Congenital nervous system disorder
MONDO:0002320An abnormality of the nervous system that is present at birth or detected in the neonatal period.
Also known as: congenital abnormality of the nervous system, congenital nervous system disorder
287 clinical trials for this condition and its sub-types, 1 tagged with Congenital nervous system disorder itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Congenital nervous system disorder
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Spina bifida 44 trials · 59 incl. sub-types
2 sub-types
- Isolated spina bifida 1 trial · 28 incl. sub-types Sub-types →
- Spina bifida occulta 2 trials Sub-types →
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Combined pituitary hormone deficiencies, genetic form 1 trial · 42 incl. sub-types
9 sub-types
- Isolated congenital growth hormone deficiency 38 trials Sub-types →
- Panhypopituitarism 2 trials Sub-types →
- Congenital isolated adrenocorticotropic hormone deficiency 1 trial
- Pituitary hormone deficiency, combined, 1 1 trial
- Septooptic dysplasia 1 trial Sub-types →
- Non-acquired combined pituitary hormone deficiency with spine abnormalities 0 trials
- Pituitary hormone deficiency, combined or isolated, 8 0 trials
- Pituitary hormone deficiency, combined, 6 0 trials
- Short stature-pituitary and cerebellar defects-small sella turcica syndrome 0 trials
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Prader-Willi syndrome 32 trials
5 sub-types
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Leber congenital amaurosis 11 trials · 13 incl. sub-types
21 sub-types
- Leber congenital amaurosis 2 3 trials
- Leber congenital amaurosis 10 2 trials
- Leber congenital amaurosis 5 2 trials
- Leber congenital amaurosis 1 1 trial
- Leber congenital amaurosis 11 0 trials
- Leber congenital amaurosis 12 0 trials
- Leber congenital amaurosis 13 0 trials
- Leber congenital amaurosis 14 0 trials
- Leber congenital amaurosis 15 0 trials
- Leber congenital amaurosis 16 0 trials
- Leber congenital amaurosis 17 0 trials
- Leber congenital amaurosis 18 0 trials
- Leber congenital amaurosis 19 0 trials
- Leber congenital amaurosis 3 0 trials
- Leber congenital amaurosis 4 0 trials
- Leber congenital amaurosis 6 0 trials
- Leber congenital amaurosis 7 0 trials
- Leber congenital amaurosis 8 0 trials
- Leber congenital amaurosis 9 0 trials
- Leber congenital amaurosis with early-onset deafness 0 trials
- Retinal aplasia 0 trials
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22q11.2 deletion syndrome 7 trials · 13 incl. sub-types
4 sub-types
- DiGeorge syndrome 11 trials
- Velocardiofacial syndrome 4 trials
- Chromosome 22q11.2 deletion syndrome, distal 0 trials
- Congenital unilateral hypoplasia of depressor anguli oris 0 trials
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Congenital muscular dystrophy 1 trial · 10 incl. sub-types
23 sub-types
- Congenital merosin-deficient muscular dystrophy 1A 3 trials
- Congenital myasthenic syndrome 10 3 trials
- Congenital muscular dystrophy due to LMNA mutation 2 trials
- Muscular dystrophy-dystroglycanopathy 0 trials · 1 incl. sub-types Sub-types →
- Rigid spine syndrome 0 trials · 1 incl. sub-types Sub-types →
- Bethlem myopathy 0 trials Sub-types →
- SNUPN-related muscular dystrophy with or without multi-system involvement 0 trials Sub-types →
- Ullrich congenital muscular dystrophy 0 trials Sub-types →
- Arthrogryposis due to muscular dystrophy 0 trials
- Autosomal recessive myogenic arthrogryposis multiplex congenita 0 trials
- Collagen 6-related congenital muscular dystrophy 0 trials Sub-types →
- Congenital muscular dystrophy 1B 0 trials
- Congenital muscular dystrophy caused by variation in POMGNT2 0 trials Sub-types →
- Congenital muscular dystrophy due to integrin alpha-7 deficiency 0 trials
- Congenital muscular dystrophy with cataracts and intellectual disability 0 trials
- Congenital muscular dystrophy with hyperlaxity 0 trials
- Congenital muscular dystrophy without intellectual disability 0 trials
- Congenital muscular dystrophy-infantile cataract-hypogonadism syndrome 0 trials
- Congenital muscular dystrophy-respiratory failure-skin abnormalities-joint hyperlaxity syndrome 0 trials
- Congenital myopathy, Paradas type 0 trials
- Megaconial type congenital muscular dystrophy 0 trials
- Muscle-eye-brain disease 0 trials Sub-types →
- Muscular dystrophy, congenital, with rapid progression 0 trials
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Chediak-Higashi syndrome 9 trials
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Congenital laryngeal palsy 9 trials
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Congenital myotonic dystrophy 9 trials
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Congenital vitreoretinal dysplasia 0 trials · 9 incl. sub-types
9 sub-types
- Trisomy 13 4 trials Sub-types →
- Incontinentia pigmenti 3 trials
- Coats disease 2 trials
- Coats plus syndrome 0 trials Sub-types →
- Norrie disease 0 trials
- Osteoporosis-pseudoglioma syndrome 0 trials
- Persistent hyperplastic primary vitreous 0 trials Sub-types →
- Retinal capillary malformation 0 trials
- Spondylo-ocular syndrome 0 trials
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Progressive external ophthalmoplegia 4 trials · 8 incl. sub-types
3 sub-types
- Kearns-Sayre syndrome 5 trials
- Progressive external ophthalmoplegia with mitochondrial DNA deletions 0 trials · 2 incl. sub-types Sub-types →
- Autosomal recessive progressive external ophthalmoplegia 0 trials Sub-types →
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Qualitative or quantitative defects of protein involved in O-glycosylation of alpha-dystroglycan 0 trials · 8 incl. sub-types
9 sub-types
- Qualitative or quantitative defects of FKRP 0 trials · 8 incl. sub-types Sub-types →
- Myopathy caused by variation in FKTN 1 trial · 2 incl. sub-types Sub-types →
- Myopathy caused by variation in CRPPA 0 trials · 1 incl. sub-types Sub-types →
- Myopathy caused by variation in GMPPB 0 trials · 1 incl. sub-types Sub-types →
- Myopathy caused by variation in POMT1 0 trials · 1 incl. sub-types Sub-types →
- Myopathy caused by variation in POMT2 0 trials · 1 incl. sub-types Sub-types →
- Qualitative or quantitative defects of protein O-mannosyltransferase 1 0 trials · 1 incl. sub-types Sub-types →
- Qualitative or quantitative defects of protein O-mannosyltransferase 2 0 trials · 1 incl. sub-types Sub-types →
- Limb-girdle muscular dystrophy due to POMK deficiency 0 trials
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RYR1-related myopathy 5 trials · 6 incl. sub-types
5 sub-types
- Central core myopathy 2 trials
- King-Denborough syndrome 0 trials
- Congenital multicore myopathy with external ophthalmoplegia 0 trials
- Congenital myopathy with myasthenic-like onset 0 trials
- Rhabdomyolysis-myalgia syndrome 0 trials
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MERRF syndrome 5 trials
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Smith-Magenis syndrome 5 trials
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Congenital nystagmus 4 trials · 5 incl. sub-types
10 sub-types
- Spinocerebellar ataxia 27A 1 trial
- Nystagmus 1, congenital, X-linked 0 trials
- Nystagmus 2, congenital, autosomal dominant 0 trials
- Nystagmus 3, congenital, autosomal dominant 0 trials
- Nystagmus 5, congenital, X-linked 0 trials
- Nystagmus 6, congenital, X-linked 0 trials
- Nystagmus 7, congenital, autosomal dominant 0 trials
- Nystagmus, congenital, autosomal recessive 0 trials
- Nystagmus, hereditary vertical 0 trials
- Nystagmus, myoclonic 0 trials
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Corpus callosum, agenesis of 4 trials
2 sub-types
- Kozlowski Ouvrier syndrome 0 trials
- Calloso-genital dysplasia 0 trials
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4 sub-types
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TTN-related myopathy 2 trials · 4 incl. sub-types
2 sub-types
- Autosomal recessive titinopathy 0 trials · 2 incl. sub-types Sub-types →
- Autosomal dominant titinopathy 0 trials Sub-types →
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Rubinstein-Taybi syndrome 3 trials
3 sub-types
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Cerebral cavernous malformation 3 trials
1 sub-type
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Familial congenital mirror movements 3 trials
4 sub-types
- Mirror movements 1 and/or agenesis of the corpus callosum 0 trials Sub-types →
- Mirror movements 2 0 trials
- Mirror movements 3 0 trials
- Mirror movements 4 0 trials
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1 sub-type
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TPM2-related myopathy 1 trial · 3 incl. sub-types
2 sub-types
- Central core myopathy 2 trials
- Congenital myopathy 23 0 trials
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Congenital hydrocephalus 1 trial · 3 incl. sub-types
8 sub-types
- X-linked hydrocephalus with stenosis of the aqueduct of Sylvius 1 trial
- Hydrocephalus, nonsyndromic, autosomal recessive 1 1 trial
- Autosomal recessive hydrocephalus due to congenital stenosis of aqueduct of Sylvius 0 trials
- Congenital communicating hydrocephalus 0 trials
- Congenital non-communicating hydrocephalus 0 trials
- Hydrocephalus, congenital, 3, with brain anomalies 0 trials
- Hydrocephalus, nonsyndromic, autosomal recessive 2 0 trials
- Hydrocephalus-blue sclerae-nephropathy syndrome 0 trials
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Postsynaptic congenital myasthenic syndrome 0 trials · 3 incl. sub-types
14 sub-types
- Congenital myasthenic syndrome 10 3 trials
- Congenital myasthenic syndrome 17 1 trial
- Congenital myasthenic syndrome 8 1 trial
- Congenital myasthenic syndrome 9 1 trial
- Congenital myasthenic syndrome 11 0 trials
- Congenital myasthenic syndrome 16 0 trials
- Congenital myasthenic syndrome 19 0 trials
- Congenital myasthenic syndrome 1A 0 trials Sub-types →
- Congenital myasthenic syndrome 2A 0 trials
- Congenital myasthenic syndrome 2C 0 trials
- Congenital myasthenic syndrome 3A 0 trials
- Congenital myasthenic syndrome 3B 0 trials
- Congenital myasthenic syndrome 3C 0 trials
- Congenital myasthenic syndrome 4 0 trials Sub-types →
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FOXG1 disorder 2 trials
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KBG syndrome 2 trials
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Mobius syndrome 2 trials
1 sub-type
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Mowat-Wilson syndrome 2 trials
2 sub-types
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PHACE syndrome 2 trials
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Congenital toxoplasmosis 2 trials
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1 sub-type
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Meningocele 2 trials
1 sub-type
- Meningoencephalocele 0 trials
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Childhood-onset nemaline myopathy 1 trial · 2 incl. sub-types
6 sub-types
- Nemaline myopathy 6 1 trial
- Congenital myopathy 23 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Congenital myopathy 4B, autosomal recessive 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 9 0 trials
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AICA-ribosiduria 1 trial
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Bohring-Opitz syndrome 1 trial
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Cohen syndrome 1 trial
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Freeman-Sheldon syndrome 1 trial
1 sub-type
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Schuurs-Hoeijmakers syndrome 1 trial
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TPM3-related myopathy 1 trial
3 sub-types
- Cap myopathy 0 trials
- Congenital myopathy 4A, autosomal dominant 0 trials
- Congenital myopathy 4B, autosomal recessive 0 trials
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Anencephaly 1 trial
4 sub-types
- Anencephaly 1 0 trials
- Anencephaly 2 0 trials
- Hydranencephaly 0 trials Sub-types →
- Isolated anencephaly 0 trials
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Polymicrogyria 1 trial
2 sub-types
- Bilateral polymicrogyria 0 trials Sub-types →
- Unilateral polymicrogyria 0 trials Sub-types →
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1 sub-type
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Cephalocele 0 trials · 1 incl. sub-types
2 sub-types
- Isolated encephalocele 1 trial Sub-types →
- Meningoencephalocele 0 trials
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Myopathy caused by variation in POMGNT1 0 trials · 1 incl. sub-types
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Neuropathy, congenital hypomelinating 0 trials · 1 incl. sub-types
3 sub-types
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Severe congenital nemaline myopathy 0 trials · 1 incl. sub-types
5 sub-types
- Nemaline myopathy 8 1 trial
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Nemaline myopathy 10 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 9 0 trials
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3q27.3 microdeletion syndrome 0 trials
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7p22.1 microduplication syndrome 0 trials
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9q31.1q31.3 microdeletion syndrome 0 trials
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9q33.3q34.11 microdeletion syndrome 0 trials
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Aase-Smith syndrome 0 trials
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Bailey-Bloch congenital myopathy 0 trials
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Bardet-Biedl syndrome 11 0 trials
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EEM syndrome 0 trials
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Houge-Janssens syndrome 1 0 trials
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Johanson-Blizzard syndrome 0 trials
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MYH7-related skeletal myopathy 0 trials
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Myhre syndrome 0 trials
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Pierpont syndrome 0 trials
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Pitt-Hopkins-like syndrome 2 0 trials
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Prader-Willi-like syndrome 0 trials
3 sub-types
- 6q16 deletion syndrome 0 trials
- BDV syndrome 0 trials
- SIM1-related Prader-Willi-like syndrome 0 trials
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Ritscher-Schinzel syndrome 0 trials
4 sub-types
- Ritscher-Schinzel syndrome 1 0 trials
- Ritscher-Schinzel syndrome 2 0 trials
- Ritscher-Schinzel syndrome 3 0 trials
- Ritscher-Schinzel syndrome 4 0 trials
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1 sub-type
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SLC39A8-CDG 0 trials
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Schinzel-Giedion syndrome 0 trials
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Wieacker-Wolff syndrome 0 trials
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1 sub-type
- Adrenal hypoplasia, cytomegalic type 0 trials
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2 sub-types
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Adult-onset nemaline myopathy 0 trials
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Alpha-actinopathy 0 trials
4 sub-types
- Cap myopathy 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Progressive scapulohumeroperoneal distal myopathy 0 trials
- Zebra body myopathy 0 trials
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3 sub-types
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Cerebellar-facial-dental syndrome 0 trials
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Congenital abducens nerve palsy 0 trials
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Congenital achiasma 0 trials
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Congenital epulis 0 trials
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Congenital myasthenic syndrome 15 0 trials
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Congenital myasthenic syndrome 18 0 trials
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Congenital myasthenic syndrome 6 0 trials
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3 sub-types
- Congenital myasthenic syndrome 12 0 trials
- Congenital myasthenic syndrome 13 0 trials
- Congenital myasthenic syndrome 14 0 trials
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Congenital oculomotor nerve palsy 0 trials
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Congenital trigeminal anesthesia 0 trials
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Craniorachischisis 0 trials
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Diastematomyelia 0 trials
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Holoprosencephaly 0 trials
17 sub-types
- Alobar holoprosencephaly 0 trials Sub-types →
- Chromosome 1q41-q42 deletion syndrome 0 trials
- Holoprosencephaly 1 0 trials
- Holoprosencephaly 10 0 trials
- Holoprosencephaly 11 0 trials
- Holoprosencephaly 12 with or without pancreatic agenesis 0 trials
- Holoprosencephaly 13, X-linked 0 trials
- Holoprosencephaly 14 0 trials
- Holoprosencephaly 2 0 trials
- Holoprosencephaly 3 0 trials Sub-types →
- Holoprosencephaly 4 0 trials
- Holoprosencephaly 6 0 trials
- Holoprosencephaly 7 0 trials
- Holoprosencephaly 8 0 trials
- Lobar holoprosencephaly 0 trials Sub-types →
- Microform holoprosencephaly 0 trials Sub-types →
- Semilobar holoprosencephaly 0 trials
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Hyaline body myopathy 0 trials
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Hypomyelinating leukodystrophy 10 0 trials
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Intellectual disability, Wolff type 0 trials
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Intermediate nemaline myopathy 0 trials
4 sub-types
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Congenital myopathy 4B, autosomal recessive 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 9 0 trials
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Isolated exencephaly 0 trials
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Lissencephaly spectrum disorders 0 trials
14 sub-types
- Baraitser-Winter cerebrofrontofacial syndrome 0 trials Sub-types →
- Warburg micro syndrome 0 trials Sub-types →
- X-linked lissencephaly with abnormal genitalia 0 trials
- Classic lissencephaly 0 trials Sub-types →
- Cobblestone lissencephaly 0 trials Sub-types →
- Craniotelencephalic dysplasia 0 trials
- Lissencephaly 10 0 trials
- Lissencephaly 7 with cerebellar hypoplasia 0 trials
- Lissencephaly 8 0 trials
- Lissencephaly spectrum disorder with complex brainstem malformation 0 trials Sub-types →
- Lissencephaly type 3 0 trials Sub-types →
- Lissencephaly with cerebellar hypoplasia 0 trials Sub-types →
- Massa casaer ceulemans syndrome 0 trials
- Microlissencephaly 0 trials Sub-types →
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Myasthenic syndrome, congenital, 22 0 trials
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Myofibrillar myopathy 1 0 trials
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Myofibrillar myopathy 3 0 trials
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Myofibrillar myopathy 4 0 trials
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Myofibrillar myopathy 5 0 trials
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Myofibrillar myopathy 7 0 trials
1 sub-type
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Periventricular nodular heterotopia 0 trials
8 sub-types
- Chromosome 5Q14.3 deletion syndrome, distal 0 trials
- Heterotopia, periventricular, X-linked dominant 0 trials
- Heterotopia, periventricular, associated with chromosome 5P anomalies 0 trials
- Periventricular heterotopia with microcephaly, autosomal recessive 0 trials
- Periventricular nodular heterotopia 6 0 trials
- Periventricular nodular heterotopia 7 0 trials
- Periventricular nodular heterotopia 8 0 trials
- Periventricular nodular heterotopia 9 0 trials
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2 sub-types
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Schizencephaly 0 trials
2 sub-types
- Acquired schizencephaly 0 trials
- Familial schizencephaly 0 trials
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Segmental spinal dysgenesis 0 trials
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Subcortical band heterotopia 0 trials
2 sub-types
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Typical nemaline myopathy 0 trials
6 sub-types
- Congenital myopathy 23 0 trials
- Congenital myopathy 2a, typical, autosomal dominant 0 trials
- Nemaline myopathy 10 0 trials
- Nemaline myopathy 2 0 trials
- Nemaline myopathy 7 0 trials
- Nemaline myopathy 9 0 trials
Most studied deeper sub-types
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One shot of gene therapy aims to fix a rare Muscle-Weakening disease
Cure Not yet recruitingResearchers are testing AMP-101, an experimental gene therapy, in people with DOK7 congenital myasthenic syndrome, a rare inherited disorder that causes muscle weakness and exercise intolerance. About 4 participants with moderate to severe DOK7 CMS will receive a single intraveno…
Phase 1 • Sponsor: Amplo Biotechnology • Aim: Cure
Last updated Sep 21, 2026 19:00 UTC
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Bladder cocktail aims to stop recurrent UTIs in spinal cord injury
Disease control Not yet recruitingThis early study tests whether washing the bladder with an antibiotic (gentamicin) followed by a probiotic (Lactobacillus) can help restore healthy bacteria and prevent urinary tract infections in people with neurogenic bladder due to spinal cord injury, multiple sclerosis, or sp…
Early phase 1 • Sponsor: Medstar Health Research Institute • Aim: Disease control
Last updated Aug 23, 2026 00:00 UTC
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One-Time gene injection aims to rescue sight in rare childhood blindness
Disease control Not yet recruitingThis study tests a gene therapy called OPGx-RDH12 for people with Leber Congenital Amaurosis (LCA) caused by mutations in the RDH12 gene, a rare condition that leads to severe vision loss from a young age. The treatment is given as a single injection under the retina in the worse…
Phase 1/2 • Sponsor: Opus Genetics, Inc • Aim: Disease control
Last updated Jul 17, 2026 00:00 UTC
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Daily pill may replace growth hormone shots for children
Disease control Not yet recruitingThis study evaluates the long-term safety of an oral medication called LUM-201 for children with growth hormone deficiency (GHD). Children who completed a prior 12-month trial can receive LUM-201 daily for up to 36 additional months. The goal is to see if the pill is safe and tol…
Phase 3 • Sponsor: Lumos Pharma • Aim: Disease control
Last updated Jul 10, 2026 00:00 UTC
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New device aims to save Kids' voices during surgery
Disease control Not yet recruitingThis study tests a nerve-monitoring device that alerts surgeons when a child's vocal cord nerve is at risk during throat, chest, or heart surgery. About 90 children under 18 will be randomly assigned to standard care or standard care plus the monitor. After surgery, doctors will …
Sponsor: University of Alberta • Aim: Disease control
Last updated Jun 27, 2026 12:36 UTC
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Scientists grow penile tissue in lab for first human trial
Disease control Not yet recruitingThis early-stage trial tests a lab-grown penile tissue made from a man's own cells to repair severe, irreversible damage to the penis. Ten men aged 18-60 who have failed standard treatments will receive the implant. The main goal is to check safety, not effectiveness.
Phase 1 • Sponsor: Wake Forest University Health Sciences • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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One-Patient trial aims to treat Ultra-Rare genetic disorder
Disease control Not yet recruitingThis study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Schuurs-Hoeijmakers syndrome, a rare genetic condition. The drug aims to correct a specific genetic mutation to improve communication and motor skills. Only one participant …
Phase 1/2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 11:01 UTC
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New Long-Acting growth hormone shot could mean fewer needles for kids
Disease control Not yet recruitingThis study tests a new medicine called GenSci134, a long-acting growth hormone, in 128 children with growth hormone deficiency (GHD). The goal is to find the best dose and see if it is safe and helps children grow taller. In the first part, children get one shot of GenSci134; in …
Phase 1/2 • Sponsor: Changchun GeneScience Pharmaceutical Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 09:11 UTC
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A 5-Day genetic test could slash time to transplant for children with rare immune disease
Diagnosis Not yet recruitingThis trial tests whether a new ultra-rapid genetic test, using third-generation sequencing, can diagnose familial lymphohistiocytosis (FHL) in children within 5 days instead of the usual 6-8 weeks. FHL is a rare, life-threatening genetic condition where the immune system attacks …
Sponsor: Assistance Publique Hopitaux De Marseille • Aim: Diagnosis
Last updated Aug 05, 2026 00:00 UTC
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Texts and calls could save lives: new study aims to boost baby vaccinations
Prevention Not yet recruitingThis study tests whether sending text messages or voice call reminders with helpful behavior change tips can improve how many babies get their full set of vaccines on time. About 7,800 caregivers of newborns in Bangladesh will be split into three groups: one gets no reminders, on…
Sponsor: Japan Institute for Health Secutiry • Aim: Prevention
Last updated Jun 27, 2026 12:07 UTC
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Can a common antipsychotic keep cannabis users out of the ER?
Symptom relief Not yet recruitingThis trial tests whether giving patients a prescription for haloperidol to use as needed at home can help control symptoms of cannabinoid hyperemesis syndrome — a condition marked by severe nausea, vomiting, and abdominal pain from heavy cannabis use. The goal is to see if having…
Early phase 1 • Sponsor: University of Illinois at Chicago • Aim: Symptom relief
Last updated Sep 03, 2026 00:00 UTC
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Can acupuncture needles Kick-Start a paralyzed stomach after cancer surgery?
Symptom relief Not yet recruitingThis study tests whether electroacupuncture (mild electrical pulses through thin needles) can help patients whose stomachs stop working properly after surgery for digestive tract tumors. Seventy-six adults who had such surgery and developed gastroparesis (stomach paralysis) will …
Sponsor: First Teaching Hospital of Tianjin University of Traditional Chinese Medicine • Aim: Symptom relief
Last updated Jun 27, 2026 12:23 UTC
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Can a common asthma drug help newborns breathe easier?
Symptom relief Not yet recruitingThis study tests whether inhaled salbutamol, a drug used for asthma, can reduce breathing distress in newborns with transient tachypnea of the newborn (TTN). TTN is a common lung condition after birth where fluid stays in the lungs, causing fast breathing. The trial will enroll j…
Sponsor: Tishreen University • Aim: Symptom relief
Last updated Jun 27, 2026 08:12 UTC
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New hope for Prader-Willi: drug targets uncontrollable hunger
Symptom relief Not yet recruitingThis study tests an experimental drug called BMB-101 in 16 adults with Prader-Willi syndrome who struggle with severe, constant hunger. The trial is double-blind and placebo-controlled, meaning some participants get the drug and some get a dummy treatment, and neither they nor th…
Phase 2 • Sponsor: Bright Minds Biosciences Pty Ltd • Aim: Symptom relief
Last updated Jun 27, 2026 08:02 UTC
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Hidden brain disease study aims to prevent stroke and dementia
Knowledge-focused Not yet recruitingThis study looks at people aged 65 and older who have signs of brain blood vessel disease on a past scan but no history of stroke, dementia, or other major brain conditions. Researchers will collect information on daily function, thinking skills, speech, and any new vascular even…
Sponsor: University of Edinburgh • Aim: Knowledge-focused
Last updated Sep 11, 2026 00:00 UTC
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Can ultrasound make throat procedures easier and safer?
Knowledge-focused Not yet recruitingThis pilot study will test whether using ultrasound during common office procedures for voice and swallowing problems is helpful and practical. Researchers will enroll 200 adults with conditions like chronic cough, vocal cord issues, or swallowing difficulties. They will measure …
Sponsor: Weill Medical College of Cornell University • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:00 UTC
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300 belgian kids to help unlock growth Hormone's metabolic secrets
Knowledge-focused Not yet recruitingThis study will follow 300 Belgian children with growth hormone deficiency for three years to see how their bodies respond to daily or weekly growth hormone shots. Researchers will measure growth, weight, blood sugar, fats, and other health markers. The goal is to understand whic…
Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:31 UTC
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Rare syndrome study aims to map dental and facial patterns
Knowledge-focused Not yet recruitingThis study looks at the teeth, mouth, and face health of 25 people with Mowat-Wilson syndrome, a rare genetic condition. Researchers will check for cavities, gum disease, and facial features, and ask about quality of life. No treatment is given—the goal is to gather information t…
Sponsor: University of Milan • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Hidden bladder risks in kids with spine defects under investigation
Knowledge-focused Not yet recruitingThis study looks at children with spinal dysraphism, a birth defect where the spine doesn't close properly. The goal is to better understand bladder and bowel problems in these children, especially those with less obvious forms. By studying 210 children treated at one hospital ov…
Sponsor: University Hospital, Angers • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:01 UTC
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Blood and urine may hold clues to rare breathing condition
Knowledge-focused Not yet recruitingThis study aims to find biological markers in blood and urine that could explain congenital central hypoventilation syndrome (CCHS), a rare disorder where the brain fails to control breathing during sleep. Researchers will collect samples from 40 adults with CCHS and healthy volu…
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:53 UTC