Medullary thyroid carcinoma

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2FDA treatments16Active trials72Specialists8Treatment centers1Financial resources

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

Medullary thyroid carcinoma (MTC) is a type of thyroid cancer that starts in special cells in the thyroid gland called C cells (also called parafollicular cells). These cells normally make a hormone called calcitonin, which helps control calcium levels in the blood. When C cells grow out of control, they form a tumor that can spread to nearby lymph nodes and, in some cases, to other parts of the body like the liver, lungs, or bones. MTC is different from the more common types of thyroid cancer because it does not respond to radioactive iodine treatment. It makes up about 3 to 4 percent of all thyroid cancers. About 75 percent of cases are sporadic, meaning they happen by chance with no family history. The other 25 percent are hereditary, meaning they are passed down in families and are linked to a condition called Multiple Endocrine Neoplasia type 2 (MEN2), which includes MEN2A and MEN2B, or Familial Medullary Thyroid Carcinoma (FMTC). Common symptoms include a lump or swelling in the neck, trouble swallowing, hoarseness, and sometimes diarrhea caused by high calcitonin levels. The main treatment is surgery to remove the thyroid gland. For advanced or spreading disease, targeted drug therapies are available. When caught early, the outlook can be very good, but late-stage disease is harder to treat.

Also known as:

Key symptoms:

A painless lump or nodule in the front of the neckSwollen lymph nodes in the neckHoarse or changed voiceDifficulty swallowingDifficulty breathingPersistent diarrhea (caused by high calcitonin levels)Flushing or redness of the faceNeck pain that may spread to the earUnexplained weight lossFatigueIn MEN2A: symptoms of high calcium (kidney stones, bone pain, muscle weakness) from overactive parathyroid glandsIn MEN2A/MEN2B: high blood pressure or rapid heartbeat from adrenal tumors (pheochromocytoma)

Clinical phenotype terms (14)— hover any for plain English
Medullary thyroid carcinomaHP:0002865Elevated circulating calcitonin concentrationHP:0003528Nodular goiterHP:0005994PheochromocytomaHP:0002666Primary hyperparathyroidismHP:0008200Neoplasm of the skeletal systemHP:0010622Abnormal liver parenchyma morphologyHP:0030146Neoplasm of the lungHP:0100526
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Jul 2026Pressurized Intraperitoneal Aerosolized Chemotherapy With Mitomycin for the Treatment of Unresectable Appendix or Colorectal Cancer With Peritoneal Metastases, The IMPACT Trial

City of Hope Medical Center — PHASE3

TrialNOT YET RECRUITING
Jun 2026SeqUential GeMcitabine and MITomycin Treatment for Favorable High-Risk Upper Urinary Tract Urothelial Carcinoma

Rutgers, The State University of New Jersey — PHASE2

TrialNOT YET RECRUITING
Apr 2026Hyaluronic Acid vs Mitomycin-C in External Dacryocystorhinostomy

All India Institute of Medical Sciences, Bhubaneswar — PHASE4

TrialNOT YET RECRUITING
Mar 2026Development of Patient Derived Xenografts (PDXs) and Analysis of Tyrosine Kinase Receptor Expression in Patients With Squamous Cell Head and Neck Cancer (HNSCC) to Study Resistance Mechanisms Induced by Standard Therapy

IRCCS Azienda Ospedaliero-Universitaria di Bologna

TrialNOT YET RECRUITING
Mar 2026Safety and Efficacy of PIPAC Using Single Agent Mitomycin in Solid Tumors

H. Lee Moffitt Cancer Center and Research Institute — PHASE1

TrialNOT YET RECRUITING
Jan 2026Hyperthermic Intravesical Chemotherapy (HIVEC) in BCG-Non-Responsive High-Grade NMIBC Patients

AC.TA. S.r.l. — NA

TrialNOT YET RECRUITING
Jan 2026Heated Versus Aerosol-based Laparoscopic Chemotherapy for Cancer That Has Spread to the Peritoneum (Abdominal Lining)

Patrick Wagner, MD, FACS — PHASE2

TrialRECRUITING
Oct 2025Toripalimab ± Sequential Intravesical Gemcitabine-Mitomycin C for BCG-Unresponsive/-Intolerant High-Risk NMIBC: Open-Label Randomized Phase 2 Study

First Affiliated Hospital of Wenzhou Medical University — PHASE2

TrialNOT YET RECRUITING
Aug 2025CTR-FAPI-guided Precision Surgery for Newly Diagnosed MTC

Cancer Institute and Hospital, Chinese Academy of Medical Sciences — PHASE3

TrialRECRUITING
Aug 2025A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer

UroGen Pharma Ltd. — PHASE3

TrialRECRUITING

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

Treatments

2 available

CAPRELSA(r)

vandetanib· Genzyme Corporation■ Boxed Warning

indicated for the treatment of symptomatic or progressive medullary thyroid cancer in patients with unresectable locally advanced or metastatic disease

RETEVMO

selpercatinib· Loxo Oncology Inc., a wholly owned subsidiary of Eli Lilly and Company
RETEVMO is indicated for the treatment of adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-ap

RETEVMO is indicated for the treatment of adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy

Clinical Trials

16 recruitingView all trials with filters →
Phase 32 trials
A Study of Selpercatinib (LY3527723) in Participants With RET-Mutant Medullary Thyroid Cancer
Phase 3
Active
PI: Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM (Eli Lilly and Company) · Sites: Birmingham, Alabama; Duarte, California +141 more · Age: 1299 yrs
CTR-FAPI-guided Precision Surgery for Newly Diagnosed MTC
Phase 3
Actively Recruiting
PI: Shaoyan Liu, MD (Chinese Academy of Medical Sciences) · Sites: Beijing, Beijing Municipality
Phase 42 trials
Study of Anlotinib in Patients With Advanced Medullary Thyroid Carcinoma
Phase 4
Actively Recruiting
· Sites: Hefei, Anhui; Beijing, Beijing Municipality +17 more · Age: 1875 yrs
A Study of Two Different Doses of Cabozantinib (XL184) in Progressive, Metastatic Medullary Thyroid Cancer
Phase 4
Active
· Sites: St Leonards, New South Wales; Herston, Queensland +47 more · Age: 1899 yrs
Phase 21 trial
A Study Using Regorafenib as Second or Third Line Therapy in Metastatic Medullary Thyroid Cancer
Phase 2
Active
PI: Kartik Seghal, MD (Dana-Farber Cancer Institute) · Sites: New Haven, Connecticut; Boston, Massachusetts · Age: 1899 yrs
Phase 12 trials
Head-to-head Study of 68Ga-MGS5 Versus 68Ga-DOTATATE PET/CT in Patients With Medullary Thyroid Carcinoma
Phase 1
Actively Recruiting
PI: Weibing Miao (First Affiliated Hospital of Fujian Medical Univer) · Sites: Fuzhou, Fujian · Age: 1880 yrs
GFRα4 CAR T Cells in MTC Patients
Phase 1
Active
PI: Roger Cohen, MD (University of Pennsylvania) · Sites: Philadelphia, Pennsylvania · Age: 1899 yrs
N/A4 trials
Patient Decision Aid in Supporting Decision-Making About When to Start or Stop New Drugs, Join Clinical Trials, or Continue Active Surveillance in Patients With Medullary Thyroid Cancer and Their Caregivers
N/A
Actively Recruiting
PI: Elizabeth G Grubbs, MD (M.D. Anderson Cancer Center) · Sites: Houston, Texas · Age: 1899 yrs
Research on the Application of 68Ga-DOTA-CCK-FS PET/CT in MTC
N/A
Actively Recruiting
PI: Yaping LUO (Peking Union Medical College Hospital) · Sites: Beijing, Beijing Municipality
Thermal Ablation of Cervical Metastases From Thyroid Carcinoma
N/A
Active
PI: Ricardo MC Freitas, PhD (Instituto do Cancer do Estado de São Paulo) · Sites: São Paulo · Age: 1899 yrs
68Ga-TCR-FAPI PET/CT Guided Precision Surgery for MTC
N/A
Actively Recruiting
· Sites: Beijing · Age: 1875 yrs
Other4 trials
Natural History Study of Children and Adults With Medullary Thyroid Cancer
Actively Recruiting
PI: John W Glod, M.D. (National Cancer Institute (NCI)) · Sites: Bethesda, Maryland
PET-TC in Thyroid Evaluation
Enrolling by Invitation
· Sites: Bologna, Bo · Age: 1899 yrs
Clinical and Genetic Studies in Familial Non-medullary Thyroid Cancer
Actively Recruiting
PI: Joanna Klubo-Gwiezdzinska, M.D. (National Institute of Diabetes and Digestive and K) · Sites: Bethesda, Maryland · Age: 790 yrs
Natural History of Medullary Thyroid Cancer to Inform Advanced Disease Management
Actively Recruiting
PI: Elizabeth G Grubbs (M.D. Anderson Cancer Center) · Sites: San Francisco, California; Houston, Texas · Age: 1899 yrs

Specialists

Showing 25 of 72View all specialists →
KM
Kartik Seghal, MD
Specialist
PI on 2 active trials
LM
Laura Locati, MD
Specialist
PI on 1 active trial1 Medullary thyroid carcinoma publication
RD
Ramona Dadu
HOUSTON, TX
Specialist
2 Medullary thyroid carcinoma publications
EM
Esmeralda Isabella Mazza
Specialist
1 Medullary thyroid carcinoma publication
SW
Steven G Waguespack
HOUSTON, TX
Specialist
2 Medullary thyroid carcinoma publications
MH
Mimi I Hu
HOUSTON, TX
Specialist
4 Medullary thyroid carcinoma publications
YZ
Yan Zhang
Specialist
2 Medullary thyroid carcinoma publications
NB
Naifa L Busaidy
HOUSTON, TX
Specialist
2 Medullary thyroid carcinoma publications
CM
Caterina Mian
Specialist
2 Medullary thyroid carcinoma publications
MZ
Mark E Zafereo
HOUSTON, TX
Specialist
2 Medullary thyroid carcinoma publications
MK
Mijin Kim
Specialist
1 Medullary thyroid carcinoma publication
IB
Isabella Merante Boschin
Specialist
1 Medullary thyroid carcinoma publication
BK
Bo Hyun Kim
Specialist
1 Medullary thyroid carcinoma publication
PT
Pierpaolo Trimboli
Specialist
1 Medullary thyroid carcinoma publication
MP
Maria Rosa Pelizzo
Specialist
1 Medullary thyroid carcinoma publication
JQ
Jianwu Qin
Specialist
2 Medullary thyroid carcinoma publications
GM
Giuseppe Procopio, MD
RAHWAY, NJ
Specialist
PI on 8 active trials1 Medullary thyroid carcinoma publication
AA
Andrea B Apolo
Birmingham, Alabama
Specialist

Rare Disease Specialist

PI on 4 active trials
MM
Michael Goggins, MD
PHILADELPHIA, PA
Specialist
PI on 2 active trials
MM
Margaret von Mehren
Specialist
PI on 4 active trials1 Medullary thyroid carcinoma publication
MS
Manisha Shah
Specialist
PI on 4 active trials177 Medullary thyroid carcinoma publications
SA
Srivandana Akshintala
WASHINGTON, DC
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

1 resources
RETEVMO(selpercatinib)Loxo Oncology Inc., a wholly owned subsidiary of Eli Lilly and Company

Travel Grants

No travel grants are currently matched to Medullary thyroid carcinoma.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Medullary thyroid carcinoma

Disease timeline:

New recruiting trial: Clinical Trial With Donor Modified Immune Cells in Living Donor Kidney Transplantation

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: Investigating the Efficacy and Safety of Neoadjuvant Intravesical Instillation of Mitomycin C in Treating High-risk NMIBC Patients

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: A First-in-Human Escalation and Expansion Study of Patients With Advanced Solid Tumors

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: Clinical Trial on HIPEC with Mitomycin C in Colon Cancer Peritoneal Metastases (GECOP-MMC)

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: Heated Versus Aerosol-based Laparoscopic Chemotherapy for Cancer That Has Spread to the Peritoneum (Abdominal Lining)

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: Intracameral Versus Intravitreal Bevacizumab Injection in NVG: RCT

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: uTRACT Jelmyto Registry: A Registry of Patients With Upper Tract Urothelial Cancer (UTUC) Treated With Jelmyto

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: MTC Versus FMT in for RCDI

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

New recruiting trial: HYbrid RObotic Surgery MulTiCentric Study

A new clinical trial is recruiting patients for Medullary thyroid carcinoma

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.Should I have genetic testing for the RET gene, and what does a positive result mean for me and my family?,Has my cancer spread beyond the thyroid, and what staging tests do I need?,What type of surgery do you recommend, and will lymph nodes be removed?,After surgery, how will we monitor for cancer coming back, and how often will I need blood tests and scans?,If my cancer is advanced or comes back, which targeted drug therapies would be appropriate for me?,Should my children, siblings, or parents be tested for a RET mutation?,Are there any clinical trials I should consider?

Common questions about Medullary thyroid carcinoma

What is Medullary thyroid carcinoma?

Medullary thyroid carcinoma (MTC) is a type of thyroid cancer that starts in special cells in the thyroid gland called C cells (also called parafollicular cells). These cells normally make a hormone called calcitonin, which helps control calcium levels in the blood. When C cells grow out of control, they form a tumor that can spread to nearby lymph nodes and, in some cases, to other parts of the body like the liver, lungs, or bones. MTC is different from the more common types of thyroid cancer because it does not respond to radioactive iodine treatment. It makes up about 3 to 4 percent of all

Are there clinical trials for Medullary thyroid carcinoma?

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

Which specialists treat Medullary thyroid carcinoma?

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

What treatment and support options exist for Medullary thyroid carcinoma?

2 patient support programs are currently tracked on UniteRare for Medullary thyroid carcinoma. See the treatments and support programs sections for copay assistance, eligibility, and contact details.