ORPHA:227510
Multiple system atrophy, cerebellar type
Also known as: Sporadic OPCA type 1 · Sporadic olivopontocerebellar atrophy type 1 · MSA, cerebellar type · MSA-c
Publications
2,144
89.6th percentile
Trials
4
Interventional, condition-specific
Researchers
1,332
Distinct authors in sample
Gene link
—
Readiness
3/6
Stages with a signal
Clinical definition (Orphanet)
Multiple system atrophy, cerebellar type (MSA-c) is a form of multiple system atrophy (MSA) with predominant cerebellar features (gait and limb , oculomotor dysfunction, and dysarthria).
How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0016418
- UMLS:C5554234
Additional Mondo synonyms (2)
sporadic OPCA type 1 · sporadic olivopontocerebellar atrophy type 1
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.”
3/6 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
2,144 matched papers (1,594 in last 10 years) Source
- Phenotype characterisedPresent
35 HPO annotations (e.g. Neuromuscular dysphagia; Progressive cerebellar ataxia; Broad-based gait) Source
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationNot found
No FDA or EMA orphan-drug designation matched this disease via UMLS or preferred name Source
- Interventional trialPresent
4 matched on ClinicalTrials.gov (2 recruiting in sample)
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)
35
Associated phenotypes · MONDO:0016418
- Neuromuscular dysphagia
- Progressive cerebellar ataxia
- Broad-based gait
- Orofacial dyskinesia
- Frequent falls
Showing 5 of 35 — 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)
No designation matched this disease via UMLS or preferred name on the FDA OOPD mirror or EMA orphan register. Absence here is not proof that none exists under another wording.
Open Targets candidates
No drugs or clinical candidates returned for this Mondo ID on Open Targets.
CTD chemicals (MyDisease.info)
No CTD chemical associations returned for this Mondo ID.
Literature
Is anyone studying this?
2,144
2,144 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.
2,144 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).
1,594 in the last 10 years · high confidence · 89.6th percentile (publications denominator)
Phrase hits: 2,144 · MeSH hits: 0
Who's working on it?
1,332
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.
- 01Lee S7 papers · 2026
Department of Neurology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
Papers in Europe PMC - 02Wang X7 papers · 2026
Department of Neurology, the Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.
Papers in Europe PMC - 03Feng T6 papers · 2026
Department of Neurology, Center for Movement Disorders, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Papers in Europe PMC - 04Kim HJ6 papers · 2026
Department of Neurology, Seoul National University Hospital, Seoul National University College of Medicine, 101 Daehak-Ro, Jongno-Gu, Seoul, 03080, Republic of Korea. movement@snu.ac.kr.
Papers in Europe PMC - 05Liu X6 papers · 2025
Department of Rehabilitation Medicine, The First Affiliated Hospital, Fujian Medical University, Fuzhou, China.
Papers in Europe PMC - 06Watanabe H6 papers · 2026
Department of Neurology, Fujita Health University, School of Medicine, Toyoake, Japan.
Papers in Europe PMC - 07Chen X5 papers · 2026
Baotou Clinical Medical College, Inner Mongolia Medical University, Baotou, Inner Mongolia, China.
Papers in Europe PMC - 08Chung SJ5 papers · 2026
Department of Neurology, Asan Medical Center, University of Ulsan College of Medicine, Seoul, Republic of Korea.
Papers in Europe PMC - 09Ito M5 papers · 2026
Department of Neurology, Fujita Health University School of Medicine, Toyoake, Aichi, Japan.
Papers in Europe PMC - 10Li Y5 papers · 2026
Department of Neurology, Xuanwu Hospital, Capital Medical University, 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; 2 currently recruiting in our sample. 127 trials are registered for multiple system atrophy, 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).
high 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.
- NCT06821256·RECRUITING·Efficacy and Safety of Transcranial dIrect Current stiMulation in Multiple System Atrophy-Cerebellar Variant
Not reviewed·Conditions: Multiple System Atrophy - Cerebellar Subtype (MSA-C)·Matched via name phrase
- NCT07514923·NOT YET RECRUITING·The Study of Safety and Preliminary Efficacy of ALT001 in Patients With MultIple System Atrophy-Cerebellar Type
Not reviewed·Conditions: Multiple System Atrophy - Cerebellar Subtype (MSA-C)·Matched via name phrase
Broader category: multiple system atrophy
127
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
- NCT06831500·RECRUITING·Feasibility Study Assessing the Effect of Carbidopa/Levodopa Ratio on Orthostatic Hypotension in Multiple System Atrophy - Parkinsonian Type and Parkinson Disease.
Not reviewed·Conditions: Multi-system Atrophy - Parkinsonian Type · Orthostatic Hypotension, Dysautonomic · Parkinson Disease·Matched via name phrase
- NCT06891703·RECRUITING·[18F]ACI-15916 PET in α-synucleinopathies
Not reviewed·Conditions: Parkinson's Disease (PD) · Multiple System Atrophy (MSA) · Dementia With Lewy Bodies (DLB)·Matched via name phrase
- NCT03042988·RECRUITING·Overnight Trials With Heat Stress in Autonomic Failure Patients With Supine Hypertension
Not reviewed·Conditions: Hypertension · Pure Autonomic Failure · Multiple System Atrophy · Autonomic Failure·Matched via name phrase
- NCT06597071·ENROLLING BY INVITATION·Parkinson Atypical Rating of Oculometric Patterns Evaluated Routinely
Not reviewed·Conditions: Parkinson Disease · Progressive Supranuclear Palsy(PSP) · Multiple System Atrophy·Matched via name phrase
- NCT07518810·NOT YET RECRUITING·Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy
Not reviewed·Conditions: Multiple System Atrophy·Matched via name phrase
- NCT02897063·RECRUITING·Effects of Midodrine and Droxidopa on Splanchnic Capacitance in Autonomic Failure
Not reviewed·Conditions: Autonomic Failure · Pure Autonomic Failure · Multiple System Atrophy · Parkinson Disease·Matched via name phrase
- NCT07604116·RECRUITING·A Study to Evaluate Satety, Tolerability, Biodistribution, Radiation Dosimetry, and Pharmacokinetics of SST001 in Healthy Volunteers, Patients With PD and Patients With MSA
Not reviewed·Conditions: Multiple System Atrophy (MSA) · Parkinson's Disease (PD)·Matched via name phrase
- NCT06098612·RECRUITING·PET Imaging Evaluation of [11C]SY08
Not reviewed·Conditions: Parkinson's Disease · Multiple System Atrophy · Dementia With Lewy Bodies · Healthy Controls·Matched via name phrase
- NCT07640542·NOT YET RECRUITING·Test-retest Trial With [11C]MODAG-005 in PD or MSA and AMHC - Pilot Phase
Not reviewed·Conditions: Parkinson Disease (PD) · MSA - Multiple System Atrophy · Healthy Adult Participants·Matched via name phrase
- NCT04246437·RECRUITING·[18F]F-DOPA Imaging in Patients With Autonomic Failure
Not reviewed·Conditions: Autonomic Failure · Pure Autonomic Failure · Parkinson Disease · Multiple System Atrophy·Matched via name phrase
- NCT04782284·RECRUITING·Comprehensive Swallowing Rehabilitation in Patients With MSA
Not reviewed·Conditions: Multiple System Atrophy·Matched via name phrase
- NCT07509125·RECRUITING·Ultra-High Resolution PET in Aging, Neurodegeneration and Psychotic Disorders
Not reviewed·Conditions: Alzheimer Dementia (AD) · ALS - Amyotrophic Lateral Sclerosis · Parkinson s Disease · REM Sleep Behavior Disorder (iRBD)·Matched via name phrase
- NCT06162013·RECRUITING·The NADAPT Study: a Randomized Double-blind Trial of NAD Replenishment Therapy for Atypical Parkinsonism
Not reviewed·Conditions: Progressive Supranuclear Palsy · Multiple System Atrophy · Corticobasal Syndrome·Matched via name phrase
- NCT07570212·RECRUITING·Individualized Transcranial Magnetic Stimulation in Parkinsonian Disorders
Not reviewed·Conditions: Parkinson's Disease · Multiple System Atrophy · Progressive Supranuclear Palsy·Matched via name phrase
- NCT07640555·RECRUITING·Evaluation of [¹⁸F]MODAG-009 PET Imaging in Synucleinopathies
Not reviewed·Conditions: Multiple System Atrophy (MSA) · Healthy Adult · Parkinson Disease·Matched via name phrase
Observational and natural-history studies
4 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.
Recruiting or not-yet-recruiting
- NCT06647641·RECRUITING·The CurePSP Genetics Program
Not reviewed·Conditions: PSP · PSP - Progressive Supranuclear Palsy · Corticobasal Syndrome · Corticobasal Syndrome(CBS)·Matched via name phrase
General rare disease registries you may be eligible for
These studies enroll across many rare conditions. They are not counted as evidence that anyone is studying this specific disease.
- NCT01793168·RECRUITING·Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Not reviewed·Conditions: Rare Disorders · Undiagnosed Disorders · Disorders of Unknown Prevalence · Cornelia De Lange Syndrome
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 16 · after dedupe 16 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 16 · dropped 0 · fetched 2026-07-30
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 (16)
- ctis·2025-524317-88-00·Authorised, recruiting·Open-Label Extension Study to Provide Access to ATH434 in Patients with Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2023-506965-72-00·Authorised, recruiting·An open-label, single-center study to evaluate the safety and test-retest characteristics of [11C]MODAG-005 as PET radioligand for imaging pathological alpha-synuclein deposition in the brains of patients with Parkinson’s disease (PD) or Multiple System Atrophy (MSA) compared to age-matched healthy controls (AMHC)
skipped — LLM skipped (--skip-llm)
- ctis·2025-521642-14-00·Authorised, ongoing·An Open-Label Extension, Multi-centered, Phase 2 Trial to Describe the Safety and Efficacy of TEV-56286 (Emrusolmin) in Participants with Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2025-522653-19-00·Authorised·(ASK-PD0-CS002) Long-Term Safety and Efficacy Follow-up of AB-1005 Gene Transfer Study Participants with Parkinson’s Disease or Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2024-517169-18-00·Authorised, recruiting·Interventional, randomized, double-blind, placebo-controlled, optional open-label extension trial of Lu AF82422 in participants with Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2024-515664-31-00·Authorised, ongoing·Phase 1 study to evaluate [18F]ACI-15916 as a potential PET radioligand for imaging α-synuclein deposits in the brain of patients with suspected α-synuclein pathology compared with healthy volunteers
skipped — LLM skipped (--skip-llm)
- ctis·2023-510488-36-01·Authorised, ongoing·PsyPal; Psilocybin Therapy for Psychological Distress in Palliative Patients
skipped — LLM skipped (--skip-llm)
- ctis·2024-515556-20-00·Cancelled·A Phase 1 Open-Label Mass Balance clinical trial to Investigate the Absorption, Metabolism, and Excretion of Single Oral Administration of [14C]-TEV-56286 Following Multiple Oral Administrations of Nonlabeled TEV-56286 to Healthy Male Participants.
skipped — LLM skipped (--skip-llm)
- ctis·2024-511222-30-00·Expired·A Phase 2a, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Dosing of Exidavnemab in Patients with Mild to Moderate Parkinson’s Disease on Stable Symptomatic Parkinson’s Disease Medication and in Patients with Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2023-509876-40-00·Cancelled·A Randomized, Double-blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous TAK-341 in Subjects With Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- ctis·2024-512528-13-00·Authorised, ongoing·Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
skipped — LLM skipped (--skip-llm)
- ctis·2023-505320-54-00·Authorised, ongoing·A Multi-centered, Double-blind, Randomized, Placebo-controlled, Parallel Group Phase 2 Study of TEV-56286 for the Treatment of Patients with Multiple System Atrophy (TOPAS-MSA)
skipped — LLM skipped (--skip-llm)
- ctis·2023-506508-18-00·Authorised, ongoing·Prospective head-to-head comparison of cardiac [18F]-MFBG PET versus [123I]-MIBG SPECT in the differentiation between Parkinson’s disease and multiple system atrophy and between dementia with Lewy bodies and Alzheimer’s disease.
skipped — LLM skipped (--skip-llm)
- ctis·2023-504876-12-00·Cancelled·A Phase 3, Multi-center, Randomized Withdrawal and Long-Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants with Multiple System Atrophy
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN44652664·No longer recruiting·A comparison of two forms of speech therapy (SLT) for people with multiple system atrophy (MSA; a rare condition of the nervous system that causes gradual damage to nerve cells in the brain) type C (that is characterised by cerebellar ataxia [poor muscle control])
skipped — LLM skipped (--skip-llm)
- isrctn·ISRCTN58435532·No longer recruiting·Amyloid imaging in Alzheimer's disease, frontotemporal dementia and healthy volunteers
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 Multiple system atrophy, cerebellar type — 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.
"Multiple system atrophy, cerebellar type" OR "Sporadic OPCA type 1" OR "Sporadic olivopontocerebellar atrophy type 1" OR "MSA, cerebellar type" OR "MSA-c"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
"Multiple system atrophy, cerebellar type" OR "Sporadic OPCA type 1" OR "Sporadic olivopontocerebellar atrophy type 1" OR "MSA, cerebellar type" OR "MSA-c"
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 · 4 observational · 0 expanded access. Only interventional studies enter the trial headline.
Parent-category trials query:
"multiple system atrophy"
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
Ingested 2026-07-27T09:59:17.252Z