Ultragenyx UK Clinical Trials

Company details

Status: Public
HQ: Newcastle upon tyne, NE128RL, GB, NOVATO, CA
Founded: 2024
Employees: 2
Programs: 201
Drugs: 168
Patents: 23

Official website ↗ All projects Drug pipeline Patents News & analysis

Recent trial activity

8 updates

Latest clinical program signals indexed for Ultragenyx UK.

Trial registry

216 studies

NCT00004274
Estrogen

Unknown completed small_molecule

Turner's Syndrome

CT.gov ↗
NCT00004275
growth hormone

Phase 2 completed small_molecule

Turner's Syndrome

CT.gov ↗
NCT00004276
thalidomide

Phase 2 completed small_molecule

HIV Infections

CT.gov ↗
NCT00004278
ganciclovir

Phase 3 completed small_molecule

Cytomegalovirus Infections

CT.gov ↗
NCT00004284
potassium citrate

Phase 3 completed small_molecule

Hypercalciuria

CT.gov ↗
NCT00004286
immune globulin

Phase 3 completed small_molecule

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

CT.gov ↗
NCT00004287
H5.001CBCFTR

Phase 1 completed gene_therapy

Cystic Fibrosis

CT.gov ↗
NCT00004288
olsalazine

Phase 2 completed small_molecule

Ankylosing Spondylitis

CT.gov ↗
NCT00004297
diazepam

Phase 3 completed small_molecule

Status Epilepticus

CT.gov ↗
NCT00004300
bromocriptine

Phase 2 on hold small_molecule

Stereotyped Behavior

CT.gov ↗
NCT00004311
leuprolide acetate

Phase 2 completed small_molecule

Acanthosis Nigricans

CT.gov ↗
NCT00004313
dehydroepiandrosterone

Phase 3 completed small_molecule

Addison's Disease

CT.gov ↗
NCT00004314
aminomidazole carboxamide riboside

Phase 2 completed small_molecule

Lesch-Nyhan Syndrome

CT.gov ↗
NCT00004317
Leucovorin calcium

Approved active small_molecule

Toxoplasmosis

CT.gov ↗
NCT00004327
octreotide

Phase 2 completed small_molecule

Hereditary Hemorrhagic Telangiectasia

CT.gov ↗
NCT00004328
enalapril

Phase 2 completed small_molecule

Pseudohypoaldosteronism

CT.gov ↗
NCT00004333
NTBC

Phase 2 completed small_molecule

Tyrosinemia, Type I

CT.gov ↗
NCT00004359
methoxsalen

Phase 2 completed small_molecule

Epidermolysis Bullosa Acquisita

CT.gov ↗
NCT00004368
colchicine

Phase 1 active small_molecule

Cirrhosis

CT.gov ↗
NCT00004376
guanfacine

Phase 3 completed small_molecule

Tourette Syndrome

CT.gov ↗
NCT00004380
relaxin

Phase 2 completed small_molecule

Systemic Sclerosis

CT.gov ↗
NCT00004381
tin mesoporphyrin

Phase 2 completed small_molecule

Glucosephosphate Dehydrogenase Deficiency

CT.gov ↗
NCT00004382
tin mesoporphyrin

Phase 2 completed small_molecule

Hyperbilirubinemia

CT.gov ↗
NCT00004386
recombinant adenovirus containing the ornithine transcarbamylase gene

Phase 1 terminated gene_therapy

Ornithine Transcarbamylase Deficiency Disease

CT.gov ↗
NCT00004387
fetal nigral transplantation

Phase 2 completed other

Parkinson Disease

CT.gov ↗
NCT00004390
desipramine

Phase 3 completed small_molecule

Pain

CT.gov ↗
NCT00004393
risperidone

Phase 2 completed small_molecule

Tourette Syndrome

CT.gov ↗
NCT00004397
heme arginate

Phase 2 completed small_molecule

Porphyria

CT.gov ↗
NCT00004398
heme arginate

Phase 2 completed small_molecule

Porphyria

CT.gov ↗
NCT00004454
lymphocyte gene therapy

Phase 2 completed gene_therapy

Mucopolysaccharidosis II

CT.gov ↗
NCT00004474
Anti-thymocyte globulin

Phase 3 completed small_molecule

Aplastic Anemia

CT.gov ↗
NCT00004478
droxidopa

Unknown completed small_molecule

Shy-Drager Syndrome

CT.gov ↗
NCT00004479
midodrine

Unknown completed small_molecule

Syncope

CT.gov ↗
NCT00004480
botulinum toxin type A

Unknown completed small_molecule

Hyperhidrosis

CT.gov ↗
NCT00004485
cyclosporine

Phase 2 completed small_molecule

Sickle Cell Anemia

CT.gov ↗
NCT00004642
SDZ MSL-109

Phase 2 completed small_molecule

Cytomegalovirus Infections

CT.gov ↗
NCT00004643
cytarabine

Phase 2 completed small_molecule

Lupus Erythematosus, Systemic

CT.gov ↗
NCT00004644
acyclovir

Phase 3 completed small_molecule

Herpes Simplex

CT.gov ↗
NCT00004645
Plasma exchange

Phase 3 active other

Acute Disseminated Encephalomyelitis

CT.gov ↗
NCT00004646
prednisone

Phase 3 completed small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT00004647
mexiletine

Phase 3 completed small_molecule

Pain

CT.gov ↗
NCT00004650
conjugated estrogens

Phase 3 completed small_molecule

Osteoporosis

CT.gov ↗
NCT00004652
pimozide

Phase 2 completed small_molecule

Tourette Syndrome

CT.gov ↗
NCT00004654
soy protein isolate

Phase 3 completed small_molecule

Hereditary Hemorrhagic Telangiectasia

CT.gov ↗
NCT00004658
ribose

Phase 2 completed small_molecule

5'-Nucleotidase Syndrome

CT.gov ↗
NCT00004659
irradiation

Phase 2 active other

Brain Neoplasms

CT.gov ↗
NCT00004662
dehydroepiandrosterone

Phase 3 completed small_molecule

Systemic Lupus Erythematosus

CT.gov ↗
NCT00004664
megestrol

Unknown completed small_molecule

Acquired Immunodeficiency Syndrome

CT.gov ↗
NCT00004665
dehydroepiandrosterone

Phase 3 completed small_molecule

Systemic Lupus Erythematosus

CT.gov ↗
NCT00004667
von Willebrand factor

Phase 1 completed small_molecule

Von Willebrand's Disease

CT.gov ↗
NCT00004669
hydrocortisone

Phase 2 completed small_molecule

Bronchopulmonary Dysplasia

CT.gov ↗
NCT00004670
pallidotomy

Phase 2 completed other

Parkinson Disease

CT.gov ↗
NCT00004744
immune globulin

Phase 3 completed small_molecule

Multiple Sclerosis

CT.gov ↗
NCT00004747
mucoid exopolysaccharide P. aeruginosa immune globulin IV

Phase 2 completed small_molecule

Cystic Fibrosis

CT.gov ↗
NCT00004748
colchicine

Phase 3 completed small_molecule

Liver Cirrhosis, Biliary

CT.gov ↗
NCT00004751
Surgery

Phase 2 active other

Spastic Diplegia

CT.gov ↗
NCT00004754
epoprostenol

Approved completed small_molecule

Hypertension, Pulmonary

CT.gov ↗
NCT00004755
allopurinol

Phase 2 completed small_molecule

Leishmaniasis

CT.gov ↗
NCT00004757
acyclovir

Phase 3 completed small_molecule

Herpes Simplex

CT.gov ↗
NCT00004758
carbamazepine

Phase 2 completed small_molecule

Spasms, Infantile

CT.gov ↗
NCT00004759
methylprednisolone

Phase 3 completed small_molecule

Spinal Cord Injury

CT.gov ↗
NCT00004762
cladribine

Phase 2 completed small_molecule

Cholangitis, Sclerosing

CT.gov ↗
NCT00004763
leuprolide

Phase 2 completed small_molecule

Hyperandrogenism

CT.gov ↗
NCT00004767
Sodium Benzoate

Phase 2 completed small_molecule

Amino Acid Metabolism, Inborn Errors

CT.gov ↗
NCT00004770
thioctic acid

Unknown completed small_molecule

Mitochondrial Myopathy

CT.gov ↗
NCT00004771
leuprolide

Phase 2 completed small_molecule

Spinal Muscular Atrophy

CT.gov ↗
NCT00004772
immune globulin

Phase 3 completed small_molecule

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

CT.gov ↗
NCT00004776
topiramate

Phase 3 completed small_molecule

Lennox-Gastaut Syndrome

CT.gov ↗
NCT00004778
betamethasone

Phase 3 completed small_molecule

Respiratory Distress Syndrome

CT.gov ↗
NCT00004779
Ad5-CB-CFTR

Phase 1 completed gene_therapy

Cystic Fibrosis

CT.gov ↗
NCT00004784
methotrexate

Phase 3 completed small_molecule

Liver Cirrhosis, Biliary

CT.gov ↗
NCT00004785
dexamethasone

Phase 3 completed small_molecule

Bronchopulmonary Dysplasia

CT.gov ↗
NCT00004786
iloprost

Phase 3 completed small_molecule

Systemic Sclerosis

CT.gov ↗
NCT00004787
filgrastim

Phase 2 completed small_molecule

Shwachman Syndrome

CT.gov ↗
NCT00004789
heme arginate

Phase 2 completed small_molecule

Porphyria

CT.gov ↗
NCT00004794
cidofovir

Phase 2 completed small_molecule

Cytomegalovirus Retinitis

CT.gov ↗
NCT00004795
dehydroepiandrosterone

Phase 3 completed small_molecule

Systemic Lupus Erythematosus

CT.gov ↗
NCT00004796
lactulose

Phase 2 completed small_molecule

Portal Hypertension

CT.gov ↗
NCT00004797
fructose

Unknown completed small_molecule

Sickle Cell Anemia

CT.gov ↗
NCT00004799
whole-cell pertussis vaccine

Phase 3 completed mab

Pertussis

CT.gov ↗
NCT00004800
diphtheria-tetanus-pertussis vaccine containing whole-cell pertussis

Phase 3 completed mab

Pertussis

CT.gov ↗
NCT00004801
monoclonal factor IX replacement therapy

Phase 2 completed small_molecule

Hemophilia B

CT.gov ↗
NCT00004802
dichlorphenamide

Phase 3 completed small_molecule

Paralysis, Hyperkalemic Periodic

CT.gov ↗
NCT00004804
interferon alfa

Phase 3 completed small_molecule

Hepatitis C

CT.gov ↗
NCT00004807
dextromethorphan

Unknown completed small_molecule

Rett Syndrome

CT.gov ↗
NCT00004808
fluconazole

Phase 2 completed small_molecule

Histoplasmosis

CT.gov ↗
NCT00004809
gene therapy

Phase 1 completed other

Familial Hypercholesterolemia

CT.gov ↗
NCT00004810
-aminosalicylic acid

Phase 2 completed small_molecule

Ulcerative Colitis

CT.gov ↗
NCT00004811
itraconazole

Phase 2 completed small_molecule

Histoplasmosis

CT.gov ↗
NCT00004812
Exercise

Phase 2 completed other

Spinal Cord Injury

CT.gov ↗
NCT00004814
copolymer 1

Phase 3 completed small_molecule

Multiple Sclerosis

CT.gov ↗
NCT00004815
growth hormone

Unknown completed small_molecule

Werner's Syndrome

CT.gov ↗
NCT00004816
monoclonal antibody M-T412

Phase 1 completed small_molecule

Multiple Sclerosis

CT.gov ↗
NCT00004817
phenytoin

Phase 3 completed small_molecule

Post-Traumatic Seizure Disorder

CT.gov ↗
NCT00004838
succimer

Unknown completed small_molecule

Lead Poisoning

CT.gov ↗
NCT00004840
thyrotropin-releasing hormone

Unknown completed small_molecule

Respiratory Distress Syndrome

CT.gov ↗
NCT00004938
fluconazole

Phase 2 completed small_molecule

Sporotrichosis

CT.gov ↗
NCT00004939
,4-diaminopyridine

Phase 3 completed small_molecule

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

CT.gov ↗
NCT00005652
rituximab

Phase 2 completed small_molecule

Purpura, Thrombocytopenic, Idiopathic

CT.gov ↗
NCT00005654
clomiphene citrate

Unknown completed small_molecule

Hyperinsulinism

CT.gov ↗
NCT00005891
cyclophosphamide

Unknown completed small_molecule

Fanconi's Anemia

CT.gov ↗
NCT00005892
cyclophosphamide

Unknown completed small_molecule

Fanconi's Anemia

CT.gov ↗
NCT00005893
anti-thymocyte globulin

Unknown completed small_molecule

Neutropenia

CT.gov ↗
NCT00005896
filgrastim

Phase 1 active small_molecule

Fanconi's Anemia

CT.gov ↗
NCT00005898
anti-thymocyte globulin

Phase 2 completed small_molecule

Fanconi's Anemia

CT.gov ↗
NCT00006054
anti-thymocyte globulin

Unknown terminated small_molecule

Immunologic Deficiency Syndromes

CT.gov ↗
NCT00006055
anti-thymocyte globulin

Unknown active small_molecule

Purpura, Schoenlein-Henoch

CT.gov ↗
NCT00006056
anti-thymocyte globulin

Unknown active small_molecule

Chediak-Higashi Syndrome

CT.gov ↗
NCT00006061
phosphatidylcholine

Unknown completed small_molecule

Methionine Adenosyltransferase Deficiency

CT.gov ↗
NCT00006063
pancrelipase with bicarbonate

Unknown completed small_molecule

Cystic Fibrosis

CT.gov ↗
NCT00006127
amifostine

Phase 1 active small_molecule

Fanconi's Anemia

CT.gov ↗
NCT00006136
arginine butyrate

Phase 2 completed small_molecule

Beta-Thalassemia

CT.gov ↗
NCT00006137
enalapril

Unknown completed small_molecule

IGA Glomerulonephritis

CT.gov ↗
NCT00006275
calcitriol

Unknown completed small_molecule

Psoriasis

CT.gov ↗
NCT00010361
cyclosporine

Unknown completed small_molecule

Metabolism, Inborn Errors

CT.gov ↗
NCT00010387
cyclophosphamide

Phase 2 completed small_molecule

Anemia, Hemolytic, Autoimmune

CT.gov ↗
NCT00010400
Cyclophosphamide

Unknown completed small_molecule

Systemic Lupus Erythematosus

CT.gov ↗
NCT00010413
cyclophosphamide

Phase 2 completed small_molecule

Pemphigus

CT.gov ↗
NCT00014755
anti-thymocyte globulin

Phase 1 completed small_molecule

Multiple Sclerosis

CT.gov ↗
NCT00016445
growth hormone

Phase 2 completed small_molecule

Cystic Fibrosis

CT.gov ↗
NCT00016458
anti-thymocyte globulin

Phase 2 completed small_molecule

Systemic Sclerosis

CT.gov ↗
NCT00017628
cyclophosphamide

Phase 1 completed small_molecule

Multiple Sclerosis

CT.gov ↗
NCT00017641
anti-thymocyte globulin

Phase 1 active small_molecule

Systemic Lupus Erythematosus

CT.gov ↗
NCT00017654
anti-thymocyte globulin

Unknown active small_molecule

Graft Versus Host Disease

CT.gov ↗
NCT00017680
Melphalan

Phase 2 completed small_molecule

Amyloidosis

CT.gov ↗
NCT00319878
Sirolimus

Phase 2 active small_molecule

Anemia, Aplastic

CT.gov ↗
NCT00345605
Sodium Phenylbutyrate

Phase 2 completed small_molecule

Argininosuccinic Aciduria

CT.gov ↗
NCT00348933
Betaine

Unknown completed small_molecule

Angelman Syndrome

CT.gov ↗
NCT00414648
Sirolimus

Phase 3 completed small_molecule

Lymphangioleiomyomatosis

CT.gov ↗
NCT00466843
Antithymocyte globulin (ATG)

Phase 2 active small_molecule

Myelodysplastic Syndrome

CT.gov ↗
NCT00468208
Abatacept

Phase 2 completed small_molecule

Wegener's Granulomatosis

CT.gov ↗
NCT00556439
Abatacept

Phase 2 completed small_molecule

Takayasu's Arteritis

CT.gov ↗
NCT00657540
Analatro

Phase 3 completed small_molecule

Latrodectism

CT.gov ↗
NCT00839501
Acetazolamide

Phase 1 terminated small_molecule

Andersen-Tawil Syndrome

CT.gov ↗
NCT00947960
Triheptanoin

Phase 2 completed small_molecule

Adult Polyglucosan Body Disease

CT.gov ↗
NCT01287221
Rifampicin

Phase 3 terminated small_molecule

Multiple System Atrophy

CT.gov ↗
NCT01359319
Sialic Acid Extended Release (SA-ER) Tablets

Phase 1 completed small_molecule

Hereditary Inclusion Body Myopathy (HIBM)

CT.gov ↗
NCT01417091
BPS804

Phase 2 completed small_molecule

Osteogenesis Imperfecta

CT.gov ↗
NCT01419691
auranofin

Phase 2 completed small_molecule

Chronic Lymphocytic Leukemia (CLL)

CT.gov ↗
NCT01427517
N-acetylcysteine

Phase 1 completed small_molecule

Parkinson's Disease

CT.gov ↗
NCT01517880
Sialic Acid Extended Release (SA-ER)

Phase 2 completed small_molecule

GNE Myopathy

CT.gov ↗
NCT01634750
ManNAc

Phase 1 completed small_molecule

Hereditary Inclusion Body Myopathy (HIBM)

CT.gov ↗
NCT01830972
SA-ER 500 mg

Phase 2 completed small_molecule

GNE Myopathy

CT.gov ↗
NCT01856218
UX003

Phase 2 completed small_molecule

Mucopolysaccharidosis Type 7

CT.gov ↗
NCT01886378
UX007

Phase 2 completed small_molecule

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

CT.gov ↗
NCT01933724
5 mg prednisone

Phase 3 active small_molecule

Granulomatosis With Polyangiitis

CT.gov ↗
NCT01940094
5 mg prednisone

Phase 3 active small_molecule

Granulomatosis With Polyangiitis

CT.gov ↗
NCT01965691
Protein intake

Unknown completed other

Phenylketonuria

CT.gov ↗
NCT01993186
UX007

Phase 3 terminated small_molecule

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

CT.gov ↗
NCT02000960
Triheptanoin

Phase 2 active small_molecule

Glucose Transporter Type 1 Deficiency Syndrome

CT.gov ↗
NCT02014883
GLUT1 DS

Phase 2 completed small_molecule

Glut1 Deficiency Syndrome

CT.gov ↗
NCT02030015
miglustat

Approved terminated small_molecule

GM1 Gangliosidoses

CT.gov ↗
NCT02036853
Triheptanoin

Phase 2 completed small_molecule

Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

CT.gov ↗
NCT02038543
Hydroxyproline and Leucine

Phase 2 completed small_molecule

Hyperoxaluria

CT.gov ↗
NCT02051868
Cisplatin

Phase 2 active small_molecule

Squamous Cell Carcinoma of the Anus

CT.gov ↗
NCT02214160
UX007

Phase 2 completed small_molecule

Carnitine Palmitoyltransferase (CPT I or CPT II) Deficiency

CT.gov ↗
NCT02230566
UX003

Phase 3 completed small_molecule

MPS 7

CT.gov ↗
NCT02232477
Intrathecal recombinant human alpha iduronidase

Unknown terminated small_molecule

Mucopolysaccharidosis I

CT.gov ↗
NCT02377921
aceneuramic acid extended-release (Ace-ER)

Phase 3 completed small_molecule

Hereditary Inclusion Body Myopathy

CT.gov ↗
NCT02396459
Tiratricol

Phase 2 active small_molecule

Allan-Herndon-Dudley Syndrome

CT.gov ↗
NCT02418455
UX003

Phase 3 completed small_molecule

Sly Syndrome

CT.gov ↗
NCT02432144
UX003

Phase 3 completed small_molecule

Sly Syndrome

CT.gov ↗
NCT02432768
Triheptanoin

Phase 2 completed small_molecule

Glycogen Storage Disease Type V

CT.gov ↗
NCT02453061
Triheptanoin oil

Phase 2 completed small_molecule

Huntington Disease

CT.gov ↗
NCT02583672
N-acetylcysteine

Phase 2 completed small_molecule

Gaucher Disease Type 1

CT.gov ↗
NCT02599961
UX007

Phase 2 terminated small_molecule

Glucose Transporter Type 1 Deficiency Syndrome

CT.gov ↗
NCT02618915
DTX101

Phase 2 terminated gene_therapy

Hemophilia B

CT.gov ↗
NCT02679235
POST Triheptanoin

Phase 2 completed small_molecule

Frontal Lobe Hypometabolism

CT.gov ↗
NCT02696044
triheptanoin

Phase 2 active small_molecule

Rett Syndrome

CT.gov ↗
NCT02731690
Aceneuramic Acid Extended-Release

Phase 2 terminated small_molecule

Hereditary Inclusion Body Myopathy

CT.gov ↗
NCT02736188
Aceneuramic Acid Extended-Release Tablets

Phase 3 terminated small_molecule

Hereditary Inclusion Body Myopathy

CT.gov ↗
NCT02778867
2015-1949

Phase 3 completed small_molecule

Eosinophilic Gastrointestinal Disorders (EGIDs)

CT.gov ↗
NCT02939573
Colchicine

Phase 2 active small_molecule

Primary Cutaneous Vasculitis

CT.gov ↗
NCT02960217
UX007

Phase 3 terminated small_molecule

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)

CT.gov ↗
NCT02991144
scAAV8OTC

Phase 2 completed gene_therapy

Ornithine Transcarbamylase (OTC) Deficiency

CT.gov ↗
NCT02991807
RAD001

Phase 2 completed small_molecule

PTEN Gene Mutation

CT.gov ↗
NCT03006146
Sargramostim

Phase 1 completed small_molecule

Autoimmune Pulmonary Alveolar Proteinosis

CT.gov ↗
NCT03029091
Losartan Potassium

Phase 2 completed small_molecule

Eosinophilic Esophagitis

CT.gov ↗
NCT03059160
Tridecanoic Acid

Phase 2 active small_molecule

Rett Syndrome

CT.gov ↗
NCT03064048
Neo-ASA

Unknown completed other

Argininosuccinate Lyase Deficiency

CT.gov ↗
NCT03506425
Triheptanoin

Phase 2 completed small_molecule

ALS

CT.gov ↗
NCT03517085
DTX401

Phase 2 completed gene_therapy

GSD1

CT.gov ↗
NCT03581591
Burosumab

Phase 3 completed mab

Hypophosphatemia

CT.gov ↗
NCT03642860
Triheptanoin

Phase 2 completed small_molecule

Tarui Disease

CT.gov ↗
NCT03665636
Triheptanoin

Phase 1 completed small_molecule

Glycogen Storage Disease Type I

CT.gov ↗
NCT03810859
Blood sample

Unknown active mab

Amelogenesis Imperfecta

CT.gov ↗
NCT03877809
Sirolimus 0.5 mg

Phase 2 active small_molecule

Beta-Thalassemia

CT.gov ↗
NCT03993821
Burosumab

Phase 1 active small_molecule

Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS)

CT.gov ↗
NCT04247750
Sirolimus 0.5 mg

Phase 2 active small_molecule

Beta-Thalassemia

CT.gov ↗
NCT04259281
GTX-102

Phase 3 active small_molecule

Angelman Syndrome

CT.gov ↗
NCT04311307
[6,6-2H2]glucose

Phase 2 completed mab

Glycogen Storage Disease Type IA

CT.gov ↗
NCT04320316
Crysvita (burosumab-twza) Treatment

Approved completed small_molecule

Epidermal Nevus Syndrome

CT.gov ↗
NCT04360265
Adjuvant Immunomodulatory (IM) Therapy

Phase 3 active small_molecule

Mucopolysaccharidosis IIIA

CT.gov ↗
NCT04848714
AS/BW/ENE-21/0003

Phase 1 completed small_molecule

Latrodectism

CT.gov ↗
NCT04884815
UX701

Phase 2 active gene_therapy

Wilson Disease

CT.gov ↗
NCT04990388
UX053

Phase 2 terminated mab

Glycogen Storage Disease Type III

CT.gov ↗
NCT05139316
DTX401

Phase 3 completed gene_therapy

Glycogen Storage Disease Type IA

CT.gov ↗
NCT05345171
DTX301

Phase 3 active gene_therapy

OTC Deficiency

CT.gov ↗
NCT05402384
AVTX-801

Phase 2 active small_molecule

SLC35A2-CDG - Solute Carrier Family 35 Member A2 Congenital Disorder of Glycosylation

CT.gov ↗
NCT05514249
CRD-TMH-001

Phase 1 active small_molecule

Duchenne Muscular Dystrophy

CT.gov ↗
NCT05579327
Tiratricol

Phase 3 completed small_molecule

Monocarboxylate Transporter 8 Deficiency

CT.gov ↗
NCT05611528
Evinacumab

Phase 3 completed small_molecule

Homozygous Familial Hypercholesterolemia

CT.gov ↗
NCT05768854
Bisphosphonate

Phase 3 active small_molecule

Osteogenesis Imperfecta

CT.gov ↗
NCT05933200
Triheptanoin

Phase 3 active small_molecule

Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

CT.gov ↗
NCT06067802
Triheptanoin

Phase 2 active small_molecule

Medium-chain Acyl-CoA Dehydrogenase Deficiency

CT.gov ↗
NCT06284577
Multistrain Probiotic

Unknown active other

Cystic Fibrosis in Children

CT.gov ↗
NCT06340685
Triheptanoin

Phase 1 active small_molecule

Pyruvate Dehydrogenase Complex Deficiency

CT.gov ↗
NCT06415344
GTX-102

Phase 3 active small_molecule

Angelman Syndrome

CT.gov ↗
NCT06614894
Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day

Phase 3 active small_molecule

Sanfilippo Syndrome

CT.gov ↗
NCT06617429
GTX-102

Phase 3 active small_molecule

Angelman Syndrome

CT.gov ↗
NCT06636071
setrusumab

Phase 3 active mab

Osteogenesis Imperfecta

CT.gov ↗
NCT07097311
Triheptanoin

Phase 2 active small_molecule

Medium-chain Acyl-CoA Dehydrogenase Deficiency

CT.gov ↗
NCT07157254
GTX-102

Phase 3 active small_molecule

Angelman Syndrome

CT.gov ↗
NCT07247058
Osilodrostat (Isturisa)

Approved active small_molecule

Mild Autonomous Cortisol Secretion (MACS)

CT.gov ↗
NCT07511556
UX016

Phase 2 active small_molecule

GNE Myopathy

CT.gov ↗
NCT07530796
scAAV9/AGA

Phase 2 active gene_therapy

Aspartylglucosaminuria

CT.gov ↗