Clinical Trials on X Linked Erythropoietic Protoporphyria

Total 1545 results

Mitsubishi Tanabe Pharma America Inc.

Recruiting

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP (INSPIRE)

Erythropoietic Protoporphyria (EPP) | X-Linked Protoporphyria (XLP)

United States, Spain, France, United Kingdom, Japan, Italy, Australia, Bulgaria, Czechia, Netherlands, Poland
Icahn School of Medicine at Mount Sinai
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); Rare... and other collaborators

Completed

Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact

Erythropoietic Protoporphyria | EPP | XLP | X-Linked Protoporphyria | XLPP | X-Linked Dominant Erythropoietic Protoporphyria | XLEPP | XLDP

United States
University of Utah
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); University... and other collaborators

Terminated

Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic Protoporphyria (INHEPP)

Erythropoietic Protoporphyria (EPP) | X Linked Erythropoietic Protoporphyria

United States
Amy K. Dickey, M.D.
Wake Forest University Health Sciences; University of Texas

Recruiting

Effect of Oral Cimetidine in the Protoporphyrias

Erythropoietic Protoporphyria | X-linked Protoporphyria

United States
Icahn School of Medicine at Mount Sinai
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Completed

Oral Iron for Erythropoietic Protoporphyrias (EPP)

Erythropoietic Protoporphyria | EPP | X-linked Protoporphyria | XLP

United States
Mitsubishi Tanabe Pharma America Inc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria (EPP)

United States
University of Alabama at Birmingham
Porphyria Rare Disease Clinical Research Consortium

Completed

Mitoferrin-1 Expression in Erythropoietic Protoporphyria (Porphyria Rare Disease Clinical Research Consortium (RDCRC))

Erythropoietic Protoporphyria (EPP)

United States
Brigham and Women's Hospital

Completed

Sorbent Therapy of the Cutaneous Porphyrias (EPP)

Erythropoietic Protoporphyria

United States
Bispebjerg Hospital

Recruiting

Protection Against Visible Light by Dihydroxyacetone in Erythropoietic Protoporphyria

Erythropoietic Protoporphyria

Denmark
Clinuvel Pharmaceuticals Limited

Completed

Phase III Confirmatory Study in Erythropoietic Protoporphyria

Erythropoietic Protoporphyria

United States
Bispebjerg Hospital

Unknown

Does Exercise and Heat Increase the Lightsensibility in Patients With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria

Denmark
Disc Medicine, Inc

Enrolling by invitation

Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

Erythropoietic Protoporphyria

United States, Australia
Clinuvel Pharmaceuticals Limited

Completed

Multicentre Phase III Erythropoietic Protoporphyria Study

Erythropoietic Protoporphyria
Bispebjerg Hospital

Completed

Light Exposure Patterns and Symptoms Among Patients With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria

Denmark
Disc Medicine, Inc

Active, not recruiting

Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

Erythropoietic Protoporphyria

United States
Clinuvel Pharmaceuticals Limited

Completed

Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Erythropoietic Protoporphyria

Netherlands, Finland, France, Germany, Ireland, United Kingdom
Clinuvel Pharmaceuticals Limited

Completed

A Safety Extension Study in Patients With Erythropoietic Protoporphyria (EPP)

Erythropoietic Protoporphyria
Clinuvel Pharmaceuticals Limited

Completed

Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)

Erythropoietic Protoporphyria

United States
Mitsubishi Tanabe Pharma America Inc.

Active, not recruiting

Extension Study to Evaluate Safety and Tolerability of Oral Dersimelagon (MT-7117) in Subjects With Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP)

EPP | XLP

United States, Germany, Japan, Spain, Italy, Sweden, Australia, Canada, Norway, United Kingdom
Mitsubishi Tanabe Pharma America Inc.

Completed

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic Protoporphyria or X-Linked Protoporphyria

EPP | XLP

Spain, United States, Germany, Norway, Italy, Australia, Canada, Japan, Sweden, United Kingdom
FDA Office of Orphan Products Development
St. Luke's-Roosevelt Hospital Center

Completed

Study of Cysteine Hydrochloride for Erythropoietic Protoporphyria

Erythropoietic Protoporphyria
Brigham and Women's Hospital

Completed

Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria

Erythropoietic Protoporphyria
Bispebjerg Hospital
Alfred Benzon Foundation

Completed

Iron Therapy in Erythropoietic Protoporphyria

Iron-deficiency | Erythropoietic Protoporphyria

Denmark
National Eye Institute (NEI)

Active, not recruiting

Study of RS1 Ocular Gene Transfer for X-linked Retinoschisis

Retinoschisis | X-Linked

United States
National Eye Institute (NEI)

Completed

Mothers Experiences With X-linked Retinoschisis Compared to Fathers Experiences

Retinoschisis | X-Linked

United States
West China Hospital

Recruiting

Safety and Efficacy of a Single Subretinal Injection of JWK002 Gene Therapy in Subjects With X-linked Retinoschisis(XLRS)

X Linked Retinoschisis

China
University of Miami
Jackson Health System

No longer available

cliniMACs HUD for T Cell Depletion

x Linked Combined Immunodeficiency

United States
Newcastle-upon-Tyne Hospitals NHS Trust

Recruiting

Myotubular and Centronuclear Myopathy Patient Registry

X-linked Myotubular Myopathy | Centronuclear Myopathy | Myotubular Myopathy | Myotubular Myopathy 1 | Myotubular (Centronuclear) Myopathy | Centronuclear Myopathy, X-Linked

United Kingdom
Retina Foundation of the Southwest
DSM Nutritional Products, Inc.; Foundation Fighting Blindness

Completed

DHA and X-Linked Retinitis Pigmentosa

Retinitis Pigmentosa | X-linked Genetic Diseases

United States
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
University of Florida
Audentes Therapeutics

Completed

Respiratory Muscle Function in Untreated X-Linked Myotubular Myopathy (XLMTM)

X-linked Myotubular Myopathy

United States
Great Ormond Street Hospital for Children NHS Foundation...

Unknown

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

X-linked Severe Combined Immunodeficiency

United Kingdom
James Dowling
Canadian Institutes of Health Research (CIHR); Cures Within Reach; The Joshua... and other collaborators

Recruiting

Tamoxifen Therapy for Myotubular Myopathy (TAM4MTM)

X Linked Myotubular Myopathy

United Kingdom, Canada, United States
National Institute of Allergy and Infectious Diseases...

Recruiting

Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)

X-linked Severe Combined Immunodeficiency (XSCID)

United States
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI); Assisi Foundation; California...

Suspended

Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

Severe Combined Immunodeficiency Disease, X-linked

United States
David Williams

Active, not recruiting

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Gene Therapy | Severe Combined Immunodeficiency, X Linked

United States, United Kingdom
Assistance Publique - Hôpitaux de Paris

Completed

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID2)

X-linked Severe Combined Immunodeficiency

France
Shenzhen Geno-Immune Medical Institute

Unknown

Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector （TYF-IL-2Rg）

SCID, X Linked

China
Murdoch Childrens Research Institute
University of Sydney; Sydney Children's Hospitals Network; Women's and Children... and other collaborators

Active, not recruiting

Mackenzie's Mission: The Australian Reproductive Carrier Screening Project

Autosomal Recessive Disorder | X-Linked Genetic Diseases

Australia
Astellas Gene Therapies

Completed

A Medical Chart Review of Patients With X-Linked Myotubular Myopathy (XLMTM)

Males With X-linked Myotubular Myopathy (XLMTM)

United States, United Kingdom
National Eye Institute (NEI)

Completed

People s Expectations When Enrolling in a Phase I/II RS1 Ocular Gene Transfer Clinical Trial

Retinoschisis | X-Linked

United States
National Institute of Allergy and Infectious Diseases...

Terminated

Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1

Growth Failure | X-linked Severe Combined Immunodeficiency (XSCID) | Growth Hormone Resistence

United States
Edimer Pharmaceuticals

Completed

A Phase 1, Open-label, Multicenter, Safety and Pharmacokinetic Study of EDI200

X-linked Hypohidrotic Ectodermal Dysplasia

United States
Hospices Civils de Lyon

Unknown

Clinical Phenotyping and Characterization of Neural Networks and Cognitive Processes Involved in Mental Retardation X-linked (XLMR)

X-linked Mental Retardation

France
Cure CMD
University of Michigan; Congenital Muscle Disease International Registr

Completed

Myotubular Myopathy Event Study (MTMES)

X-linked Myotubular Myopathy

United States
University of California, Davis
University of Alberta; St. Justine's Hospital

Recruiting

A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X Linked

United States, Canada
University Hospital Erlangen
Edimer Pharmaceuticals

Completed

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

X-linked Hypohidrotic Ectodermal Dysplasia

Germany
Edimer Pharmaceuticals

Completed

Phenotypic Properties in Individuals Affected With XLHED (ECP-014)

X Linked Hypohidrotic Ectodermal Dysplasia

United States
National Institute of Allergy and Infectious Diseases...

Unknown

Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency (LVXSCID-OC)

X-Linked Combined Immunodeficiency Diseases

United States
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

Clinical Trials on X Linked Erythropoietic Protoporphyria

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