KIT Gene Mutations: Linked Conditions and Clinical Trials

The KIT gene sits on chromosome 4q12, and its full name is KIT proto-oncogene, receptor tyrosine kinase. Orphanet links KIT to 18 rare conditions and lists variants in the gene as a cause of 16 of them. Trial Friend's Mastocytosis, Systemic Mastocytosis and Gastrointestinal Stromal Tumor pages list KIT among the genes behind each condition. As of September 29, 2026, 14 recruiting or soon-opening trials on ClinicalTrials.gov name KIT as a gene.

Other symbols for KIT in gene databases: C-Kit, CD117, SCFR.

See the trialsSearch another gene

What the KIT gene does

KIT makes a receptor on the surface of certain cells that switches on when a protein called stem cell factor attaches to it. Its signals control cell growth, division, survival and movement.

Summarized from the KIT page on MedlinePlus Genetics, U.S. National Library of Medicine, checked September 29, 2026.

Conditions linked to KIT on Trial Friend

These condition pages list KIT among the genes behind the condition, checked against genetics references when each page was written.

Mastocytosis

Most systemic mastocytosis carries an acquired (somatic) activating KIT variant tested in blood or bone marrow; it is not inherited. Additional somatic variants (e.g. TET2, SRSF2, ASXL1, RUNX1) mark more aggressive disease.

Systemic Mastocytosis

Most cases carry a somatic activating KIT variant (usually D816V) in mast cells; additional somatic changes in TET2, SRSF2, ASXL1, RUNX1 or DNMT3A mark more aggressive disease.

Gastrointestinal Stromal Tumor

About 80% of GISTs carry a somatic KIT mutation and 10% PDGFRA; SDH-deficient GIST (under 10%) can reflect a germline SDHx variant, and rare familial KIT/PDGFRA GIST is autosomal dominant.

KIT mutation clinical trials

These recruiting or soon-opening trials mention KIT as a gene in their title, summary, eligibility rules or keywords. Some want people with a variant in it, and others study it more broadly, which is why each trial's own words are shown. In cancer trials the gene change is usually one found in the tumor rather than one a person inherited.

14 trials, checked September 29, 2026. The list refreshes from ClinicalTrials.gov every few hours.

RecruitingPhase 1NCT07171203
Preoperative Imatinib and Fampridine in KIT Mutant Gastrointestinal Stromal Tumor
Gastrointestinal Stromal Tumors
Why it's listed: “…patients with gastrointestinal stromal tumor (GIST) with a DNA mutation in exon 11 of the KIT gene.”
RecruitingPhase 3NCT05208047
(Peak) A Phase 3 Randomized Trial of CGT9486+Sunitinib vs. Sunitinib in Subjects With Gastrointestinal Stromal Tumors
Advanced Gastrointestinal Stromal Tumors · Metastatic Cancer
Why it's listed: “…and sunitinib as first-line (1L) treatment of GIST in approximately 40 participants with KIT exon 9 mutations and no prior systemic therapy (with the exception of up to 10 subjects with ongoing imati…”
RecruitingNCT04557969
Surgery in Gastrointestinal Stromal Tumors (GISTs) for Treatment, Tumor Modeling, and Genomic Analysis
Gastric Cancer · Gastric Neoplasm · Gastrointestinal Stromal Sarcoma
Why it's listed: Lists KIT among its conditions or keywords
RecruitingPhase 2NCT07559864
Comparing Regorafenib Combined With Envafolimab to Physician's Choice in Patients With Metastatic Gastrointestinal Stromal Tumors Harboring KIT Exon 17 Mutations Refractory to Standard Treatment
GIST - Gastrointestinal Stromal Tumor · KIT Gene Mutation
Why it's listed: “Primary or secondary KIT exon 17 mutation detected by genetic testing;”
RecruitingNCT07562542
Peripheral Blood KIT-D816V Mutation in Adult Systemic Mastocytosis
Systemic Mastocytosis · KIT-D816V Mutation
Why it's listed: “…ational study aims to evaluate the diagnostic value and clinical utility of detecting the KIT-D816V mutation in the peripheral blood of adult patients with systemic mastocytosis (SM), using droplet…”
RecruitingPhase 1, Phase 2NCT07028073
A New Treatment of Newly Diagnosed KIT Mutation CBF-Acute Myeloid Leukemia
Acute Myeloid Leukemia With T(8;21)(Q22;Q22) · Acute Myeloid Leukemia With T(16;16)(P13;Q22) · KIT Gene Mutation
Why it's listed: “…n therapy works to treat newly diagnosed adult acute myeloid leukemia (AML) patients with KIT mutations and t(8;21)(q22;q22.1); inv(16)(p13.1q22) or t(16;16)(p13.1;q22). It will also investigat…”
RecruitingPhase 1, Phase 2NCT06630234
A Master Protocol to Evaluate DCC-3009 in Gastrointestinal Stromal Tumor (GIST)
Gastrointestinal Stromal Tumor (GIST)
Why it's listed: Lists KIT among its conditions or keywords
RecruitingPhase 2, Phase 3NCT06972641
Molecular Genetics Guide the Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation
AML · MDS
Why it's listed: “…tion, no TP53 mutation, and no AML/MR. ARM3 Initial bone marrow gene II sequencing showed KIT mutations (loci: D816, N822, exon 8, VAF ≥2%, excluding germline mutations); no TP53 mutations, AML…”
RecruitingNCT07143669
Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations
Clonal Mast Cell Disease · KIT D816V Mutation · Suspected KITD816V Mutated Clonal Mast Cell Disease
Why it's listed: “This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.”
RecruitingPhase 2NCT06316960
Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation
AML, Childhood · Relapse/Recurrence · Refractory AML
Why it's listed: “…tinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.”
RecruitingPhase 2NCT05366816
ctDNA-Guided Sunitinib And Regorafenib Therapy for GIST
Gastrointestinal Stromal Tumors
Why it's listed: “…f DNA or RNA containing information that has the instructions for making proteins) in the KIT gene can be used to predict the body's response to standard of care treatment.”
RecruitingPhase 2NCT04116541
A Study Evaluating the Activity of Anti-cancer Treatments Targeting Tumor Molecular Alterations/Characteristics in Advanced / Metastatic Tumors.
Malignant Solid Tumor
Why it's listed: “Cohort Avapritinib : Activating mutations of KIT exon 17 or PDGFRA exon 18 associated or not to mutation on KIT exon 11 or PDGFRA exon 12/14”
Opening soonPhase 2NCT06772233
A Study of Regorafenib Combined With Envafolimab for Metastatic Gastrointestinal Stromal Tumors With Kit Gene Exon 17 Mutation That Failed Standard Treatment
Metastatic Gastrointestinal Stromal Tumors (GIST)
Why it's listed: “…physician's choice in patients with metastatic gastrointestinal stromal tumors harboring KIT exon 17 mutations who have failed standard treatments.”
Opening soonPhase 2NCT06765915
Avapritinib Maintenance for AML With KIT Mutations
AML, Adult
Why it's listed: “…logeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients with KIT mutation.”

All conditions linked to KIT gene mutations

Orphanet records how each gene relates to each condition. Orphanet links KIT to 18 rare conditions and lists variants in the gene as a cause of 16 of them.

  • Causes it. Disease-causing variants in this gene are a direct cause of the condition.
  • Causes it, acquired. Variants that arise during life in the tumor or affected cells cause the condition. They are not inherited.
  • Tested as a marker. The gene is tested to help diagnose or classify the condition rather than as its cause.
Bullous diffuse cutaneous mastocytosisCauses itTrial Friend pageOrphanet
Gastrointestinal stromal tumorCauses it, acquiredTrial Friend pageOrphanet
Isolated bone marrow mastocytosisCauses it, acquiredTrial Friend pageOrphanet
Pseudoxanthomatous diffuse cutaneous mastocytosisCauses itTrial Friend pageOrphanet
Smoldering systemic mastocytosisCauses it, acquiredTrial Friend pageOrphanet
Systemic mastocytosis with associated hematologic neoplasmCauses it, acquiredTrial Friend pageOrphanet
Acute mast cell leukemiaCauses itOrphanet
Acute myeloblastic leukemia with maturationCauses it, acquiredOrphanet
Chronic mast cell leukemiaCauses itOrphanet
Cutaneous mastocytomaCauses itOrphanet
Nodular urticaria pigmentosaCauses itOrphanet
PiebaldismCauses itOrphanet
Plaque-form urticaria pigmentosaCauses itOrphanet
Telangiectasia macularis eruptiva perstansCauses itOrphanet
Testicular seminomatous germ cell tumorCauses it, acquiredOrphanet
Typical urticaria pigmentosaCauses itOrphanet
Acute myeloid leukemia with abnormal bone marrow eosinophils inv(16)(p13q22) or t(16;16)(p13;q22)Tested as a markerOrphanet
Acute myeloid leukemia with t(8;21)(q22;q22) translocationTested as a markerOrphanet

KIT genetic test results and what a variant can mean

Labs sort each variant they find into 5 groups, from pathogenic and likely pathogenic through uncertain significance to likely benign and benign. Only the first 2 are usually treated as the cause of a condition. A variant of uncertain significance, or VUS, is one without enough evidence either way yet, and labs can reclassify it as research builds.

A genetic counselor or your doctor can explain what a specific result means for you and your family. Our genetic testing guide covers the terms, and MedlinePlus Genetics from the National Library of Medicine has a plain-language page on KIT.

KIT gene questions

What does the KIT gene do?

KIT makes a receptor on the surface of certain cells that switches on when a protein called stem cell factor attaches to it. Its signals control cell growth, division, survival and movement. This summary follows MedlinePlus Genetics from the U.S. National Library of Medicine.

Are there clinical trials for people with KIT gene variants?

As of September 29, 2026, 14 recruiting or soon-opening trials on ClinicalTrials.gov name KIT as a gene. Each one is listed on this page with the words from the trial that mention the gene. ClinicalTrials.gov also lists 21 open trials for Mastocytosis, 13 for Systemic Mastocytosis and 66 for Gastrointestinal Stromal Tumor, and Match Me on Trial Friend can check those against age, sex, location and treatment history.

What conditions are linked to the KIT gene?

Orphanet links KIT to 18 rare conditions and lists variants in the gene as a cause of 16 of them. They include Bullous diffuse cutaneous mastocytosis, Gastrointestinal stromal tumor, Isolated bone marrow mastocytosis, Pseudoxanthomatous diffuse cutaneous mastocytosis, Smoldering systemic mastocytosis, Systemic mastocytosis with associated hematologic neoplasm and 12 more. Orphanet is the European rare disease database run by INSERM in Paris.

Is Mastocytosis inherited?

Most systemic mastocytosis carries an acquired (somatic) activating KIT variant tested in blood or bone marrow; it is not inherited. Additional somatic variants (e.g. TET2, SRSF2, ASXL1, RUNX1) mark more aggressive disease.

Is Systemic Mastocytosis inherited?

Most cases carry a somatic activating KIT variant (usually D816V) in mast cells; additional somatic changes in TET2, SRSF2, ASXL1, RUNX1 or DNMT3A mark more aggressive disease.

What does a KIT variant of uncertain significance mean?

A variant of uncertain significance, or VUS, is a change in the gene that labs don't yet have enough evidence to call disease-causing or harmless. It is not used on its own to diagnose a condition, and labs can reclassify it as research builds. A genetic counselor can explain what a specific result means for you and your family.

Sources

  • Gene and condition links from Orphadata (Orphanet, INSERM US14), used under CC BY 4.0, read September 29, 2026.
  • Trials from ClinicalTrials.gov, read September 29, 2026.
  • KIT gene, MedlinePlus Genetics, U.S. National Library of Medicine.
  • Inheritance and gene notes from the Trial Friend condition pages above, each checked against genetics references.

Other gene pages

The gene search covers every gene Orphanet links to a rare disease.