchers

1,167

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

intracranial hypertension is a neurological disorder characterized by isolated increased intracranial pressure manifesting with recurrent and persistent headaches, nausea, vomiting, and transient obstruction of the visual field, papilledema. Visual loss can be irreversible.

How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (5)

Idiopathic Intracranial Hypertension · benign intracran. hypt. · benign intracranial hypertension · idiopathic intracranial hypertension · pseudotumor cerebri

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

4/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    12,386 matched papers (6,426 in last 10 years) Source

  3. Phenotype characterisedPresent

    24 HPO annotations (e.g. Papilledema; Hypertension; Increased intracranial pressure) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    1 FDA · 2 EMA designations (1 FDA orphan-indication approval) — e.g. exenatide Source

  6. Interventional trialPresent

    37 matched on ClinicalTrials.gov (10 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

24

Associated phenotypes · MONDO:0009468

  • Papilledema
  • Hypertension
  • Increased intracranial pressure
  • Visual loss
  • Photophobia

Showing 5 of 24 — open Monarch for the full list.

Animal models (Monarch / Alliance)

None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

3

Designations · 1 with FDA orphan-indication approval

  • FDA exenatideIdiopathic Intracranial Hypertension · 2017-05-16 · Not FDA Approved for Orphan Indication
  • EMA exenatideTreatment of idiopathic intracranial hypertension · 21/03/2016 · PositiveEMA designation
  • EMA octreotide acetateTreatment of idiopathic intracranial hypertension · 10/12/2021 · PositiveEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

13

Drugs / clinical candidates · MONDO_0009468

CTD chemicals (MyDisease.info)

43 associated chemicals. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Acetazolamide · therapeutic
  • Dexamethasone · therapeutic
  • Prednisone · therapeutic
  • Topiramate · therapeutic
  • Vitamin A · therapeutic
  • Warfarin · therapeutic
  • Adrenal Cortex Hormones · marker/mechanism
  • Alitretinoin · marker/mechanism
  • Amiodarone · marker/mechanism
  • Ampicillin · marker/mechanism
  • Betamethasone · marker/mechanism
  • Budesonide · marker/mechanism

MyDisease.info · MONDO:0009468

Literature

Is anyone studying this?

12,386

12,386 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

12,386 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

6,426 in the last 10 years · medium confidence · 95.7th percentile (publications denominator)

Phrase hits: 12,386 · MeSH hits: 125

Open Europe PMC search

Who's working on it?

1,167

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Mollan SP6 papers · 2026

    Metabolism and Systems Science, School of Medical Sciences, College of Medicine and Health, University of Birmingham, Birmingham, UK.

    Papers in Europe PMC
  2. 02
    Hansen NS5 papers · 2026

    Department of Neurology, Danish Headache Center, Copenhagen University Hospital - Rigshospitalet, Glostrup, Denmark.

    Papers in Europe PMC
  3. 03
    Jensen RH5 papers · 2026

    Department of Neurology, Danish Headache Center, Copenhagen University Hospital - Rigshospitalet, Glostrup, Denmark.

    Papers in Europe PMC
  4. 04
    Beier D4 papers · 2026

    Department of Neurology, Odense University Hospital, Odense, Denmark.

    Papers in Europe PMC
  5. 05
    Bender MT4 papers · 2026

    Department of Neurosurgery, University of Rochester Medical Center, Rochester, NY, USA.

    Papers in Europe PMC
  6. 06
    Bsteh G4 papers · 2026

    Department of Neurology, Medical University of Vienna, Waehringer Guertel 18-20, Vienna, 1090, Austria. gabriel.bsteh@meduniwien.ac.at.

    Papers in Europe PMC
  7. 07
    Davies JM4 papers · 2026

    Department of Neurosurgery, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo, Buffalo , New York , USA.

    Papers in Europe PMC
  8. 08
    Fargen KM4 papers · 2025

    Neurological Surgery and Radiology, Wake Forest University, Winston-Salem, North Carolina, USA.

    Papers in Europe PMC
  9. 09
    Grech O4 papers · 2026

    Metabolism and Systems Science, School of Medical Sciences, College of Medicine and Health, University of Birmingham, Birmingham, UK.

    Papers in Europe PMC
  10. 10
    Pemp B4 papers · 2026

    Department of Ophthalmology, Medical University of Vienna, Vienna, Austria.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

37

interventional trials for this specific condition

37 interventional trials matched this specific condition name; 10 currently recruiting in our sample. 44 trials are registered for intracranial hypertension, the broader category — shown separately because they may or may not enrol this specific subtype.

Data as of 11 September 2026

37 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 96.5th percentile).

medium confidence · 96.5th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

37 interventional trials matched after quoted-phrase search and title/condition post-filter.

Broader category: intracranial hypertension

44

Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.

Worth raising with a clinician. How we count trials.

Recruiting under the broader category

Observational and natural-history studies

16 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 17 · after dedupe 17 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 17 · dropped 0 · fetched 2026-07-30

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (17)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Idiopathic intracranial hypertension — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Idiopathic intracranial hypertension" OR "Benign intracranial hypertension" OR "Pseudotumor cerebri" OR "benign intracran. hypt."

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Pseudotumor Cerebri

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Idiopathic intracranial hypertension" OR "Benign intracranial hypertension" OR "Pseudotumor cerebri" OR "benign intracran. hypt."

Interventional trials matched via: both, phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 37 interventional · 16 observational · 0 expanded access. Only interventional studies enter the trial headline.

Parent-category trials query:

"intracranial hypertension"

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: IIH

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T10:25:12.940Z