Clinical Trials on Albright Hereditaire Osteodystrofie

Total 2630 results

Maastricht University Medical Center
ZonMw: The Netherlands Organisation for Health Research and Development

Completed

DMEK Versus DSAEK Study (DMEK)

Fuchs' Endothelial Dystrophy

Netherlands
Oregon Health and Science University

Terminated

OCT-guided DSAEK Graft Shaping and Smoothing (DSAEK)

Fuchs' Endothelial Dystrophy

United States
Byron Lam
National Eye Institute (NEI)

Active, not recruiting

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy

United States
Santhera Pharmaceuticals

Completed

RHODOS Follow-up Single-visit Study (RHODOS-OFU)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
Santhera Pharmaceuticals

No longer available

Expanded Access Program for Idebenone in Patients With Leber's Hereditary Optic Neuropathy Who Completed the LEROS Study (US EAP LHON)

Leber's Hereditary Optic Neuropathy
Stealth BioTherapeutics Inc.

Completed

A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

Leber's Hereditary Optic Neuropathy

United States
University of Rochester
Livionex Inc

Completed

A Pilot Study to Assess the Preliminary Efficacy of EDTA Eye Drops on Band Keratopathy

Corneal Dystrophy, Band-Shaped

United States
Centre Hospitalier Universitaire de Besancon

Completed

Long Term Cornea Graft Survival Study (BELIEvE)

Keratoconus | Fuchs' Endothelial Dystrophy

France
University Hospital, Rouen

Completed

Comparison of Two Techniques in Achieving Corneal Graft

Fuch's Endothelial Dystrophy

France
Avellino Labs USA, Inc.

Unknown

Study of the Prevalence of TGFBI Corneal Dystrophies

Corneal Dystrophy
Maastricht University Medical Center

Completed

Fuchs' Torsional Phaco Study

Fuchs' Endothelial Dystrophy | Phacoemulsification

Netherlands
National Eye Institute (NEI)

Completed

Studies in Families With Corneal Dystrophy or Other Inherited Corneal Diseases

Corneal Dystrophies, Hereditary | Corneal Disease

United States, China, Italy
Hospices Civils de Lyon

Completed

Determination of Circulating Autotaxin in Patients With GNAS or PTH Abnormalities (GNAS-AUTAX)

Hyperparathyroidism | Hypoparathyroidism | Fibrous Dysplasia | Adult Children | Pseudo Hypoparathyroidism | Albright Syndrome

France
Boston Children's Hospital
CureAP4 Foundation

Recruiting

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia (HSP)

Hereditary Spastic Paraplegia | SPG50 | SPG47 | SPG51 | SPG52 | AP4-related Hereditary Spastic Paraplegia | Early Onset Hereditary Spastic Paraplegia | SPG4 | SPG3A | SPG15 | SPG11

United States
Shanghai Vitalgen BioPharma Co., Ltd.

Not yet recruiting

Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

Bietti Crystalline Dystrophy

China
Beijing Tongren Hospital

Recruiting

Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

Bietti Crystalline Dystrophy

China
Octapharma

Recruiting

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema

Romania
Hospital Universitari Vall d'Hebron Research Institute
Hospital Universitario La Paz

Not yet recruiting

Identification and Characterization of Genetic Variants in Hereditary Angioedema (GENOMAEH_01)

Hereditary Angioedema With C1 Esterase Inhibitor Deficiency

Spain
First Affiliated Hospital of Fujian Medical University

Recruiting

PCSK9 Inhibitor Treatment for Patients With SPG5

Hereditary Spastic Paraplegia Type 5

China
CSL Behring

Completed

Postmarketing Immunogenicity Study in HAE Subjects Treated With Berinert

Hereditary Angioedema Types I and II

Bulgaria, Hungary, Poland, Romania
Santhera Pharmaceuticals

Completed

Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy (RHODOS)

Leber's Hereditary Optic Neuropathy

Canada, Germany, United Kingdom
CSL Behring

Completed

A Study to Evaluate the Safety and Pharmacokinetics of Two Formulations of C1-esterase Inhibitor

Hereditary Angioedema Types I and II

Germany
dr. Laura C. G. de Graaff-Herder

Recruiting

GROWing Up With Rare GENEtic Syndromes (GROW UR GENES)

Disorders of Sex Development | Congenital Adrenal Hyperplasia | Tuberous Sclerosis | Kallmann Syndrome | Prader-Willi Syndrome | Neurofibromatosis | Rett Syndrome | 22q11 Deletion Syndrome | Turner Syndrome | Noonan Syndrome | Allan-Herndon-Dudley Syndrome | Saethre-Chotzen Syndrome | Congenital Hypopituitarism | Cornelia... and other conditions

Netherlands
Medical University of Vienna

Completed

Assessment of Corneal Graft Attachment in Patients With Fuchs Endothelial Corneal Dystrophy Following DMEK Using Ultra-high Resolution OCT

Fuchs' Endothelial Dystrophy

Austria
Centre Hospitalier Régional Metz-Thionville

Completed

Predictive Factors of Good Results After Primary Descemet's Membrane Endothelial Keratoplasty (DMEK) (GOODRMEK)

Endothelial Corneal Dystrophy
Eye Hospital Pristina Kosovo

Enrolling by invitation

Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery

Macular Corneal Dystrophy

Kosovo
Cornea Research Foundation of America

Completed

Trial of Netarsudil for Prevention of Corticosteroid-induced Intraocular Pressure Elevation

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

United States
Glostrup University Hospital, Copenhagen

Unknown

Air Versus SF6 for Descemet's Membrane Endothelial Keratoplasty (DMEK)

Fuchs' Endothelial Dystrophy | Bullous Keratopathy

Denmark
University Hospital, Grenoble

Recruiting

Status of Dental Care Practices in Patients With Hereditary Angioedema (PRADA)

Angio Edema Hereditary

France
Takeda

Recruiting

A Survey of Icatibant in Pediatric Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan
Shire
Takeda Development Center Americas, Inc.

Active, not recruiting

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II (ENABLE)

Hereditary Angioedema (HAE)

Germany, Switzerland, Spain, Israel, Italy, Austria, Portugal, Kuwait
Shire
Takeda

Completed

FIRAZYR General Drug Use-Results Survey (Japan)

Hereditary Angioedema (HAE)

Japan
Takeda

Completed

A Study of Lanadelumab (SHP643) in Chinese Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

China
Hôpital Necker-Enfants Malades
European Georges Pompidou Hospital; CLAIROP

Completed

Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy (Béfinohl)

Safety Issues | Efficacy, Self

France
BioCryst Pharmaceuticals

Completed

Study to Evaluate the Efficacy and Safety of BCX7353 as an Oral Treatment for the Prevention of HAE Attacks in Japan (APeX-J)

Hereditary Angioedema, HAE

Japan
Shire

Completed

Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United States
Shire

Completed

Long-term Safety and Efficacy Study of DX-2930 (SHP643) to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE

Hereditary Angioedema (HAE)

United States, United Kingdom, Germany, Canada, Italy, Jordan, Puerto Rico
Shire

Completed

Study to Evaluate the Clinical Efficacy and Safety of Subcutaneously Administered C1 Esterase Inhibitor for the Prevention of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

Hereditary Angioedema (HAE)

Spain, Israel, United States, Hungary, Germany, Canada, Romania
Shire

Completed

CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

Hereditary Angioedema (HAE)

United States, Germany, Hungary
Shire

Completed

Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema Attacks

Hereditary Angioedema (HAE)

United States, Germany, Spain, Sweden
Shire

Completed

Open-label, Single-arm Study to Assess the Pharmacokinetics, Safety, and Tolerability of a Single Subcutaneous Dose of Icatibant in Healthy Japanese Volunteers

Hereditary Angioedema (HAE)

United States
Shire

Completed

Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary Angioedema

Hereditary Angioedema (HAE)

Israel, Germany, Mexico, United States, Romania
Shire

Completed

A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants

Hereditary Angioedema (HAE)

Japan
Shire

Completed

A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants

Hereditary Angioedema (HAE)

United States, Jordan, Italy
BioCryst Pharmaceuticals

Completed

Efficacy and Safety of BCX7353 to Prevent Angioedema Attacks in Subjects With Hereditary Angioedema (APeX-1)

Hereditary Angioedema (HAE)

Canada, United Kingdom, Germany, Spain, Australia, Hungary, Italy, Austria, North Macedonia, Denmark, Switzerland
Edison Pharmaceuticals Inc

No longer available

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

Leber's Hereditary Optic Neuropathy

United States
Takeda

Not yet recruiting

A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China

Hereditary Angioedema (HAE)
CSL Behring

Recruiting

CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema

Hereditary Angioedema (HAE)

United States, Israel
Takeda

Recruiting

A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)

Hereditary Angioedema (HAE)

United Kingdom
Takeda

Recruiting

A Survey of Lanadelumab in Participants With Hereditary Angioedema

Hereditary Angioedema (HAE)

Japan

Clinical Trials on Albright Hereditaire Osteodystrofie

Sponsors and Collaborators

Medical Conditions

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Rare Diseases

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