Clinical Trials on Hypophosphatemic Rickets, X-Linked Dominant in Germany

Total 166 results

Wuerzburg University Hospital
Kyowa Kirin, Inc.

Active, not recruiting

Burden of Disease and Functional Impairment in XLH (IdeFIX)

X Linked Hypophosphatemia

Germany
Kyowa Kirin Pharmaceutical Development Ltd

Active, not recruiting

Registry for Patients With X-Linked Hypophosphatemia (XLH Registry)

X-Linked Hypophosphatemia

Spain, Italy, United Kingdom, France, Belgium, Denmark, Germany, Hungary, Portugal, Norway, Switzerland, Israel, Netherlands, Czechia, Bulgaria, Sweden, Slovenia, Ireland, Latvia, Slovakia
Wuerzburg University Hospital
Kyowa Kirin, Inc.

Completed

Anti-FGF23 (Burosumab) in Adult Patients With XLH (BurGER)

X-linked Hypophosphatemia

Germany
University of Rostock

Unknown

Effects of GH on Body Proportions and Final Height in X-Linked Hypophosphatemic Rickets

Growth Disorders | Hypophosphatemic Rickets | Somatropin Therapy

Germany
Inozyme Pharma

Recruiting

Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

Generalized Arterial Calcification of Infancy | Autosomal Recessive Hypophosphatemic Rickets | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency

United States, United Kingdom, Canada, France, Germany
Westfälische Wilhelms-Universität Münster
ICON plc

Unknown

Natural History of GACI With or Without ARHR2 or PXE

Pseudoxanthoma Elasticum | Generalized Arterial Calcification in Infancy | Autosomal Recessive Hypophosphatemic Rickets

Germany
University Hospital Erlangen
Edimer Pharmaceuticals

Completed

Investigation of Chronic Inflammatory Processes in Male Individuals With Hypohidrotic Ectodermal Dysplasia

X-linked Hypohidrotic Ectodermal Dysplasia

Germany
Novartis Pharmaceuticals

Terminated

EXPLAIN -FragilE X Registry: An exPlorative Longitudinal Study for chAracterIzation, Treatment Pathways and patieNt-related Outcomes (EXPLAIN)

Fragile-X Syndrome

Germany
Astellas Gene Therapies

Completed

A Clinical Assessment Study in X-Linked Myotubular Myopathy (XLMTM) Subjects (INCEPTUS)

X-linked Myotubular Myopathy

United States, France, United Kingdom, Canada, Germany
Hubert Serve, Prof., MD

Unknown

Gene Therapy for X-CGD

X-linked Chronic Granulomatous Disease

Germany
Edimer Pharmaceuticals

Active, not recruiting

Extension Study of XLHED-Affected Male Subjects Treated With EDI200 in Protocol ECP-002 (ECP-002e)

X-linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
EspeRare Foundation
Iqvia Pty Ltd; Pierre Fabre Medicament

Recruiting

Intraamniotic Administrations of ER004 to Male Subjects With X-linked Hypohidrotic Ectodermal Dysplasia (EDELIFE)

X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

United States, Spain, France, Germany, Italy, United Kingdom
Viking Therapeutics, Inc.

Recruiting

A Study to Assess the Pharmacodynamics of VK0214 in Male Subjects With AMN

Adrenomyeloneuropathy Form (AMN) of X-linked Adrenoleukodystrophy (X-ALD)

United States, France, Germany, Italy, United Kingdom
Leipzig University Medical Center

Recruiting

Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD_QoL)

X-linked Adrenoleukodystrophy

Germany
Leipzig University Medical Center
European Leukodystrophy Association

Completed

SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)

X-linked Adrenoleukodystrophy

Germany
University Hospital Erlangen
Pervormance GmbH

Completed

Short Term Effects and Risks of Physical Exercise in Subjects With Hypohidrotic Ectodermal Dysplasia

X-Linked Hypohidrotic Ectodermal Dysplasia | Exercise-induced Rise of Body Temperature

Germany
STZ eyetrial
University Hospital Tuebingen

Completed

THOR - Tübingen Choroideremia Gene Therapy Trial (THOR)

Choroideremia

Germany
NightstaRx Ltd, a Biogen Company

Enrolling by invitation

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

Choroideremia | X-Linked Retinitis Pigmentosa

United States, United Kingdom, Netherlands, Germany, France, Brazil, Canada, Denmark, Finland
Novartis Pharmaceuticals

Terminated

Long-term, Safety, Tolerability and Efficacy Study of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

Germany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
bluebird bio

Terminated

Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
Astellas Gene Therapies

Active, not recruiting

Gene Transfer Clinical Study in X-Linked Myotubular Myopathy (ASPIRO)

X-Linked Myotubular Myopathy

United States, Canada, France, Germany
Novartis Pharmaceuticals

Terminated

Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, Denmark, Italy, Netherlands, Spain, Sweden, United Kingdom, France
Edimer Pharmaceuticals

Completed

Phase 2 Study to Evaluate Safety, Pharmacokinetics, Immunogenicity and Pharmacodynamics/Efficacy of EDI200 in Male Infants With X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) (ECP-002)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
bluebird bio

Active, not recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

Cerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)

United States, Argentina, Australia, Brazil, France, Germany, Netherlands, United Kingdom, Italy
Rocket Pharmaceuticals Inc.

Recruiting

Danon Disease Natural History Study

Danon Disease

United States, Germany
Biogen

Completed

A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111 (GEMINI)

Choroideremia

United States, Germany, France
Edimer Pharmaceuticals

Completed

Natural History and Outcomes in X-Linked Hypohidrotic Ectodermal Dysplasia (ECP-015)

X-Linked Hypohidrotic Ectodermal Dysplasia

United States, United Kingdom, France, Germany, Italy
Novartis Pharmaceuticals

Completed

Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Fragile X Syndrome

United States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
University Hospital Heidelberg

Completed

Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III

Mucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Coping Behavior | Mucopolysaccharidosis Type III | Behavior Disorders

Germany
Novartis Pharmaceuticals

Completed

Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy

Spinal and Bulbar Muscular Atrophy

United States, Germany, Italy, Denmark
ArmaGen, Inc

Completed

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome

Mucopolysaccharidosis II

United States, Germany, Philippines
University Hospital Erlangen
Edimer Pharmaceuticals

Completed

Characterization of Sweat Gland Function in Patients With Recessively Inherited Hypohidrotic Ectodermal Dysplasia

Hypohidrotic Ectodermal Dysplasia

Germany
Charite University, Berlin, Germany

Completed

Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy (SUNIMUD)

Duchenne Muscular Dystrophy

Germany
Takeda
Takeda Development Center Americas, Inc.

Active, not recruiting

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Hunter Syndrome

United States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
University of Erlangen-Nürnberg Medical School

Completed

Non-invasive Imaging of Muscle Structure in Duchenne Muscular Dystrophy Using Multispectral Optoacoustic Tomography (MSOT_DMD)

Muscular Dystrophies | Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne

Germany
MedDay Pharmaceuticals SA

Completed

MD1003-AMN MD1003 in Adrenomyeloneuropathy

Adrenoleukodystrophy | Adrenomyeloneuropathy | AMN

France, Germany, Spain
Goethe University
Society for Thrombosis and Haemostasis Research (Germany)

Recruiting

German Pediatric Hemophilia Research Database (GEPHARD)

Hemophilia A | Hemophilia B | Children | Drug Specific Antibodies

Germany
Biogen

Completed

Efficacy and Safety of BIIB111 for the Treatment of Choroideremia (STAR)

Choroideremia

United States, Finland, France, Denmark, Netherlands, Germany, United Kingdom, Canada
Shire

Completed

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Mucopolysaccharidosis II

United States, United Kingdom, Brazil, Germany
Hoffmann-La Roche
Sarepta Therapeutics, Inc.

Recruiting

A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) (ENVOL)

Duchenne Muscular Dystrophy

Spain, Germany
CSL Behring

Active, not recruiting

Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Hemophilia B

Netherlands, Germany
CSL Behring

Completed

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

Hemophilia B

Germany, Denmark, Netherlands
Johann Wolfgang Goethe University Hospital
German Federal Ministry of Education and Research

Unknown

Gene Therapy for Chronic Granulomatous Disease (CGD)

Granulomatous Disease, Chronic

Germany
AB2 Bio Ltd.

Completed

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency

NLRC4-MAS | XIAP Deficiency

United States, Canada, Germany
JCR Pharmaceuticals Co., Ltd.

Recruiting

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Mucopolysaccharidosis II

United States, Germany, Spain, France, Brazil, Argentina, Turkey, United Kingdom, Poland, Italy
NightstaRx Ltd, a Biogen Company

Completed

Natural History of the Progression of Choroideremia Study (NIGHT)

Choroideremia

Finland, United States, Canada, Denmark, Netherlands, Germany, United Kingdom, Brazil, France
AB2 Bio Ltd.

Active, not recruiting

Therapeutic Use of Tadekinig Alfa in NLRC4 Mutation and XIAP Deficiency as Open Label Extension

NLRC4-MAS | XIAP Deficiency

United States, Canada, Germany
Minoryx Therapeutics, S.L.

Active, not recruiting

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT) (NEXUS)

Cerebral Adrenoleukodystrophy

Spain, Germany, France, Argentina
bluebird bio

Completed

A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

Cerebral Adrenoleukodystrophy (CALD)

United States, France, Germany, Italy, Netherlands, United Kingdom

Clinical Trials on Hypophosphatemic Rickets, X-Linked Dominant in Germany

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