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National clinical trial(s)
AUSTRALIA
Victoria
ADDRESS: NOT PROVIDED - AU
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) -AU
Institution: Information not provided - AU
GERMANY
Berlin
ADDRESS: NOT PROVIDED - DE
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) -DE
Institution: Information not provided - DE
UNITED KINGDOM
Greater London
ADDRESS: NOT PROVIDED - GB
A Phase Ia/Ib, Multiple-site Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of KL1333 After a Single and Multiple Ascending Oral Doses in Healthy Subjects and Patients With Primary Mitochondrial Disease-GB
Institution: Information not provided - GB
UNITED KINGDOM
Greater London
ADDRESS: NOT PROVIDED - GB
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) -GB
Institution: Information not provided - GB
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
An Open-label Long-Term Safety and Efficacy Extension Study in Subjects with Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Previously Enrolled in UX007 or Triheptanoin Studies
Institution: Information not provided - US
FRANCE
HAUTS-DE-FRANCE
AMIENS
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - FR
CHU Amiens-Picardie - Site Sud
FRANCE
ILE-DE-FRANCE
CRÉTEIL
A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency - FR
Hôpitaux Universitaires Henri Mondor
Unité des Maladies Génétiques du Globule Rouge
FRANCE
ILE-DE-FRANCE
CRÉTEIL
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - FR
Hôpitaux Universitaires Henri Mondor
CHU Henri Mondor
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - FR
CHU de Marseille - Hôpital de la Timone
GERMANY
Bayern
WÜRZBURG
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies -DE-
Universitätsklinikum Würzburg
IRELAND
County Dublin
DUBLIN
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - IE
St James's Hospital
Department of Haematology
IRELAND
County Dublin
DUBLIN
An Open-Label Study To Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency - IE
St James's Hospital
Department of Haematology
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - IT
Institution: Information not provided - IT
JAPAN
JAPAN
ADDRESS : NOT PROVIDED - JP
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - JP
Institution: Information not provided - JP
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies -ES
Institution: Information not provided - ES
SPAIN
Madrid
MADRID
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD -ES
Hospital Universitario Fundación Jiménez Díaz
Servicio de Hematología y Hemoterapia
SWITZERLAND
Suisse Romande
ADDRESS: NOT PROVIDED - CH
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies - CH
Institution: Information not provided - CH
UNITED KINGDOM
Cambridgeshire
CAMBRIDGE
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies -GB
Addenbrooke's Hospital
Addenbrookes Hospital
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Phase 3 Trial of Dichloroacetate in Pyruvate Dehydrogenase Complex Deficiency: (DCA/PDCD)
Institution: Information not provided - US
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) - IT
Institution: Information not provided - IT
SPAIN
Madrid
ADDRESS: NOT PROVIDED - ES
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD) -ES
Institution: Information not provided - ES
AUSTRIA
SALZBURG
SALZBURG
MitoFibrate CT1: Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies (phase II) - AT
Universitätsklinikum der Paracelsus Medizinischen Privatuniversität
Universitätsklinik für Kinder- und Jugendheilkunde
AUSTRIA
STEIERMARK
GRAZ
OPA1: Raxone® treatment for patients with dominant optic atrophy due to OPA1 gene mutation
Medizinische Universität Graz
Universitäts-Augenklinik
ITALY
LAZIO
ROMA
Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease (Phase II) - IT
IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
U.O.C. Malattie Neuromuscolari e Neurodegenerative - Laboratorio di Medicina Molecolare
AUSTRIA
WIEN
ADDRESS: NOT PROVIDED - AT
LEROS: External Natural History Controlled, Open-Label Intervention Study to Assess the Efficacy and Safety of Long-Term Treatment With Raxone® in Leber's Hereditary Optic Neuropathy (LHON) (Phase 4) - AT
Institution: Information not provided - AT
FRANCE
ILE-DE-FRANCE
PARIS
RESCUE: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene (Phase III) - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
REVERSE: Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene (Phase III) - FR
Centre hospitalier national d'ophtalmologie des Quinze-Vingts
Centre de Référence REFERET
FRANCE
ILE-DE-FRANCE
PARIS
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene (Phase I-II)
Institut de la Vision
Centre d'Investigation Clinique (CIC) de l'hôpital des Quinze-Vingts
FRANCE
ILE-DE-FRANCE
PARIS
AOA1 : Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10 (Phase III)
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127
GERMANY
Bayern
MÜNCHEN
RESCUE: A Randomized, Double-Masked, Sham-Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for 6 Months or Less by LHON Due to the G11778A Mutation in the Mitochondrial ND4 Gene (Phase III) -DE-
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
GERMANY
Bayern
MÜNCHEN
RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials -DE-
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
GERMANY
Bayern
MÜNCHEN
The KHENERGYZE Study: A Phase IIb Double-blind, Randomised, Placebo-controlled, Multi-centre, Confirmative Three-way Cross-over Study on Cognitive Function With Two Doses of KH176 in Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation
Friedrich-Baur-Institut
Friedrich-Baur-Institut an der Neurologischen Klinik und Poliklinik
ITALY
LOMBARDIA
MILANO
A Phase 2 Open-label Study to Evaluate the Efficacy of Allogeneic Human Cord Blood-derived Mesenchymal Stromal Cells in Maintaining Remission After Immunosuppressive Therapy Withdrawal in Pediatric Patients With Steroid-dependent Nephrotic Syndrome
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
U.O.C. di Nefrologia, Dialisi e Trapianto Pediatrico
JAPAN
JAPAN
ADDRESS : NOT PROVIDED - JP
Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders?
Institution: Information not provided - JP
SPAIN
Madrid
MADRID
REFLECT: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for Up to One Year -ES
Hospital Universitario Ramón y Cajal
Servicio de Oftalmología
UNITED KINGDOM
Greater London
LONDON
RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials-GB
Moorfield's Eye Hospital
UNITED KINGDOM
Greater London
LONDON
TEETPIM:Trial of Erythrocyte Encapsulated Thymidine Phosphorylase In Mitochondrial Neurogastrointestinal Encephalomyopathy-GB
St George's University of London
Molecular and Clinical Sciences Reseach Institute
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
An Open-label Dose Escalation Study of an Adeno-associated Virus Vector (scAAV2-P1ND4v2) for Gene Therapy of Leber's Hereditary Optic Neuropathy (LHON) Caused by the G11778A Mutation in Mitochondrial DNA
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
MNGIE (Mitochondrial Neurogastrointestinal Encephalomyopathy) AHSCT (Allogeneic Hematopoietic Stem Cell Transplant) Safety Study
Institution: Information not provided - US
SPAIN
Madrid
MADRID
A Phase 3b Single Arm Clinical Study to Evaluate the Efficacy and Safety of MT1621 in Nucleos(t)Ide Treatment Naïve Pediatric and Adolescent Subjects With Thymidine Kinase 2 (TK2) Deficiency - ES
Hospital Universitario 12 de Octubre
Servicio de Neurología-Neurofisiología
SPAIN
Madrid
MADRID
A Phase 2 Open-Label Study of Continuation Treatment With Combination Pyrimidine Nucleosides in Patients With Thymidine Kinase 2 Deficiency (TK2)
Hospital Universitario 12 de Octubre
Unidad multidisciplinar de enfermedades neuromusculares y ELA
Multinational clinical trial(s)
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
NuPower Study: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects With Primary Mitochondrial Disease Resulting From Pathogenic Nuclear DNA Mutations (nPMD).
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies
Institution: Information not provided - US
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
RESCUE/REVERSE: Long-term Follow-up of ND4 LHON Subjects Treated With GS010 Ocular Gene Therapy in the RESCUE or REVERSE Phase III Clinical Trials
Institution: Information not provided - FR
GERMANY
Baden-Württemberg
LÖRRACH
A double-blind, randomised, placebo-controlled study of the efficacy, safety and tolerability of idebenone in the treatment of patients with Leber's Hereditary Optic Neuropathy (LHON) (Phase II) (coordination)
Santhera Pharmaceuticals (Deutschland) GmbH
NETHERLANDS
Gelderland
NIJMEGEN