RecruitingPhase 3NCT07751133
Neuromuscular
Kennedy Disease
Also known as SBMA, spinal and bulbar muscular atrophy, X-linked bulbospinal neuropathy
Kennedy Disease, or Spinal and Bulbar Muscular Atrophy, is an X-linked motor neuron disease caused by a CAG trinucleotide repeat expansion in exon 1 of the androgen receptor (AR) gene, resulting in toxic gain-of-function of the mutant polyg
ORPHA:481 ↗Gene ARPrevalence 1 in 40,000 malesOnset Adult males, typically 3rd–5th decadeX-linked recessive (trinucleotide repeat expansion)
53
studies recruiting now
as of 7 Sept 2026
228
studies registered in total
as of 7 Sept 2026
11
countries with a recruiting site
as of 7 Sept 2026
7 Aug 2026
most recent study posted
among recruiting studies
Recruiting trials
RecruitingPhase 3NCT07723248
A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)
RecruitingNot applicableNCT05647564
PET/CT Characterization of Treatment Resistance
RecruitingPhase 2NCT06854250
Prostate Cryoablation Combined With Metronomic Cyclophosphamide for Metastatic Prostate Cancer
RecruitingPhase 3NCT06855277
Study Comparing AAA817+ARPI Versus Standard of Care in Adult Participants With PSMA-positive mCRPC
Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.
See all 53 recruiting studiesWhere recruiting studies are running
Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.
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About Kennedy Disease
Kennedy Disease, or Spinal and Bulbar Muscular Atrophy, is an X-linked motor neuron disease caused by a CAG trinucleotide repeat expansion in exon 1 of the androgen receptor (AR) gene, resulting in toxic gain-of-function of the mutant polyglutamine-expanded AR protein in motor neurons and muscles. It affects only males (females are carriers) and is characterised by progressive limb and bulbar muscle weakness, androgen insensitivity features, and sensory neuronopathy. Disease progression is slow compared to ALS.
Common clinical features
Slowly progressive proximal limb muscle weaknessBulbar dysfunction (dysarthria, dysphagia, tongue fasciculations)Gynecomastia and reduced fertility (androgen insensitivity)Postural tremor of the handsMuscle cramps and fasciculationsSensory abnormalities (reduced vibration sense)Elevated serum creatine kinase
From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.
Treatments being studied
3 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.
Phase 2/3Mexiletine Hydrochloride (Mexiletine hydrochloride)
Phase 2Clenbuterol (Spiropent)
Phase 2Dutasteride (Avodart)
Before you apply
Things trial teams commonly ask about for Kennedy Disease. Not eligibility rules; those are set by each study.
- Genetic confirmation of CAG repeat length in the AR gene (≥38 repeats) is required; repeat length correlates inversely with age of onset and may be a stratification variable in trials
- Testosterone levels are frequently collected as a pharmacodynamic biomarker; baseline hormonal profile including LH, FSH, and testosterone should be documented
- SBMA Functional Rating Scale (SBMAFRS) and 40-metre walk test are standard clinical endpoints; formal neuromuscular assessment documentation strengthens your application
Related conditions
Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).