Hereditary nonpolyposis colon cancer

Last reviewed

🖨 Print for my doctorAdvocacy Hub →
ORPHA:443909
Who is this for?
Show terms as
9FDA treatments10Active trials36Specialists8Treatment centers8Financial resources

Where are you in your journey?

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

Overview

Hereditary nonpolyposis colon cancer (HNPCC), also widely known as Lynch syndrome, is an inherited condition that significantly raises a person's risk of developing certain cancers — most commonly colorectal (colon and rectal) cancer, but also cancers of the uterus (endometrium), ovaries, stomach, small intestine, urinary tract, bile ducts, and brain. Unlike some other inherited colon cancer syndromes, HNPCC does not typically cause large numbers of polyps (growths) in the colon, which is why it is called 'nonpolyposis.' The condition is caused by inherited changes (mutations) in genes that normally help repair mistakes in DNA. When these repair genes do not work properly, errors build up in cells over time, which can lead to cancer. People with HNPCC may not have any symptoms until a cancer develops. When colorectal cancer does occur, symptoms can include changes in bowel habits, blood in the stool, abdominal pain, unexplained weight loss, and fatigue. Women with HNPCC also face a high lifetime risk of uterine cancer, which may cause abnormal vaginal bleeding. The good news is that with regular screening and surveillance — such as colonoscopies starting at a young age — cancers can often be caught early or even prevented. Preventive surgeries are also an option for some people. Genetic counseling and testing are key parts of managing this condition for both patients and their family members.

Also known as:

Key symptoms:

Changes in bowel habits (diarrhea, constipation, or narrowing of stool) lasting more than a few daysBlood in the stool or rectal bleedingPersistent abdominal cramping or painUnexplained weight lossFeeling that the bowel does not empty completelyUnusual tiredness or fatigueAbnormal vaginal bleeding or bleeding between periods (in women, may signal uterine cancer)Pelvic pain (in women)Nausea or vomiting that does not go awayUrinary symptoms such as blood in the urine (if urinary tract cancer develops)Jaundice or yellowing of the skin (if bile duct cancer develops)

Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Jun 2026Preventive Dendritic Cell Vaccination for Lynch Syndrome

Radboud University Medical Center — PHASE1, PHASE2

TrialNOT YET RECRUITING
Apr 2026Study Aiming to Test Whether Non-invasive Liquid Biopsies Can Safely Reduce Invasive Surveillance Methods in Lynch Syndrome

UNICANCER — NA

TrialNOT YET RECRUITING
Mar 2026Overcoming Barriers to Uptake of Cascade Screening

UNC Lineberger Comprehensive Cancer Center — NA

TrialNOT YET RECRUITING
Feb 2026Evaluation of Capsule Colonoscopy in Patients With Lynch Syndrome

Region Skane

TrialNOT YET RECRUITING
Dec 2025Determining the Prevalence of Muir-Torre Syndrome in Patients With Lynch Syndrome

Centre Hospitalier Universitaire de Nīmes — NA

TrialNOT YET RECRUITING
Sep 2025First in Human Pilot Study to Assess the Safety and Efficacy of Dendritic Cells Loaded With Frameshift Derived Neopeptides for the Prevention of Cancer in of Lynch Syndrome Carriers

Fundacion Clinic per a la Recerca Biomédica — PHASE1

TrialRECRUITING
Jul 2025Lynch Syndrome in Colorectal Cancer Surgery

Izmir Ataturk Training and Research Hospital

TrialNOT YET RECRUITING
Mar 2025A Study to Assess the Safety, Tolerability, and Efficacy of NDI-219216 in Patients With Advanced Solid Tumors.

Nimbus Wadjet, Inc. — PHASE1, PHASE2

TrialRECRUITING
Mar 2025Predictive Value of PREMM5, MMRpredict, and Universal Tumor Screening for Lynch Syndrome in Vietnam

University of Medicine and Pharmacy at Ho Chi Minh City

TrialNOT YET RECRUITING
Jan 2025DYNAMICS-LYNCH:DNA Methylation Analysis in Stool for Screening of Lynch Syndrome-Associated Colorectal Cancer

Sun Yat-sen University

TrialNOT YET RECRUITING

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

Treatments

9 available

LONSURF

trifluridine/tipiracil· Taiho Oncology, Inc.
LONSURF, as a single agent or in combination with bevacizumab, is indicated for the treatment of adult patients with metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin

LONSURF, as a single agent or in combination with bevacizumab, is indicated for the treatment of adult patients with metastatic colorectal cancer previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF biological therapy, and if RAS wild-type, an anti-EGFR therapy.

Braftovi

encorafenib· Array BioPharma Inc. (a wholly owned subsidiary of Pfizer Inc.)
in combination with cetuximab and fluorouracil-based chemotherapy, for the treatment of adult patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-author

in combination with cetuximab and fluorouracil-based chemotherapy, for the treatment of adult patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-authorized test

Avastin

bevacizumab· Genentech, Inc.
Avastin is a vascular endothelial growth factor inhibitor indicated for the treatment of: Metastatic colorectal cancer, in combination with intravenous fluorouracil-based chemotherapy for first- or se

Avastin is a vascular endothelial growth factor inhibitor indicated for the treatment of: Metastatic colorectal cancer, in combination with intravenous fluorouracil-based chemotherapy for first- or second-line treatment.

Stivarga

regorafenib· Bayer HealthCare Pharmaceuticals, Inc.■ Boxed Warning
indicated for the treatment of adult patients with metastatic colorectal cancer (CRC) who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-V

indicated for the treatment of adult patients with metastatic colorectal cancer (CRC) who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if RAS wild-type, an anti-EGFR therapy

Tukysa

tucatinib· Seagen Inc.Accelerated Approval
in combination with trastuzumab for the treatment of adult patients with RAS wild-type HER2-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyr

in combination with trastuzumab for the treatment of adult patients with RAS wild-type HER2-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy

CYRAMZA

ramucirumab· Eli Lilly and Company

in combination with FOLFIRI, for the treatment of adults with metastatic colorectal cancer with disease progression on or after prior therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine

Erbitux

cetuximab· ImClone Systems Incorporated■ Boxed Warning

in combination with encorafenib, for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, as detected by an FDA-approved test, after prior therapy

Fusilev

levoleucovorin· Acrotech Biopharma LLC

treatment of adults with metastatic colorectal cancer in combination with fluorouracil

Lumakras

sotorasib· Amgen Inc.Orphan Drug
in combination with panitumumab, for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC), as determined by an FDA-approved test, who have received prior fluoropy

in combination with panitumumab, for the treatment of adult patients with KRAS G12C-mutated metastatic colorectal cancer (mCRC), as determined by an FDA-approved test, who have received prior fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy

Clinical Trials

10 recruitingView all trials with filters →
Phase 31 trial
Effect of Chemoprevention by Low-dose Aspirin of New or Recurrent Colorectal Adenomas in Patients With Lynch Syndrome
Phase 3
Active
PI: Robert BENAMOUZIG, Pr (Assistance Publique - Hôpitaux de Paris) · Sites: Bobigny · Age: 1875 yrs
Phase 21 trial
Testing a Combination of Vaccines for Cancer Prevention in Lynch Syndrome
Phase 2
Active
PI: Ajay Bansal (University of Kansas) · Sites: Phoenix, Arizona; Tucson, Arizona +12 more · Age: 1899 yrs
Phase 12 trials
Cancer Preventive Vaccine Nous-209 for Lynch Syndrome Patients
Phase 1
Active
PI: Eduardo Vilar-Sanchez (M.D. Anderson Cancer Center) · Sites: Duarte, California; Philadelphia, Pennsylvania +2 more · Age: 1899 yrs
First in Human Pilot Study to Assess the Safety and Efficacy of Dendritic Cells Loaded With Frameshift Derived Neopeptides for the Prevention of Cancer in of Lynch Syndrome Carriers
Phase 1
Actively Recruiting
· Sites: Barcelona, Barcelona · Age: 1899 yrs
N/A2 trials
A Study for Imaging the Lower Gastrointestinal Tract Using a Retro-TCE Capsule
N/A
Actively Recruiting
PI: Guillermo Tearney, M.D., PhD (Massachusetts General Hospital) · Sites: Boston, Massachusetts · Age: 1875 yrs
CAD-EYE System for the Detection of Neoplastic Lesions in Patients With Lynch Syndrome
N/A
Actively Recruiting
· Sites: Paris · Age: 1890 yrs
Other3 trials
Lynch Syndrome X-Talk of Enteral Mucosa With Immune System
Actively Recruiting
PI: Giulia Martina Cavestro, MD, PhD (IRCCS San Raffaele Scientific Institute) · Sites: Monrovia, California; Milan, Lombardy +3 more · Age: 1899 yrs
Multi-Organ Screening Recommendations in Patients With Lynch Syndrome
Active
PI: Zsofia Stadler, MD (Memorial Sloan Kettering Cancer Center) · Sites: Basking Ridge, New Jersey; Middletown, New Jersey +6 more · Age: 1899 yrs
Metagenomic Evaluation of the Gut Microbiome in Patients With Lynch Syndrome and Other Hereditary Colonic Polyposis Syndromes
Active
PI: Zsofia Stadler, MD (Memorial Sloan Kettering Cancer Center) · Sites: New York, New York · Age: 1899 yrs

Specialists

Showing 25 of 36View all specialists →
MM
Michael Goggins, MD
PHILADELPHIA, PA
Specialist
PI on 2 active trials
FS
Frank A Sinicrope
ROCHESTER, MN
Specialist
PI on 2 active trials
RP
Robert BENAMOUZIG, Pr
Specialist
PI on 2 active trials
AP
Ann-Sofie Backman, MD PhD
Specialist
PI on 1 active trial
AS
Ann (Annie) W Silk
Los Angeles, California
Specialist

Rare Disease Specialist

PI on 1 active trial
AB
Ajay Bansal
KANSAS CITY, MO
Specialist
PI on 1 active trial41 Hereditary nonpolyposis colon cancer publications
KL
Karen Lu
HOUSTON, TX
Specialist
PI on 1 active trial31 Hereditary nonpolyposis colon cancer publications
KM
Kim E. Nichols, MD
Memphis, Tennessee
Specialist

Rare Disease Specialist

PI on 2 active trials
YM
Ying Yuan, MD
RENO, NV
Specialist
PI on 1 active trial
HP
Hans F Vasen, MD PhD
Specialist
PI on 1 active trial
JM
Jose Rauh-Hain, MD,MPH
HOUSTON, TX
Specialist
PI on 1 active trial
NM
Niloy Jewel Samadder, M.D.
SCOTTSDALE, AZ
Specialist
PI on 1 active trial
JP
Jan J Koornstra, MD PhD
Specialist
PI on 2 active trials
JP
Jan H Kleibeuker, MD PhD
Specialist
PI on 1 active trial
AD
Anja Wagner, Dr.
Specialist
PI on 1 active trial
JP
Jussi Nikkola, MD, PhD
Specialist
PI on 1 active trial
MP
Maria C Katapodi, PhD
Specialist
PI on 1 active trial
SP
Susan Peterson, PhD
Specialist
PI on 4 active trials
KB
Karen M Basen-Engquist
Specialist
PI on 2 active trials
DM
Debra Mathews, PhD, MA
SACRAMENTO, CA
Specialist
PI on 1 active trial126 Hereditary nonpolyposis colon cancer publications
KP
Katrina AB Goddard, PhD
TACOMA, WA
Specialist
PI on 1 active trial
MJ
Michelle N Meyer, PhD, JD
Specialist
PI on 1 active trial
AP
Amanda J Cross, PhD
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

Financial Resources

8 resources
LONSURF(trifluridine/tipiracil)Taiho Oncology, Inc.
Braftovi(encorafenib)Array BioPharma Inc. (a wholly owned subsidiary of Pfizer Inc.)
Avastin(bevacizumab)Genentech, Inc.
Stivarga(regorafenib)Bayer HealthCare Pharmaceuticals, Inc.
CYRAMZA(ramucirumab)Eli Lilly and Company
Fusilev(levoleucovorin)Acrotech Biopharma LLC
Lumakras(sotorasib)Amgen Inc.

Qfitlia

Takeda Pharmaceuticals

Metastatic Colorectal Cancer

Verified 1d ago
copay card
Copay CardPatient Assistance
Accepting applications

Travel Grants

No travel grants are currently matched to Hereditary nonpolyposis colon cancer.

Search all travel grants →NORD Financial Assistance ↗

Community

Open Hereditary nonpolyposis colon cancerForum →

No community posts yet. Be the first to share your experience with Hereditary nonpolyposis colon cancer.

Start the conversation →

Latest news about Hereditary nonpolyposis colon cancer

Disease timeline:

New recruiting trial: How Epigenetic Changes in hMLH1 Connect Lab Research With Diagnosis in Gastric Cancer

A new clinical trial is recruiting patients for Hereditary nonpolyposis colon cancer

New recruiting trial: First in Human Pilot Study to Assess the Safety and Efficacy of Dendritic Cells Loaded With Frameshift Derived Neopeptides for the Prevention of Cancer in of Lynch Syndrome Carriers

A new clinical trial is recruiting patients for Hereditary nonpolyposis colon cancer

New recruiting trial: Provider-Mediated Communication of Genetic Testing Results to At-Risk Relatives of Cancer Patients to Improve Genetic Counseling and Testing Rates, Family HOPE Study

A new clinical trial is recruiting patients for Hereditary nonpolyposis colon cancer

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.Which gene mutation do I have, and what does that mean for my specific cancer risks?,What screening tests do I need, and how often should I have them?,Should I consider preventive surgery, and what are the pros and cons for my situation?,Should my children, siblings, and parents be tested for this condition?,Are there any medications or lifestyle changes that can lower my cancer risk?,What signs or symptoms should prompt me to seek medical attention right away?,Are there clinical trials I should know about for Lynch syndrome prevention or treatment?

Common questions about Hereditary nonpolyposis colon cancer

What is Hereditary nonpolyposis colon cancer?

Hereditary nonpolyposis colon cancer (HNPCC), also widely known as Lynch syndrome, is an inherited condition that significantly raises a person's risk of developing certain cancers — most commonly colorectal (colon and rectal) cancer, but also cancers of the uterus (endometrium), ovaries, stomach, small intestine, urinary tract, bile ducts, and brain. Unlike some other inherited colon cancer syndromes, HNPCC does not typically cause large numbers of polyps (growths) in the colon, which is why it is called 'nonpolyposis.' The condition is caused by inherited changes (mutations) in genes that no

How is Hereditary nonpolyposis colon cancer inherited?

Hereditary nonpolyposis colon cancer follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Hereditary nonpolyposis colon cancer typically begin?

Typical onset of Hereditary nonpolyposis colon cancer is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Hereditary nonpolyposis colon cancer?

Yes — 10 recruiting clinical trials are currently listed for Hereditary nonpolyposis colon cancer on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Hereditary nonpolyposis colon cancer?

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

What treatment and support options exist for Hereditary nonpolyposis colon cancer?

1 patient support program are currently tracked on UniteRare for Hereditary nonpolyposis colon cancer. See the treatments and support programs sections for copay assistance, eligibility, and contact details.