Central nervous system disorder

Summary

Central nervous system disorder (MONDO:0002602) is a disease (an umbrella term covering 19 Mondo subtypes) with 3 cohort genes and 164 clinical trials. Top therapeutic interventions include dexmedetomidine, donanemab, and gadobenate dimeglumine.

At a glance

• Umbrella term: 19 Mondo subtypes
• Cohort genes: 3
• ClinVar variants: 3
• Clinical trials: 164

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	central nervous system disorder
Mondo ID	MONDO:0002602
EFO	EFO:0009386
MeSH	D002493
DOID	DOID:331
NCIT	C2934
SNOMED CT	23853001
UMLS	C4021765
MedGen	892343
Anatomy (UBERON)	UBERON:0001017
Is cancer (heuristic)	no

Also known as: central nervous disease · central nervous system disease · central nervous system disease or disorder · central nervous system disorder · CNS disorder · disease of central nervous system · disease of the central nervous system · disease or disorder of central nervous system · disorder of central nervous system

Data availability: 3 ClinVar variants.

Disease family

An umbrella term covering 19 Mondo subtypes.

Classification path: disease › human disease › disease by body system or component › nervous system disorder › central nervous system disorder

Related subtypes (71): congenital nervous system disorder, autoimmune disorder of the nervous system, cranial nerve neuropathy, peripheral nervous system disorder, neuronitis, diplegia of upper limb, retinal disorder, developmental disability, restless legs syndrome, movement disorder, toxic encephalopathy, Barre-Lieou syndrome, Gerstmann syndrome, drug-induced akathisia, drug-induced dyskinesia, stiff-person syndrome, Worster-Drought syndrome, corneal-cerebellar syndrome, pachygyria-intellectual disability-epilepsy syndrome, porencephaly-cerebellar hypoplasia-internal malformations syndrome, symmetrical thalamic calcifications, neonatal brainstem dysfunction, primary orthostatic hypotension, rippling muscle disease with myasthenia gravis, periodic paralysis, qualitative or quantitative protein defects in neuromuscular diseases, specific learning disability, cerebellar hypoplasia-tapetoretinal degeneration syndrome, locked-in syndrome, dopa-responsive dystonia, idiopathic recurrent stupor, chronic lymphocytic inflammation with pontine perivascular enhancement responsive to steroids, spontaneous periodic hypothermia, Sydenham chorea, duplication of the pituitary gland, Balint syndrome, paraneoplastic neurologic syndrome, persistent idiopathic facial pain, serotonin syndrome, hypothalamic adipsic hypernatraemia syndrome, exercise-induced malignant hyperthermia, perineural cyst, neuromuscular disease, neuromyelitis optica, AL amyloidosis, AA amyloidosis, neuroleptic malignant syndrome, infectious disorder of the nervous system, central nervous system malformation, synaptopathy, nervous system neoplasm, sensory ganglionopathy, radiculitis, wet beriberi, perceptual disorders, prepubertal anorexia nervosa, neurocutaneous syndrome, neurovascular disorder, Wallerian degeneration, nervous system injury, neurosarcoidosis, neuroendocrine disorder, tubulinopathy, atactic disorder, hereditary neurological disease, meningitis-retention syndrome, KIF1A related neurological disorder, neurological pain disorder, neurodevelopmental disorder, post 5-alpha-reductase inhibitors treatment syndrome, post-selective serotonin reuptake inhibitor sexual dysfunction

Subtypes (19): autoimmune disorder of central nervous system, autonomic nervous system disorder, optic nerve disorder, spinal cord disorder, high pressure neurological syndrome, central nervous system vasculitis, encephalomyelitis, neurodegenerative disease, brain disorder, central nervous system neoplasm, palsy, trigeminal neuralgia, infantile cerebral and cerebellar atrophy with postnatal progressive microcephaly, sporadic fetal brain disruption sequence, congenital narrowing of cervical spinal canal, central nervous system infectious disorder, cerebrospinal fluid leak, SPAST-related motor disorder, tinnitus

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

ClinVar germline variants

3 retrieved; paginated sample, class counts are floors:

1 conflicting classifications of pathogenicity, 1 likely benign, 1 likely pathogenic

ClinVar	Variant (HGVS)	Gene	Classification	Review
523472	NM_004380.3(CREBBP):c.6185_6195del (p.Ile2062fs)	CREBBP	Likely pathogenic	criteria provided, single submitter
2118762	NM_183357.3(ADCY5):c.2179A>G (p.Arg727Gly)	ADCY5	Conflicting classifications of pathogenicity	criteria provided, conflicting classifications
4072177	NM_020822.3(KCNT1):c.2944-2_2944-1del	KCNT1	Likely benign	criteria provided, single submitter

Genes & proteins

Mendelian disease overlap and somatic drivers

GenCC: 0 · Orphanet: 8 · OMIM-shared: 0 · Dual-evidence (GWAS+Mendelian): 0

Orphanet rare-disease linkage (cohort genes)

Gene	Orphanet ID	Rare disease
KCNT1	Orphanet:293181	Epilepsy of infancy with migrating focal seizures
KCNT1	Orphanet:98784	Sleep-related hypermotor epilepsy
CREBBP	Orphanet:353277	Rubinstein-Taybi syndrome due to CREBBP mutations
CREBBP	Orphanet:353281	Rubinstein-Taybi syndrome due to 16p13.3 microdeletion
CREBBP	Orphanet:370026	Acute myeloid leukemia with t(8;16)(p11;p13) translocation
CREBBP	Orphanet:592574	Menke-Hennekam syndrome
ADCY5	Orphanet:1429	Benign hereditary chorea
ADCY5	Orphanet:324588	Familial dyskinesia and facial myokymia

Cohort genes → proteins

3 cohort genes, 3 distinct canonical proteins.

Evidence partition

Subset	Genes
multi_evidence	3

Cohort genes (full)

Symbol	HGNC	Ensembl	UniProt	Name	Evidence
KCNT1	HGNC:18865	ENSG00000107147	Q5JUK3	Potassium channel subfamily T member 1	clinvar
CREBBP	HGNC:2348	ENSG00000005339	Q92793	CREB-binding protein	clinvar
ADCY5	HGNC:236	ENSG00000173175	O95622	Adenylate cyclase type 5	clinvar

Cohort function summary

Lead sentence per gene, UniProt-curated.

Symbol	Protein name	Function (lead sentence)
KCNT1	Potassium channel subfamily T member 1	Sodium-activated K(+) channel.
CREBBP	CREB-binding protein	Acetylates histones, giving a specific tag for transcriptional activation.
ADCY5	Adenylate cyclase type 5	Catalyzes the formation of the signaling molecule cAMP in response to G-protein signaling.

Protein-family classification

Druggable: 2 · Difficult: 1 · Unknown: 0 · Druggable fraction: 0.67

Family distribution

Cohort families vs a genome-wide background (hypergeometric, BH-FDR; fold = observed/expected). Counts kept; sorted by enrichment, so the catch-all Other/Unknown bucket no longer leads.

Family	Genes	Fold	FDR
Ion channel	1	37.2×	0.080
Enzyme (other)	1	4.0×	0.321
Transcription factor	1	2.8×	0.321

Per-gene assignment

Symbol	Family	Druggable?	EC	InterPro (top 3)
KCNT1	Ion channel	yes		RCK_N, K_chnl_BK_asu, K_chnl_dom
CREBBP	Transcription factor	no	2.3.1.48	Znf_TAZ, Znf_ZZ, Bromodomain
ADCY5	Enzyme (other)	yes	4.6.1.1	A/G_cyclase, Adcy_conserved_dom, A/G_cyclase_CS

Expression context

Cohort genes with no expression data: 0.

3 cohort genes are a single-cell marker in ≥1 SCXA experiment.

Breadth distribution (Bgee present_calls)

Bucket	Genes
narrow (1-5 tissues)	0
moderate (6-20)	0
broad (>20)	3
unknown	0

Top tissues across cohort

Tissue	Cohort genes
cerebellar cortex	1
cerebellar hemisphere	1
right hemisphere of cerebellum	1
amniotic fluid	1
sural nerve	1
tibia	1
apex of heart	1
lower esophagus	1
lower esophagus muscularis layer	1

Per-gene tissue summary (top 30)

Symbol	Bgee breadth	FANTOM5 breadth	SCXA	Top tissues
KCNT1	153	tissue_specific	marker	right hemisphere of cerebellum, cerebellar hemisphere, cerebellar cortex
CREBBP	297	ubiquitous	marker	sural nerve, tibia, amniotic fluid
ADCY5	193	broad	marker	apex of heart, lower esophagus muscularis layer, lower esophagus

Protein interactions among cohort

Intra-cohort edges: 0.

Hub genes (top 10 by interactor count)

Symbol	Interactor count
CREBBP	6,959
ADCY5	1,992
KCNT1	1,562

Structural data

PDB: 3 · AlphaFold-only: 0 · No structure: 0

Cohort genes with PDB structures (top 30)

Symbol	UniProt	PDB entries
CREBBP	Q92793	144
KCNT1	Q5JUK3	6
ADCY5	O95622	2

Function

Pathway analysis

Distinct Reactome pathways touched by cohort: 169. Enrichment computed across 3 evidence-associated genes (2 with Reactome annotation).

Pathways by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 2 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

Pathway	Cohort genes	Fold	FDR	Sample cohort genes
LRR FLII-interacting protein 1 (LRRFIP1) activates type I IFN production	1	1142.0×	0.014	CREBBP
NFE2L2 regulating inflammation associated genes	1	1142.0×	0.014	CREBBP
NFE2L2 regulating ER-stress associated genes	1	1142.0×	0.014	CREBBP
RUNX1 regulates transcription of genes involved in differentiation of myeloid cells	1	713.8×	0.014	CREBBP
NFE2L2 regulates pentose phosphate pathway genes	1	713.8×	0.014	CREBBP
NFE2L2 regulating MDR associated enzymes	1	713.8×	0.014	CREBBP
Regulation of NFE2L2 gene expression	1	713.8×	0.014	CREBBP
Adenylate cyclase activating pathway	1	571.0×	0.014	ADCY5
Regulation of FOXO transcriptional activity by acetylation	1	571.0×	0.014	CREBBP
Regulation of gene expression by Hypoxia-inducible Factor	1	475.8×	0.014	CREBBP
Activation of the TFAP2 (AP-2) family of transcription factors	1	475.8×	0.014	CREBBP
NFE2L2 regulating tumorigenic genes	1	475.8×	0.014	CREBBP
Cellular response to hypoxia	1	439.2×	0.014	CREBBP
Phosphorylation of CLOCK, acetylation of BMAL1 (ARNTL) at target gene promoters	1	439.2×	0.014	CREBBP
RUNX3 regulates NOTCH signaling	1	407.9×	0.014	CREBBP
Adenylate cyclase inhibitory pathway	1	380.7×	0.014	ADCY5
TRAF3-dependent IRF activation pathway	1	380.7×	0.014	CREBBP
R-HSA-1368082	1	356.9×	0.014	CREBBP
Regulation of beta-cell development	1	356.9×	0.014	CREBBP
Regulation of gene expression in late stage (branching morphogenesis) pancreatic bud precursor cells	1	356.9×	0.014	CREBBP
FOXO-mediated transcription of cell death genes	1	356.9×	0.014	CREBBP
Maternal to zygotic transition (MZT)	1	356.9×	0.014	CREBBP
The CRY:PER:kinase complex represses transactivation by the BMAL:CLOCK (ARNTL:CLOCK) complex	1	356.9×	0.014	CREBBP
PKA activation in glucagon signalling	1	335.9×	0.014	ADCY5
Zygotic genome activation (ZGA)	1	335.9×	0.014	CREBBP
PKA activation	1	317.2×	0.014	ADCY5
Transcriptional regulation by the AP-2 (TFAP2) family of transcription factors	1	317.2×	0.014	CREBBP
Activation of GABAB receptors	1	300.5×	0.014	ADCY5
PKA-mediated phosphorylation of CREB	1	285.5×	0.014	ADCY5
NOTCH4 Intracellular Domain Regulates Transcription	1	285.5×	0.014	CREBBP

GO biological processes by enrichment

Over-representation of cohort genes vs the genome-wide background (hypergeometric test, Benjamini-Hochberg FDR; fold = observed/expected over 3 annotated cohort genes). Counts and members are kept as ground-truth; sorted by enrichment.

GO term	Cohort genes	Fold	FDR	Sample cohort genes
N-terminal peptidyl-lysine acetylation	1	1872.4×	0.009	CREBBP
adenylate cyclase-inhibiting dopamine receptor signaling pathway	1	1123.5×	0.009	ADCY5
G protein-coupled adenosine receptor signaling pathway	1	802.5×	0.009	ADCY5
negative regulation of transcription by RNA polymerase I	1	802.5×	0.009	CREBBP
homeostatic process	1	561.7×	0.009	CREBBP
adenylate cyclase-activating dopamine receptor signaling pathway	1	510.7×	0.009	ADCY5
cAMP biosynthetic process	1	468.1×	0.009	ADCY5
protein acetylation	1	468.1×	0.009	CREBBP
cAMP/PKA signal transduction	1	468.1×	0.009	CREBBP
cellular response to forskolin	1	374.5×	0.010	ADCY5
regulation of cellular response to heat	1	351.1×	0.010	CREBBP
regulation of insulin secretion involved in cellular response to glucose stimulus	1	312.1×	0.011	ADCY5
cellular response to glucagon stimulus	1	280.9×	0.011	ADCY5
stimulatory C-type lectin receptor signaling pathway	1	244.2×	0.011	CREBBP
vascular endothelial cell response to laminar fluid shear stress	1	244.2×	0.011	ADCY5
regulation of smoothened signaling pathway	1	208.1×	0.012	CREBBP
positive regulation of transforming growth factor beta receptor signaling pathway	1	175.5×	0.013	CREBBP
renal water homeostasis	1	170.2×	0.013	ADCY5
cellular response to nutrient levels	1	156.0×	0.013	CREBBP
positive regulation of protein localization to nucleus	1	130.6×	0.015	CREBBP
positive regulation of double-strand break repair via homologous recombination	1	127.7×	0.015	CREBBP
canonical NF-kappaB signal transduction	1	122.1×	0.015	CREBBP
neuromuscular process controlling balance	1	110.1×	0.016	ADCY5
embryonic digit morphogenesis	1	100.3×	0.016	CREBBP
cellular response to UV	1	98.5×	0.016	CREBBP
protein destabilization	1	96.8×	0.016	CREBBP
rhythmic process	1	83.8×	0.018	CREBBP
protein homotetramerization	1	79.1×	0.018	KCNT1
locomotory behavior	1	59.8×	0.023	ADCY5
potassium ion transmembrane transport	1	45.3×	0.029	KCNT1

Therapeutics

Drugs indicated for this disease

0 approved, 3 in late-stage (phase 3) trials. Disease-direct ChEMBL indications, not inferred from the associated-gene cohort below.

Drug	Development status
Gadobutrol	Phase 3 (in late-stage trials)
Gadoterate Meglumine	Phase 3 (in late-stage trials)
Gadoteridol	Phase 3 (in late-stage trials)

Earlier-phase candidates (phase 2, investigational — efficacy not yet established): Gadopiclenol.

Drug target analysis

Approved (phase 4): 2 · Phase ≥3: 2 · Phased (≥1): 2 · Undrugged: 1

Druggability breadth: 3 of 3 evidence-associated genes (100%) have a ChEMBL target (buckets above are over the deeply-mined display cohort).

Genes with an approved drug

The molecule shown is one approved compound that hits the gene — not necessarily a drug of choice or one indicated for this disease.

Symbol	Example approved molecule
KCNT1	BEPRIDIL
CREBBP	COLCHICINE

Top cohort targets by molecule count

Symbol	Molecules	Max phase
CREBBP	13	4
KCNT1	2	4
ADCY5	0	0

Drugs targeting cohort genes (top 30)

Molecule	Max phase	Targets in cohort
BEPRIDIL	4	KCNT1
QUINIDINE	4	KCNT1
COLCHICINE	4	CREBBP
ALTRETAMINE	4	CREBBP
CURCUMIN	3	CREBBP
PAPAVERINE	3	CREBBP
EPIGALOCATECHIN GALLATE	3	CREBBP
MOLIBRESIB	2	CREBBP
FISETIN	2	CREBBP
ETAZOLATE	2	CREBBP
LUNRESERTIB	2	CREBBP
TRACAZOLATE	2	CREBBP
NOCODAZOLE	2	CREBBP
INOBRODIB	1	CREBBP
AZD-5153	1	CREBBP

Bioactivity and enzyme data

Enzyme cohort genes (≥1 EC): 2.

Cohort genes with ChEMBL bioactivity (full, sorted by assay count)

Symbol	Assays	Type breakdown
CREBBP	687	Binding:644, Functional:43
ADCY5	43	Binding:33, Functional:9, ADMET:1
KCNT1	24	Binding:24

Cohort enzymes (BRENDA EC)

Symbol	EC numbers	Names
CREBBP	2.3.1.48	histone acetyltransferase
ADCY5	4.6.1.1	adenylate cyclase

Cohort genes with high screening signal

≥100 ChEMBL assays — a studied-ness signal; see Therapeutics for approved-drug status.

Symbol	ChEMBL assays
CREBBP	687

Pharmacogenomics

Cohort genes with a PharmGKB record: 3; with CPIC/DPWG dosing guidelines: 0.

No cohort gene has a CPIC/DPWG genotype-guided dosing guideline (PharmGKB).

Chemical tractability of cohort targets

15 approved/phased compounds have measured bioactivity against a cohort gene (and aren’t yet in disease-level trials). This is a research / tractability signal, NOT a therapeutic recommendation — a bioactivity row often reflects off-target or screening binding (e.g. promiscuous kinase inhibitors against a cohort kinase), implying no disease mechanism.

Compound	Max phase	Cohort target (bioactivity)
BEPRIDIL	4	KCNT1
QUINIDINE	4	KCNT1
COLCHICINE	4	CREBBP
ALTRETAMINE	4	CREBBP
CURCUMIN	3	CREBBP
PAPAVERINE	3	CREBBP
EPIGALOCATECHIN GALLATE	3	CREBBP
MOLIBRESIB	2	CREBBP
FISETIN	2	CREBBP
ETAZOLATE	2	CREBBP
LUNRESERTIB	2	CREBBP
TRACAZOLATE	2	CREBBP
NOCODAZOLE	2	CREBBP
INOBRODIB	1	CREBBP
AZD-5153	1	CREBBP

Druggability pyramid

Cohort genes binned by druggability tier (high → low):

Tier	Definition	Genes	Symbols
A	Approved (phase 4 drug)	2	KCNT1, CREBBP
B	Phased (≥1) drug, not yet approved	0
C	Druggable family + PDB, no drug	1	ADCY5
D	Druggable family + AlphaFold only, no drug	0
E	Difficult family or no structure, no drug	0

Undrugged target profiles

1 cohort genes are undrugged. Ranked by ‘starting-point quality’ (assay depth + drugged-partner adjacency).

Symbol	ChEMBL assays	Drugged partners (top 3)
ADCY5	43	—

Clinical trials & evidence

Clinical trials

Clinical trials: 164.

Phase distribution (across all retrieved trials)

Phase	Trials
Not specified	113
PHASE2	19
PHASE1	11
PHASE3	9
PHASE4	8
PHASE1/PHASE2	2
PHASE2/PHASE3	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01340950	PHASE4	COMPLETED	Clinical Trial of Brain-Penetrating HIV Drugs to Prevent Cognitive Impairment in China
NCT01445639	PHASE4	COMPLETED	Dexmedetomidine in Patients After Intracranial Surgery
NCT01662414	PHASE4	COMPLETED	Effect of Undenatured Cysteine-Rich Whey Protein Isolate (HMS 90®) in Patients With Parkinson’s Disease
NCT04399343	PHASE4	UNKNOWN	Dexmedetomidine for Prevention of Postoperative Delirium After Intracranial Operation for Brain Tumor
NCT04494828	PHASE4	COMPLETED	Impact Dexmedetomidine on Postoperative Delirium in Patients After Intracranial Operation for Brain Tumor
NCT04871464	PHASE4	UNKNOWN	Role and Mechanism of Probiotics in Improving Motor Symptoms in Mild to Moderate Parkinson’s Disease
NCT04898270	PHASE4	COMPLETED	Adjunctive Use of Fute (Flupentixol) in Multi-acting Receptor-targeted Antipsychotics Treated Schizophrenia Patients
NCT05068349	PHASE4	UNKNOWN	For Patients With Ischemic Stroke, Clinically Study the Effectiveness and Safety of Butylphthalide.
NCT05508789	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Donanemab (LY3002813) in Participants With Early Symptomatic Alzheimer’s Disease (TRAILBLAZER-ALZ 5)
NCT05738486	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Different Donanemab (LY3002813) Dosing Regimens in Adults With Early Alzheimer’s Disease (TRAILBLAZER-ALZ 6)
NCT05892510	PHASE2/PHASE3	RECRUITING	Post-thrombectomy Intra-arterial Tenecteplase for Acute manaGement of Non-retrievable Thrombus and No-reflow in Emergent Stroke
NCT00323310	PHASE3	TERMINATED	Safety and Efficacy of MultiHance in Pediatric Patients
NCT00395460	PHASE3	COMPLETED	Efficacy and Safety Study to Evaluate Gadavist (Gadobutrol) as Contrast Agent in Magnetic Resonance Imaging (MRI) of Brain or Spine Diseases in Chinese Patients
NCT00623467	PHASE3	COMPLETED	Safety and Efficacy of Gadobutrol 1.0 Molar ( Gadavist ) in Patients for Central Nervous System (CNS) Imaging
NCT00709852	PHASE3	COMPLETED	Safety and Efficacy of Gadobutrol 1.0 Molar (Gadavist) in Patients for Central Nervous System (CNS) Imaging
NCT01211873	PHASE3	COMPLETED	Safety and Efficacy Evaluation of DOTAREM® in MRI of Central Nervous System (CNS) Lesions
NCT04639310	PHASE3	TERMINATED	XEN496 (Ezogabine) in Children With KCNQ2 Developmental and Epileptic Encephalopathy
NCT04912856	PHASE3	TERMINATED	An Open-Label Extension of the Study XEN496 (Ezogabine) in Children With KCNQ2-DEE
NCT04460872	PHASE2	RECRUITING	Locomotor Training With Testosterone to Promote Bone and Muscle Health After Spinal Cord Injury
NCT05386108	PHASE1/PHASE2	RECRUITING	Study of Abemaciclib and Elacestrant in Participants With Brain Metastasis Due to ER+/HER-2- Breast Cancer
NCT07326566	PHASE2	RECRUITING	Study of Silevertinib With Temozolomide for the Treatment of Newly Diagnosed GBM With Unmethylated MGMT and EGFRvIII
NCT00406029	PHASE2	COMPLETED	Dyskinesia in Parkinson’s Disease (Study P04501)
NCT00537017	PHASE2	COMPLETED	Follow Up Safety Study of SCH 420814 in Subjects With Parkinson’s Disease (P05175)
NCT00968851	PHASE2	COMPLETED	Safety and Cognitive Function Study of EVP-6124 in Patients With Schizophrenia
NCT01073228	PHASE2	COMPLETED	Safety and Cognitive Function Study of EVP-6124 in Patients With Mild to Moderate Alzheimer’s Disease
NCT02248701	PHASE2	TERMINATED	Testosterone Plus Finasteride Treatment After Spinal Cord Injury
NCT03008486	PHASE2	COMPLETED	Long Term Effects of Soft Splints on Stroke Patients and Patients With Disorders of Consciousness
NCT03127514	PHASE2	COMPLETED	AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)
NCT03448159	PHASE2	COMPLETED	Fluoxetine Opens Window to Improve Motor Recovery After Stroke
NCT03926351	PHASE2	UNKNOWN	High Dose Omega 3 in People at Risk for Dementia
NCT04445831	PHASE1/PHASE2	COMPLETED	A Study to Evaluate the Safety, Tolerability and Immunogenicity of Tau Targeted Vaccines in Participants With Early Alzheimer’s Disease
NCT04640077	PHASE2	COMPLETED	A Follow-On Study of Donanemab (LY3002813) With Video Assessments in Participants With Alzheimer’s Disease (TRAILBLAZER-EXT)
NCT04912115	PHASE2	SUSPENDED	Randomized, Double-Blind, Active Placebo-Controlled Study of Ketamine to Treat Levodopa-Induced Dyskinesia
NCT04937452	PHASE2	COMPLETED	Dopaminergic Therapy for Frontotemporal Dementia Patients
NCT05318976	PHASE2	COMPLETED	A Study of XPro1595 in Patients With Early Alzheimer’s Disease With Biomarkers of Inflammation
NCT05321498	PHASE2	WITHDRAWN	Study to Assess the Efficacy of XPro1595 in Patients With Mild Cognitive Impairment With Biomarkers of Inflammation
NCT05375240	PHASE2	UNKNOWN	Propranolol on Post Stroke Immune Status and Infection
NCT05522387	PHASE2	TERMINATED	An Open-Label Extension of XPro1595 in Patients With Alzheimer’s Disease
NCT05590884	PHASE2	COMPLETED	Gadopiclenol Pharmacokinetics, Safety and Efficacy in Children < 2 Years of Age
NCT03911388	PHASE1	RECRUITING	HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
DEXMEDETOMIDINE	4	3
DONANEMAB	4	3
GADOBENATE DIMEGLUMINE	4	3
GADOBUTROL	4	3
RIVASTIGMINE	4	3
EZOGABINE	4	2
GADOTERIDOL	4	2
LEVODOPA	4	2
SODIUM CHLORIDE	4	2
TESTOSTERONE ENANTHATE	4	2
CEFTRIAXONE	4	1
ELACESTRANT	4	1
FINASTERIDE	4	1
GADOPENTETATE DIMEGLUMINE	4	1
GADOPICLENOL	4	1
OLIVE OIL	4	1
ROTIGOTINE	4	1
ENCENICLINE	3	8
PRELADENANT	3	2
RAC-3-N-BUTYLPHTHALIDE	3	2
FLUPENTIXOL	3	1
MEDETOMIDINE	3	1
OMEGA-3 FATTY ACIDS	3	1
VELIPARIB	3	1
PEGIPANERMIN	2	3
LUFENURON	2	1
TASADENOTUREV	2	1
CHEMBL249818	0	2
CHEMBL4777013	0	1
CHEMBL104383	0	1