Craniopharyngioma

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:54595D44.4
Who is this for?
Show terms as
8Active trials29Specialists8Treatment centers

Where are you in your journey?

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

Overview

Craniopharyngioma is a rare, slow-growing brain tumor that forms near the pituitary gland and hypothalamus — two very important areas at the base of the brain that control hormones, growth, and many body functions. Even though craniopharyngiomas are not cancerous (they are classified as benign or low-grade tumors), they can cause serious problems because of where they grow. As the tumor expands, it can press on nearby structures including the optic nerves (which carry vision signals), the pituitary gland, and the hypothalamus. The most common symptoms include headaches, vision problems, and signs of hormone imbalances such as slow growth in children, fatigue, weight gain, and problems with thirst and urination. Many people are diagnosed after noticing changes in their vision or after a child stops growing normally. The tumor can also cause increased pressure inside the skull, which leads to nausea and vomiting. Treatment usually involves surgery to remove as much of the tumor as safely possible, often followed by radiation therapy. Because the tumor sits near such delicate brain structures, complete removal is not always possible without causing harm. Hormone replacement therapy is almost always needed after treatment because the pituitary gland is often damaged. Long-term follow-up with a team of specialists is essential, as the tumor can come back and the effects on hormones and quality of life can be lifelong.

Key symptoms:

Persistent or worsening headachesBlurred or double visionLoss of peripheral (side) visionNausea and vomitingUnusual thirst and frequent urination (diabetes insipidus)Unexplained weight gainExtreme tiredness and low energySlow or stopped growth in childrenDelayed puberty or loss of menstrual periodsMemory problems or difficulty concentratingMood changes or depressionSensitivity to cold temperaturesLow blood pressure or dizziness

Clinical phenotype terms (46)— hover any for plain English
Abnormal hypothalamus morphologyHP:0012286Intracranial cystic lesionHP:0010576Neoplasm of the anterior pituitaryHP:0011750Enlarged pituitary glandHP:0012505HypopituitarismHP:0040075Central diabetes insipidusHP:0000863PapilledemaHP:0001085Slow decrease in visual acuityHP:0007924Progressive visual field defectsHP:0007987Central adrenal insufficiencyHP:0011734
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

9 events
Feb 2026Food Preferences and Craniopharyngiomas

Assistance Publique - Hôpitaux de Paris

TrialNOT YET RECRUITING
Jul 2025ImmunoPET Targeting Trophoblast Cell-surface Antigen 2 (Trop-2) in Craniopharyngioma Patients

Deling Li — NA

TrialRECRUITING
Mar 2025Craniopharyngioma and Pregnancies

Assistance Publique - Hôpitaux de Paris

TrialRECRUITING
Sep 2023Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma

Eva Marie Erfurth, MD, PhD — PHASE2

TrialRECRUITING
Sep 2022Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults

Sabine Mueller, MD, PhD — PHASE2

TrialRECRUITING
Mar 2019Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy

University of Colorado, Denver

TrialENROLLING BY INVITATION
Jan 2018Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma

Alliance for Clinical Trials in Oncology — PHASE2

TrialACTIVE NOT RECRUITING
Jun 2016A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection

St. Jude Children's Research Hospital — PHASE2

TrialACTIVE NOT RECRUITING
Aug 2011A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma or Observation After Radical Resection

St. Jude Children's Research Hospital — PHASE2

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Craniopharyngioma.

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

View clinical trials →

Clinical Trials

8 recruitingView all trials with filters →
Phase 25 trials
Vemurafenib and Cobimetinib in Treating Patients With BRAF V600E Mutation Positive Craniopharyngioma
Phase 2
Active
PI: Priscilla K. Brastianos, MD (Massachusetts General Hospital) · Sites: Birmingham, Alabama; Auburn, California +104 more · Age: 1899 yrs
Treatment of BRAF ( B-Rapidly Accelerated Fibrosarcoma) Mutated Papillary Craniopharyngioma
Phase 2
Actively Recruiting
PI: Peter Siesjö, MD. PhD. (Department of Neurosurgery, SUS, Lund Sweden) · Sites: Lund · Age: 1899 yrs
A Phase II Trial of Intensity-Modulated Proton Therapy for Incompletely Resected Craniopharyngioma and Observation for Craniopharyngioma After Radical Resection
Phase 2
Active
PI: Thomas E. Merchant, DO, PhD (St. Jude Children's Research Hospital) · Sites: Memphis, Tennessee · Age: 021 yrs
Tovorafenib for Treatment of Craniopharyngioma in Children and Young Adults
Phase 2
Actively Recruiting
PI: Sabine Mueller, MD, PhD, MAS (University of California, San Francisco) · Sites: Birmingham, Alabama; San Diego, California +17 more · Age: 139 yrs
A Phase II Trial of Limited Surgery and Proton Therapy for Craniopharyngioma or Observation After Radical Resection
Phase 2
Active
PI: Thomas E Merchant, DO, PhD (St. Jude Children's Research Hospital) · Sites: Jacksonville, Florida; Memphis, Tennessee · Age: 021 yrs
N/A1 trial
ImmunoPET Targeting Trophoblast Cell-surface Antigen 2 (Trop-2) in Craniopharyngioma Patients
N/A
Actively Recruiting
PI: Deling Li, MD (Beijing Tiantan Hospital) · Sites: Beijing, Beijing Municipality · Age: 1480 yrs
Other2 trials
Craniopharyngioma and Pregnancies
Actively Recruiting
PI: Anne BACHELOT, MD, PhD (Assistance Publique - Hôpitaux de Paris) · Sites: Paris · Age: 1899 yrs
Prospective Pilot Study Identifying Clinically Relevant Biological Targets for Medical Therapy
Enrolling by Invitation
PI: Todd Hankinson, MD (University of Colorado, Denver) · Sites: Aurora, Colorado · Age: 021 yrs

Specialists

Showing 25 of 29View all specialists →
MM
Maryam Fouladi, MD
COLUMBUS, OH
Specialist
PI on 10 active trials
TP
Thomas E. Merchant, DO, PhD
MEMPHIS, TN
Specialist
PI on 3 active trials
SP
Stanislaw R. Burzynski, MD, PhD
HOUSTON, TX
Specialist
PI on 29 active trials
TM
Tobey MacDonald
WASHINGTON, DC
Specialist
PI on 2 active trials5 Craniopharyngioma publications
JM
Jana Portnow, MD
DUARTE, CA
Specialist
PI on 3 active trials
AC
Alexander Colevas
PALO ALTO, CA
Specialist
PI on 2 active trials1 Craniopharyngioma publication
VP
Valerie Crabtree, PhD
STARKVILLE, MS
Specialist
PI on 2 active trials
DE
Danielle Eekers
Specialist
PI on 1 active trial
JJ
J Jaspers
WENATCHEE, WA
Specialist
PI on 1 active trial
IC
Inge Compter
Specialist
PI on 1 active trial
AG
Amar Gajjar
MEMPHIS, TN
Specialist
PI on 4 active trials4 Craniopharyngioma publications
JZ
Jaap Zindler
Specialist
PI on 1 active trial
AO
Antonio Omuro
STANFORD, CA
Specialist
PI on 1 active trial
HW
Hiske van der Weide
Specialist
PI on 1 active trial1 Craniopharyngioma publication
AR
A Mendez Romero
Specialist
PI on 1 active trial11 Craniopharyngioma publications
YK
Yvonne Klaver
Specialist
PI on 1 active trial
MK
M Kroesen
MILFORD, MA
Specialist
PI on 1 active trial
SM
Scot C. Remick, MD
SCARBOROUGH, ME
Specialist
PI on 9 active trials
KW
Katherine Warren
Specialist
PI on 1 active trial
JM
John Suh, MD
Specialist
PI on 1 active trial
HM
Hermann L Müller, MD
Specialist
PI on 1 active trial
BM
Bernd Hofmann, MD
SAN DIEGO, CA
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 Craniopharyngioma.

Search all travel grants →NORD Financial Assistance ↗

Community

Open CraniopharyngiomaForum →

No community posts yet. Be the first to share your experience with Craniopharyngioma.

Start the conversation →

Latest news about Craniopharyngioma

No recent news articles for Craniopharyngioma.

Follow this condition to be notified when news becomes available.

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What subtype of craniopharyngioma do I have, and does that affect my treatment options?,What is the goal of surgery — complete removal or partial removal — and what are the risks to my vision and hormone function?,Will I need radiation therapy, and if so, what type is best for my situation?,Which hormones will I need to replace, and how will we monitor whether the doses are right?,What is the chance the tumor will come back, and how often will I need MRI scans?,Are there any clinical trials or targeted therapies I should consider?,What support is available for managing weight, mood, memory, and school or work challenges?

Common questions about Craniopharyngioma

What is Craniopharyngioma?

Craniopharyngioma is a rare, slow-growing brain tumor that forms near the pituitary gland and hypothalamus — two very important areas at the base of the brain that control hormones, growth, and many body functions. Even though craniopharyngiomas are not cancerous (they are classified as benign or low-grade tumors), they can cause serious problems because of where they grow. As the tumor expands, it can press on nearby structures including the optic nerves (which carry vision signals), the pituitary gland, and the hypothalamus. The most common symptoms include headaches, vision problems, and s

How is Craniopharyngioma inherited?

Craniopharyngioma follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for Craniopharyngioma?

Yes — 8 recruiting clinical trials are currently listed for Craniopharyngioma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Craniopharyngioma?

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