ORPHA:3260
Idiopathic hypereosinophilic syndrome
Publications
1,561
Trials
4
Interventional, condition-specific
Researchers
1,068
Distinct authors in sample
Gene link
—
Readiness
4/6
Stages with a signal
Clinical definition (Orphanet)
A rare hematologic disease characterized by eosinophilia without evidence of clonality persisting for at least six months, for which no underlying cause can be identified. The condition is associated with signs of organ damage and dysfunction. Clinical manifestations are highly variable, depending on the organ systems involved, and include rapidly developing, life-threatening cardiovascular or neurological complications.
How rare: How common this is has not been clearly measured.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0011895
- OMIM:607685
- UMLS:C0206141
Additional Mondo synonyms (1)
hypereosinophilic syndrome, idiopathic, resistant to imatinib, isolated cases, somatic mutation
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
4/6 stages with a signal
An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
1,561 matched papers (586 in last 10 years) Source
- Phenotype characterisedPresent
100 HPO annotations (e.g. Chest pain; Abnormal eosinophil morphology; Abdominal pain) Source
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationPresent
2 FDA designations (1 FDA orphan-indication approval) — e.g. Dexpramipexole Source
- Interventional trialPresent
4 matched on ClinicalTrials.gov
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
100
Associated phenotypes · MONDO:0011895
- Chest pain
- Abnormal eosinophil morphology
- Abdominal pain
- Abnormal pleura morphology
- Abnormal pattern of respiration
Showing 5 of 100 — open Monarch for the full list.
Animal models (Monarch / Alliance)
None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.
Monarch fetch 2026-07-29
Therapies
Designations, candidates, and chemicals
FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.
Orphan designation (FDA · EMA)
2
Designations · 1 with FDA orphan-indication approval
- FDA DexpramipexoleHypereosinophilic syndrome Hypereosinophilic syndrome · 2019-04-22 · Not FDA Approved for Orphan Indication
- FDA Imatinib mesylate (Gleevec)Idiopathic Hypereosinophilic Syndrome Chronic eosinophilic leukemia · 2005-08-25
Sources: FDA OOPD · EMA orphan designations
Open Targets candidates
4
Drugs / clinical candidates · MONDO_0011895
- CLADRIBINE·phase 2
- CYTARABINE·phase 2
- IMATINIB·phase 2
- IMATINIB MESYLATE·phase 2
CTD chemicals (MyDisease.info)
No CTD chemical associations returned for this Mondo ID.
Literature
Is anyone studying this?
1,561
1,561 papers have been published on this condition. That is a real research literature — still far smaller than common diseases (breast cancer: over 700,000 papers). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
1,561 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).
586 in the last 10 years · low confidence
Phrase hits: 1,561 · MeSH hits: 0
Who's working on it?
1,068
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01Takahashi H5 papers · 2025
Division of Respiratory Medicine, Department of Medicine Showa General Hospital Tokyo Japan.
Papers in Europe PMC - 02Zhang J4 papers · 2026
Queen's Heart Institute, The Queen's Medical Center, Honolulu, Hawaii, USA.
Papers in Europe PMC - 03Li J3 papers · 2025
Department of Rheumatology and Immunology, Peking University International Hospital, Beijing, China.
Papers in Europe PMC - 04Lübke J3 papers · 2026
Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University , ,
Papers in Europe PMC - 05Reiter A3 papers · 2026
Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University , ,
Papers in Europe PMC - 06Schwaab J3 papers · 2026
Department of Hematology and Oncology, University Hospital Mannheim, Heidelberg University , ,
Papers in Europe PMC - 07Singh S3 papers · 2026
Internal Medicine, Advocate Christ Medical Center, Oak Lawn, USA.
Papers in Europe PMC - 08Wang X3 papers · 2025
Department of Pathology, The 6th People's Hospital of Chengdu, Chengdu 610051, China.
Papers in Europe PMC - 09Zhang L3 papers · 2026
Department of Rheumatology and Immunology, Peking University International Hospital, Beijing, China.
Papers in Europe PMC - 10Zhang Y3 papers · 2026
Department of Rheumatology and Immunology, Peking University International Hospital, Beijing, China.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
4
interventional trials for this specific condition
4 interventional trials matched this specific condition name; none in our sample are currently recruiting. 24 trials are registered for hypereosinophilic syndrome, the broader category — shown separately because they may or may not enrol this specific subtype.
Data as of 11 September 2026
4 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 88.1th percentile).
low confidence · 88.1th percentile (trials denominator)
Recruiting interventional trials
From the matched ClinicalTrials.gov set
4 interventional trials matched after quoted-phrase search and title/condition post-filter.
No currently recruiting studies in the matched set. Open the same search on ClinicalTrials.gov.
Broader category: hypereosinophilic syndrome
24
Interventional trials for the parent category, exclusive of NCT IDs already counted above. Eligibility for this subtype is not guaranteed.
Worth raising with a clinician. How we count trials.
Recruiting under the broader category
- NCT05334368·RECRUITING·Depemokimab in Participants With Hypereosinophilic Syndrome, Efficacy, and Safety Trial
Not reviewed·Conditions: Hypereosinophilic Syndrome·Matched via name phrase
- NCT07275190·RECRUITING·The Use of Machine Learning Techniques for the Differential Diagnosis Between Eosinophilic Granulomatosis With Polyangiitis and Hypereosinophilic Syndrome
Not reviewed·Conditions: EGPA - Eosinophilic Granulomatosis With Polyangiitis · HES - Hypereosinophilic Syndrome·Matched via name phrase
- NCT07444567·NOT YET RECRUITING·Roll-over Study for Participants Who Have Completed a Previous Clinical Study With Benralizumab (Fasenra) and Benefit From Continued Treatment
Not reviewed·Conditions: Asthma · Eosinophilic Granulomatosis With Polyangiitis (EGPA) · Hypereosinophilic Syndrome (HES)·Matched via name phrase
- NCT06477653·RECRUITING·Dupilumab as Add-On Therapy for Hypereosinophilic Syndrome With Partial Clinical Response to Eosinophil-Depleting Biologic Agents
Not reviewed·Conditions: Hypereosinophilic Syndrome·Matched via name phrase
- NCT03801434·RECRUITING·Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders
Not reviewed·Conditions: BCR-JAK2 Fusion Protein Expression · Blasts 20 Percent or Less of Peripheral Blood White Cells · Blasts More Than 5 Percent of Bone Marrow Nucleated Cells · Blasts More Than 5 Percent of Peripheral Blood White Cells·Matched via name phrase
Other registries (secondary)
Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.
raw 60 · after dedupe 60 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 60 · dropped 0 · fetched 2026-07-29
Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri
No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).
Uncertain / not reviewed (60)
- ctis·2024-519123-14-00·Authorised·A Randomized, Placebo-controlled, Double-blind, Dose-finding Phase 1b Study to Assess the Safety, Immunogenicity and Pharmacodynamics of TRB-001 in Early Idiopathic Parkinson’s Disease Patients
skipped — LLM skipped (--skip-llm)
- ctis·2023-508533-14-00·Authorised·Phase III, Open-label Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, Efficacy, and Immunogenicity of Benralizumab in Children with Eosinophilic Disease (CLIPS)
skipped — LLM skipped (--skip-llm)
- ctis·2025-523832-38-00·Authorised·Multicenter Interventional Study Somatrogon Impact on Outcomes in Naive Small for Gestational Age or Idiopathic Short Stature paediatric patients compared with daily growth hormone
skipped — LLM skipped (--skip-llm)
- ctis·2026-525611-13-00·Authorised·Treatment strategies for juvenile idiopathic arthritis patients with sustained inactive disease: A phase 4, multicentre, randomised trial comparing maintenance versus tapered TNF alpha inhibitor monotherapy - the Treat-JIA trial
skipped — LLM skipped (--skip-llm)
- ctis·2025-522373-10-00·Authorised·IM0271016: An Open-label, Multi-center, Long-term Extension Study to Evaluate the Long-term Safety and Tolerability of Admilparant in Participants with Pulmonary Fibrosis
skipped — LLM skipped (--skip-llm)
- ctis·2025-521145-24-01·Authorised·A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active Juvenile Idiopathic Inflammatory Myopathy
skipped — LLM skipped (--skip-llm)
- ctis·2026-526135-18-00·Authorised, ongoing·A randomised, open-label, single-centre, drug-drug-interaction trial to evaluate the impact of carboxylesterase 2 (CES2)- and CYP3A4-inhibition on the pharmacokinetics of buloxibutid in healthy volunteers
skipped — LLM skipped (--skip-llm)
- ctis·2024-514246-37-00·Authorised·A Phase IIb, multicentre, randomised, doubleblind, placebocontrolled, three-arm parallel-group study to evaluate the efficacy, safety, and tolerability at Week 24 of 2 doses of CHF10067 (zampilimab), with an optional 24-week doubleblind, placebocontrolled extension phase in participants with idiopathic pulmonary fibrosis
skipped — LLM skipped (--skip-llm)
- ctis·2025-524100-29-00·Authorised·An Open-Label, Phase 1b, Multiple Ascending Dose Study of OM336 in Participants with Active Sjogren’s Disease or Idiopathic Inflammatory Myopathy
skipped — LLM skipped (--skip-llm)
- ctis·2025-524822-16-00·Authorised, ongoing·Prevention of chronic headache with anti-CGRP antibody treatment in patients with idiopathic intracranial hypertension
skipped — LLM skipped (--skip-llm)
- ctis·2024-511593-70-00·Authorised, recruiting·Multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of filgotinib in children and adolescents from 8 years to less than 18 years of age with polyarticular-course juvenile idiopathic arthritis
skipped — LLM skipped (--skip-llm)
- ctis·2025-523239-21-00·Authorised, ongoing·Open Label Extension (OLE), multiple dose study to evaluate pharmacokinetics, safety, tolerability and efficacy of filgotinib in children and adolescents from 8 years to less than 18 years of age with juvenile idiopathic arthritis (JIA).
skipped — LLM skipped (--skip-llm)
- ctis·2025-523431-19-00·Authorised, ongoing·A Randomized, Double-Blind, Placebo-Controlled, Phase 2 Proof of Concept (POC) Study Evaluating the Safety, Tolerability, and Efficacy of Nintedanib Solution for Inhalation (AP02) in Participants with Idiopathic Pulmonary Fibrosis (IPF) (AURA-IPF)
skipped — LLM skipped (--skip-llm)
- ctis·2025-524883-39-00·Authorised·A Phase 1, open label, randomised, 2-period, 2 sequence, 7-days repeat-dosing, crossover oral pharmacokinetic trial comparing multiple dosing of CS014 to valproic acid in healthy adults.
skipped — LLM skipped (--skip-llm)
- ctis·2024-518013-25-00·Authorised, recruiting·A Phase 2a Multicenter Platform Study of Investigational Products for the Treatment of Adult Subjects with Idiopathic Pulmonary Fibrosis
skipped — LLM skipped (--skip-llm)
- ctis·2025-522834-30-01·Authorised·Fibroblast markers to tackle fibrosis in immune-mediated inflammatory diseases
skipped — LLM skipped (--skip-llm)
- ctis·2025-523079-44-00·Authorised, ongoing·HighLiGHts - A Pivotal, Parallel-Arm, Phase 3, Open-Label, Active-controlled, Global, Multicenter, Randomized Basket Trial Investigating the Efficacy and Safety of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Children and Adolescents with Growth Failure or Short Stature due to Growth Hormone Sufficient Disorders – Turner Syndrome, SHOX Deficiency, Small for Gestational Age, and Idiopathic Short Stature
skipped — LLM skipped (--skip-llm)
- ctis·2025-523306-33-00·Authorised, ongoing·Obinutuzumab Treatment in Frequently Relapsing and Rituximab-Dependent Idiopathic Nephrotic Syndrome in Adults: a fully academic, single-arm, open, prospective, intervention trial
skipped — LLM skipped (--skip-llm)
- ctis·2025-522987-34-00·Authorised, ongoing·A Phase 2, Long-term Extension Study of the Safety and Efficacy of ORX750 in Participants with Narcolepsy and Idiopathic Hypersomnia Who Completed a Sponsored ORX750 Clinical Trial
skipped — LLM skipped (--skip-llm)
- ctis·2025-520923-25-00·Authorised·Trial of Sequential Medications AfteR TNFi Failure in Juvenile Idiopathic Arthritis (SMART-JIA)
skipped — LLM skipped (--skip-llm)
- ctis·2025-522857-20-00·Authorised, ongoing·An open label, phase I/II study investigating the safety and efficacy of the bispecific T-cell engaging antibody cizutamig (BCMAxCD3) in patients with immune-mediated inflammatory diseases – the SPLENDID Trial
skipped — LLM skipped (--skip-llm)
- ctis·2025-521530-28-00·Authorised, ongoing·A phase I/II study of CAR-T cells in AutoiMmune disease resistant to B cell Abrogation - CARAMBA
skipped — LLM skipped (--skip-llm)
- ctis·2024-513710-35-00·Authorised·SERORL Effect of aspirin and folic acid for sudden sensorineural hearing loss
skipped — LLM skipped (--skip-llm)
- ctis·2025-520658-12-00·Authorised, ongoing·A double-blind, randomised, placebo-controlled, parallel group, Phase IIa trial to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of BI 765423 administered intravenously with or without standard of care in patients with idiopathic pulmonary fibrosis
skipped — LLM skipped (--skip-llm)
- ctis·2025-520715-13-00·Authorised, ongoing·A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability and Efficacy of Caveolin-1-Scaffolding-Protein-Derived Peptide (LTI-03) in Patients with Idiopathic Pulmonary Fibrosis (RENEW)
skipped — LLM skipped (--skip-llm)
Where to find support
Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.
Orphanet entry for Idiopathic hypereosinophilic syndrome — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Idiopathic hypereosinophilic syndrome" OR "hypereosinophilic syndrome, idiopathic, resistant to imatinib, isolated cases, somatic mutation"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Idiopathic hypereosinophilic syndrome" OR "hypereosinophilic syndrome, idiopathic, resistant to imatinib, isolated cases, somatic mutation"
Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).
Study-type breakdown: 4 interventional · 0 observational · 0 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"hypereosinophilic syndrome"
Query health: ok — strategies attempted: phrase; with hits: phrase
Run this search on ClinicalTrials.gov
Confidence reasoning
- Preferred label is multi-word and distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus
- Publication count (1561) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity
Ingested 2026-07-26T22:39:43.222Z