Indolent systemic mastocytosis

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ORPHA:98848D47.0
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1FDA treatments5Active trials38Specialists8Treatment centers1Financial resources

Where are you in your journey?

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

Clinical phenotype terms— hover any for plain English:

Abnormal bone marrow cell morphologyHP:0005561Abnormal skin morphologyHP:0011121Increased proportion of CD25+ mast cellsHP:0031408Abnormal mast cell morphologyHP:0100494MastocytosisHP:0100495AllergyHP:0012393Darier's signHP:0025081FlushingHP:0031284Elevated total serum tryptaseHP:0031901Abdominal crampsHP:0032155Maculopapular exanthemaHP:0040186Anaphylactic shockHP:0100845
Orphanet ↗NORD ↗

FDA & Trial Timeline

6 events
Feb 2026An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)

Blueprint Medicines Corporation

TrialNOT YET RECRUITING
Dec 2025A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany

Blueprint Medicines Corporation

TrialRECRUITING
Jun 2022(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

Cogent Biosciences, Inc. — PHASE2

TrialACTIVE NOT RECRUITING
Nov 2021(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Blueprint Medicines Corporation — PHASE2, PHASE3

TrialRECRUITING
Oct 2020Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis

Telios Pharma, Inc. — PHASE2

TrialRECRUITING
Apr 2019(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

Blueprint Medicines Corporation — PHASE2

TrialACTIVE NOT RECRUITING

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

Treatments

1 available

Ayvakit

avapritinib· Blueprint Medicines Corporation

the treatment of adult patients with ISM

View Details →FDA Label ↗Copay Card ↗Patient Assistance ↗

Clinical Trials

5 recruitingView all trials with filters →
Phase 23 trials
(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
Phase 2
Active
· Sites: Birmingham, Alabama; Phoenix, Arizona +47 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
Phase 2
Active
PI: Rachael Easton, MD, PhD (Cogent Biosciences) · Sites: Birmingham, Alabama; Phoenix, Arizona +55 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis
Phase 2
Actively Recruiting
· Sites: Aurora, Colorado; Rochester, Minnesota +50 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Other1 trial
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
Actively Recruiting
· Sites: Berlin; Dresden +2 more · Age: 18–99 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 38View all specialists →
FS
Frank Siebenhaar
Specialist
7 Indolent systemic mastocytosis publications
SB
Sigurd Broesby-Olsen
Specialist
4 Indolent systemic mastocytosis publications
TG
Tracy I George
Specialist
5 Indolent systemic mastocytosis publications
KH
Karin Hartmann
DENVER, CO
Specialist
5 Indolent systemic mastocytosis publications
MC
Mariana Castells
Specialist
5 Indolent systemic mastocytosis publications
SC
Sonia Cerquozzi
ROCHESTER, MN
Specialist
3 Indolent systemic mastocytosis publications
CA
Cem Akin
NORTHVILLE, MI
Specialist
8 Indolent systemic mastocytosis publications
CY
Cecilia Arana Yi
Specialist
3 Indolent systemic mastocytosis publications
AR
Andreas Reiter
Specialist
3 Indolent systemic mastocytosis publications
ID
Ingunn Dybedal
Specialist
3 Indolent systemic mastocytosis publications
PV
Peter Valent
Specialist
4 Indolent systemic mastocytosis publications
VS
Vito Sabato
Specialist
4 Indolent systemic mastocytosis publications
MM
Marcus Maurer, MD
PUEBLO, CO
Specialist
PI on 5 active trials
FM
Frank Siebenhaar, MD
Specialist
PI on 2 active trials
OP
Olivier Lortholary, MD, PhD
Specialist
PI on 5 active trials
RP
Rachael Easton, MD, PhD
TROY, MI
Specialist
PI on 3 active trials
HM
Hirsh D Komarow, M.D.
BETHESDA, MD
Specialist
PI on 3 active trials
CM
Cristina Bulai Livideanu, MD
Specialist
PI on 3 active trials
AP
Anthony L Boral, MD, PhD
CAMBRIDGE, MA
Specialist
PI on 1 active trial
BM
Brenton Mar
BOSTON, MA
Specialist
3 Indolent systemic mastocytosis publications
HL
Hui-Min Lin
Specialist
3 Indolent systemic mastocytosis publications
IB
Ilda Bidollari
Specialist
4 Indolent systemic mastocytosis publications
IA
Iván Alvarez-Twose
Specialist
2 Indolent systemic mastocytosis publications
AS
Alan L Shields
MA
Specialist
3 Indolent systemic mastocytosis publications
TG
Tanya Green
MA
Specialist
3 Indolent systemic mastocytosis publications

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
Ayvakit(avapritinib)— Blueprint Medicines Corporation

Travel Grants

No travel grants are currently matched to Indolent systemic mastocytosis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Indolent systemic mastocytosis

Disease timeline:

New recruiting trial: Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis

A new clinical trial is recruiting patients for Indolent systemic mastocytosis

New recruiting trial: A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany

A new clinical trial is recruiting patients for Indolent systemic mastocytosis

New recruiting trial: (HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

A new clinical trial is recruiting patients for Indolent systemic mastocytosis

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.

Common questions about Indolent systemic mastocytosis

Are there clinical trials for Indolent systemic mastocytosis?

Yes — 5 recruiting clinical trials are currently listed for Indolent systemic mastocytosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Indolent systemic mastocytosis?

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