Idiopathic intracranial hypertension

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ORPHA:238624OMIM:243200G93.2
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16Active trials27Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Idiopathic intracranial hypertension (IIH), also known as pseudotumor cerebri or benign intracranial hypertension, is a condition where the pressure of the fluid surrounding the brain (called cerebrospinal fluid or CSF) becomes abnormally high without an obvious cause such as a tumor, infection, or blood clot. The word 'idiopathic' means the exact cause is unknown. This increased pressure pushes on the brain and the nerves behind the eyes, leading to a range of symptoms. The most common symptom is severe, persistent headaches that may worsen with eye movement or straining. Many people also experience visual problems, including blurred vision, double vision, brief episodes of vision going dark (called visual obscurations), and in some cases, permanent vision loss if the condition is not treated. Other symptoms can include pulsatile tinnitus (a whooshing sound in the ears that matches your heartbeat), neck pain, back pain, and nausea. IIH most commonly affects women of childbearing age, particularly those who are overweight or obese. Treatment focuses on lowering the pressure inside the skull to protect vision and relieve symptoms. This typically includes weight loss, medications like acetazolamide (Diamox) to reduce fluid production, and in more severe cases, surgical procedures such as optic nerve sheath fenestration or CSF shunting. With proper treatment, many patients can manage their symptoms effectively, though some may experience recurrences.

Also known as:

Key symptoms:

Severe persistent headachesBlurred or dimmed visionBrief episodes of vision going dark (visual obscurations)Double visionWhooshing or pulsing sound in the ears (pulsatile tinnitus)Pain behind the eyesNeck stiffness or painBack painNausea and vomitingDizzinessSwelling of the optic nerve (papilledema)Difficulty with peripheral (side) visionFatigueDifficulty concentrating or brain fogShoulder pain

Clinical phenotype terms (21)— hover any for plain English
PapilledemaHP:0001085AllergyHP:0012393Scintillating scotomaHP:0010822Abnormal emotional stateHP:0100851Pulsatile tinnitusHP:0008629
Inheritance

Multifactorial

Caused by a mix of several genes and environmental factors

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Feb 2026Tirzepatide in Idiopathic Intracranial Hypertension Trial

Duke University — PHASE4

TrialNOT YET RECRUITING
Jan 2026Venous Stenting Evaluation in Patients With Intracranial Hypertension Under Long-term Acetazolamide

University Hospital, Montpellier — NA

TrialRECRUITING
Mar 2025Advanced Neuroimaging in Idiopathic Intracranial Hypertension

Thomas Jefferson University

TrialRECRUITING
Mar 2025Dural Venous Sinus Stent in Idiopathic Intracranial Hypertension

Assiut University — NA

TrialNOT YET RECRUITING
Jan 2025Prospective Ocular Imaging for Intracranial Pressure Evaluation

King's College Hospital NHS Trust

TrialRECRUITING
Nov 2024Study SOLACE SEPSIS

University Hospital Pilsen — PHASE2

TrialRECRUITING
Jul 2024Pseudotumor Cerebri Headache Ten Years Outcome

Bnai Zion Medical Center

TrialENROLLING BY INVITATION
Jun 2024ICP & Outflow Study

Guy's and St Thomas' NHS Foundation Trust

TrialNOT YET RECRUITING
Jun 2024Current Treatment Methods of Idiopathic Intracranial Hypertension

Assiut University

TrialNOT YET RECRUITING
May 2024Ocular Perfusion in Patients With Idiopathic Intracranial Hypertension

Medical University of Vienna

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Idiopathic intracranial hypertension.

16 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

16 recruitingView all trials with filters →
Phase 31 trial
Stent Implantation Versus Medical Therapy for Idiopathic IntracraniaL Hypertension (SIMPLE)
Phase 3
Actively Recruiting
PI: Dapeng Mo, MD (Beijing Tiantan Hospital) · Sites: Beijing, Beijing Municipality · Age: 1860 yrs
Phase 21 trial
Study SOLACE SEPSIS
Phase 2
Actively Recruiting
· Sites: Pilsen, Czech Republic · Age: 1890 yrs
N/A8 trials
Venous Sinus Stenting With the River Stent in IIH
N/A
Active
PI: Athos Patsalides, MD (Northwell Health) · Sites: Jacksonville, Florida; Buffalo, New York +4 more · Age: 1899 yrs
Multifocal Chromatic Pupilloperimetry in Patients With Pseudotumor Cerebri and Healthy Subjects.
N/A
Actively Recruiting
· Sites: Tel Litwinsky · Age: 1880 yrs
Assessment of Venous Drainage in Idiopathic Intracranial Hypertension
N/A
Active
· Sites: Salouël · Age: 1899 yrs
Stenting Versus Neurosurgical Treatment of Idiopathic Intracranial Hypertension.
N/A
Actively Recruiting
PI: Pierre Bourdillon, MD (Fondation A. de Rothschild) · Sites: Paris, Paris · Age: 1899 yrs
Venous Stenting Evaluation in Patients With Intracranial Hypertension Under Long-term Acetazolamide
N/A
Actively Recruiting
PI: Vincent COSTALAT, MD, PhD (University hospital of Montpellier, FRANCE) · Sites: Montpellier; Reims +1 more · Age: 1899 yrs
Evaluating Raised Intracranial Pressure Using MR Elastography
N/A
Active
PI: John J Chen, M.D., Ph.D. (Mayo Clinic) · Sites: Rochester, Minnesota · Age: 1899 yrs
IIH Intervention: A Clinical Trial Comparing 2 Treatments (Shunts and Stents) Evaluation Of Clinical Effectiveness And Cost Effectiveness
N/A
Actively Recruiting
· Sites: Birmingham; Bristol +13 more · Age: 1863 yrs
Biomarkers in the Etiology of Idiopathic Intracranial Hypertension
N/A
Actively Recruiting
PI: Federico CAGNAZZO, MD (University Hospital of Montpellier - Gui de Chauli) · Sites: Montpellier · Age: 1899 yrs
Other6 trials
Prospective Ocular Imaging for Intracranial Pressure Evaluation
Actively Recruiting
· Sites: London, London · Age: 1899 yrs
Pseudotumor Cerebri Headache Ten Years Outcome
Enrolling by Invitation
PI: jacob Genizi (Bnai Zion Medical Center) · Sites: Haifa
Advanced Neuroimaging in Idiopathic Intracranial Hypertension
Actively Recruiting
PI: Hsiangkuo Yuan, MD PhD (Thomas Jefferson University) · Sites: Philadelphia, Pennsylvania · Age: 1855 yrs
Diagnostic and Prognostic Biomarkers of Idiopathic Intracranial Hypertension
Actively Recruiting
PI: Rigmor Jensen, M.D., Dr.Med. (The Danish Headache Center, Rigshospitalet-Glostru) · Sites: Copenhagen, Glostrup; Odense, Region Syddanmark · Age: 1899 yrs
Astronaut Vision Issues in a Ground Analog Population: Polycystic Ovary Syndrome
Active
PI: Scott M Smith, PhD (National Aeronautics and Space Administration (NAS) · Sites: Rochester, Minnesota · Age: 1850 yrs
Ocular Perfusion in Patients With Idiopathic Intracranial Hypertension
Actively Recruiting
· Sites: Vienna · Age: 1890 yrs

Specialists

Showing 25 of 27View all specialists →
FM
Federico CAGNAZZO, MD
Specialist
PI on 1 active trial
OK
Oğuzhan Kurşun
Specialist
PI on 1 active trial
AP
Alexandra Sinclair, MbChb PhD
Specialist
PI on 1 active trial
JP
John J Chen, M.D., Ph.D.
Specialist
PI on 1 active trial
AM
Alice Y Chang, MD
Specialist
PI on 1 active trial1 Idiopathic intracranial hypertension publication
RD
Rigmor Jensen, M.D., Dr.Med.
Specialist
PI on 1 active trial
SP
Scott M Smith, PhD
Specialist
PI on 1 active trial
KM
Kyle Fargen, MD
WINSTON SALEM, NC
Specialist
PI on 1 active trial
RD
Rigmor Jensen, Dr.med.
Specialist
PI on 1 active trial
DM
Dapeng Mo, MD
Specialist
PI on 1 active trial
ZM
Zhongrong Miao, MD
Specialist
PI on 1 active trial
AM
Athos Patsalides, MD
NEW YORK, NY
Specialist
PI on 1 active trial1 Idiopathic intracranial hypertension publication
AM
Athos Patsalides, MD, MPH
NEW YORK, NY
Specialist
PI on 2 active trials
RP
Rigmor H Jensen, Professor
Specialist
PI on 1 active trial2 Idiopathic intracranial hypertension publications
HP
Hsiangkuo Yuan, MD PhD
PHILADELPHIA, PA
Specialist
PI on 1 active trial
JM
Jai Shankar, MD
VICTORVILLE, CA
Specialist
PI on 2 active trials
DP
Dagmar Beier, M.D., Ph.D.
Specialist
PI on 1 active trial
VP
Vincent COSTALAT, MD, PhD
Specialist
PI on 1 active trial
SP
Sherese Fralin, MSN, FNP, PhD
CHICAGO, IL
Specialist
PI on 1 active trial
AL
Andrew W Lawton
CHICAGO, IL
Specialist
PI on 1 active trial
NA
Naomi Abel
TAMPA, FL
Specialist
PI on 1 active trial
MP
Mohammed Ahmed Zaki, Ass. Prof
Specialist
PI on 1 active trial
MW
Michael A. Williams
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Idiopathic intracranial hypertension.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Idiopathic intracranial hypertension

Disease timeline:

New recruiting trial: Multifocal Chromatic Pupilloperimetry in Patients With Pseudotumor Cerebri and Healthy Subjects.

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

New recruiting trial: Stent Implantation Versus Medical Therapy for Idiopathic IntracraniaL Hypertension (SIMPLE)

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

New recruiting trial: Ocular Perfusion in Patients With Idiopathic Intracranial Hypertension

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

New recruiting trial: Biomarkers in the Etiology of Idiopathic Intracranial Hypertension

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

New recruiting trial: Diagnostic and Prognostic Biomarkers of Idiopathic Intracranial Hypertension

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

New recruiting trial: Prospective Ocular Imaging for Intracranial Pressure Evaluation

A new clinical trial is recruiting patients for Idiopathic intracranial hypertension

Caregiver Resources

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Questions for your doctor

Bring these to your next appointment

  • Q1.How severe is the swelling of my optic nerves, and is my vision currently at risk?,What is my current cerebrospinal fluid pressure, and what is the target we are aiming for?,What are the side effects of acetazolamide, and what should I do if I cannot tolerate it?,How much weight loss is recommended for my situation, and can you refer me to a weight management program?,How often do I need follow-up eye exams and visual field tests?,At what point would surgery be considered, and what are the options?,Are there any activities or medications I should avoid that could worsen my condition?

Common questions about Idiopathic intracranial hypertension

What is Idiopathic intracranial hypertension?

Idiopathic intracranial hypertension (IIH), also known as pseudotumor cerebri or benign intracranial hypertension, is a condition where the pressure of the fluid surrounding the brain (called cerebrospinal fluid or CSF) becomes abnormally high without an obvious cause such as a tumor, infection, or blood clot. The word 'idiopathic' means the exact cause is unknown. This increased pressure pushes on the brain and the nerves behind the eyes, leading to a range of symptoms. The most common symptom is severe, persistent headaches that may worsen with eye movement or straining. Many people also ex

How is Idiopathic intracranial hypertension inherited?

Idiopathic intracranial hypertension follows a multifactorial inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Idiopathic intracranial hypertension typically begin?

Typical onset of Idiopathic intracranial hypertension is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Idiopathic intracranial hypertension?

Yes — 16 recruiting clinical trials are currently listed for Idiopathic intracranial hypertension on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Idiopathic intracranial hypertension?

25 specialists and care centers treating Idiopathic intracranial hypertension are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.