120. Hereditary dystonia
[
15 clinical trials,
17 drugs（DrugBank:
6 drugs),
2 target genes / 2 target pathways ]
Searched query = "Hereditary dystonia", "DYT1 dystonia", "DYT2 dystonia", "DYT3 dystonia", "X linked dystonia parkinsonism", "Lubag", "DYT4 dystonia", "DYT5 dystonia", ... show all
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03726996 | January 30, 2019 | 4 November 2019 | Desipramine in Infantile Neuroaxonal Dystrophy (INAD). | Novel Off-label Use of Desipramine in Infantile Neuroaxonal Dystrophy: Targeting the Sphingolipid Metabolism Pathway to Reduce Accumulation of Ceramide. | Infantile Neuroaxonal Dystrophy | Drug: Desipramine | Duke University | Not recruiting | 3 Years | 17 Years | All | 4 | Phase 4 | United States | |
| 2 | NCT03570931 | November 5, 2018 | 22 July 2019 | A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy | A Prospective Open-label Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy | Infantile Neuroaxonal Dystrophy | Drug: RT001 | Retrotope, Inc. | Not recruiting | 18 Months | 10 Years | All | 19 | Phase 2/Phase 3 | United States | |
| 3 | ChiCTR1900021076 | 2018-09-04 | 28 January 2019 | A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration | A study for efficacy of pantethine in the treatment of pantothenate kinase-associated neurodegeneration | pantothenate kinase-associated neurodegeneration | Experimental group:Standardized basal treatment + pantethine;Control group:Standardized basal treatment; | Department of Pediatrics, Peking University First Hospital | Recruiting | 0 | 16 | Both | Experimental group:12;Control group:6; | Other | China | |
| 4 | EUCTR2016-001955-29-ES | 16/10/2017 | 23 October 2017 | Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN) | Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension | Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease. MedDRA version: 19.1 Level: PT Classification code 10053643 Term: Neurodegenerative disorder System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Fosmetpantotenate Product Code: RE-024 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: Fosmetpantotenate CAS Number: 1858268-66-2 Current Sponsor code: RE-024 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 75- Pharmaceutical form of the placebo: Powder for oral suspension Route of administration of the placebo: Oral use | Retrophin, Inc. | Authorised | Female: yes Male: yes | 82 | Phase 3 | France;United States;Czech Republic;Canada;Spain;Norway;Germany;Italy;United Kingdom | |||
| 5 | NCT03041116 | July 17, 2017 | 14 January 2019 | Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients | Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Patients: A Randomized, Double Blind, Placebo Controlled Study With an Open Label Extension | Pantothenate Kinase-Associated Neurodegeneration | Drug: Placebo;Drug: fosmetpantotenate (RE-024) | Retrophin, Inc. | Not recruiting | 6 Years | 65 Years | All | 82 | Phase 3 | United States;Canada;Czechia;France;Germany;Italy;Norway;Poland;Spain | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03019458 | February 10, 2017 | 16 December 2017 | MINGO Supplemental Trial in X-linked Dystonia-Parkinsonism Patients | MINGO Supplemental Trial in X-linked Dystonia Parkinsonism Patients: A Prospective Randomized, Open-labeled, Parallel Group Trial | X-Linked Dystonia Parkinsonism | Dietary Supplement: MINGO | Sunshine Care Foundation | Jose R. Reyes Memorial Medical Center;Massachusetts General Hospital | Not recruiting | 18 Years | 90 Years | All | 50 | N/A | Philippines |
| 7 | EUCTR2014-001427-79-GB | 30/12/2015 | 28 May 2018 | An international open label trial of deferiprone in patients with iron storage brain disorders - an extension to TIRCON2012V1 (a randomised controlled trial of deferiprone in patients with iron storage brain disorders). | Long-term Safety and Efficacy Study of Deferiprone in Patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN) - TIRCON2012V1-EXT | Pantothenate Kinase-Associated Neurodegeneration (PKAN) MedDRA version: 18.1 Level: PT Classification code 10053643 Term: Neurodegenerative disorder System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ferriprox Product Name: Deferiprone 80 mg/mL oral solution Pharmaceutical Form: Oral solution INN or Proposed INN: Deferiprone CAS Number: 30652-11-0 Other descriptive name: Deferiprone Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 80- | ApoPharma Inc | Not Recruiting | Female: yes Male: yes | 89 | Phase 3 | United States;Germany;Italy;United Kingdom | |||
| 8 | NCT02408354 | March 2015 | 16 December 2017 | Pilot Study, Comparative, Single-center, Randomized, Crossover, Double-blind, Against Placebo, Testing the Effectiveness of Triheptanoin Oil in Alternating Hemiplegia of Childhood | Etude Pilote, Comparative, Monocentrique, randomisée, en Cross Over, en Double Aveugle, Contre Placebo, Testant l'efficacité de l'Huile triheptanoïne Dans Les Hémiplégies Alternantes de l'Enfant HEMIHEP | Alternating Hemiplegia of Childhood | Drug: Triheptanoin;Drug: Placebo | Institut National de la Santé Et de la Recherche Médicale, France | Not recruiting | 15 Years | N/A | All | 10 | Phase 2 | France | |
| 9 | EUCTR2012-000845-11-GB | 24/10/2013 | 30 April 2019 | An international trial of deferiprone in patients with iron storage brain disorders | A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCON | Pantothenate Kinase-Associated Neurodegeneration (PKAN) MedDRA version: 16.0 Level: PT Classification code 10053643 Term: Neurodegenerative disorder System Organ Class: 10029205 - Nervous system disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Ferriprox Product Name: Deferiprone 80 mg/mL oral solution (Ferriprox) Pharmaceutical Form: Oral solution INN or Proposed INN: Deferiprone CAS Number: 30652-11-0 Current Sponsor code: V03AC02 Other descriptive name: Deferiprone Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 80- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use | ApoPharma Inc | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Poland;Germany;Italy;United Kingdom | |||
| 10 | NCT01741532 | December 13, 2012 | 15 July 2019 | Efficacy and Safety Study of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN) | A Randomized, Double-blind, Placebo-controlled Trial of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN) | Pantothenate Kinase-Associated Neurodegeneration | Drug: Deferiprone oral solution;Drug: Placebo | ApoPharma | Food and Drug Administration (FDA) | Not recruiting | 4 Years | N/A | All | 89 | Phase 3 | United States;Germany;Italy;United Kingdom;Poland |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2012-000845-11-DE | 16/07/2012 | 21 November 2016 | An international trial of deferiprone in patients with iron storage brain disorders | A randomized, double-blind, placebo-controlled trial of deferiprone in patients with pantothenate kinase-associated neurodegeneration (PKAN) - TIRCON | Pantothenate kinase-associated neurodegeneration (PKAN);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Deferiprone 80 mg/mL oral solution Pharmaceutical Form: Oral solution INN or Proposed INN: Deferiprone CAS Number: 30652-11-0 Current Sponsor code: V03AC02 Other descriptive name: Deferiprone Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 80- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use | ApoPharma Inc. | Not Recruiting | Female: yes Male: yes | 90 | Phase 3 | United States;Poland;Germany;United Kingdom;Italy | |||
| 12 | NCT03333200 | January 11, 2012 | 11 November 2019 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | Recruiting | N/A | N/A | All | 1500 | Phase 1 | United States | |
| 13 | NCT02635841 | October 2009 | 16 December 2017 | Compassionate Use of Deferiprone in Patients With PKAN | The Compassionate Use of Deferiprone in Patients With Pantothenate Kinase-Associated Neurodegeneration | Pantothenate Kinase-Associated Neurodegeneration | Drug: Deferiprone | ApoPharma | Not recruiting | 7 Years | N/A | All | N/A | |||
| 14 | NCT00931164 | August 2009 | 19 October 2017 | Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO) | Single-center Phase I/II Trial of Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO Trial) | Alternating Hemiplegia of Childhood | Drug: Sodium Oxybate | University of Utah | Alternating Hemiplegia of Childhood Foundation;Jazz Pharmaceuticals | Not recruiting | 6 Months | 25 Years | All | 6 | Phase 1/Phase 2 | United States |
| 15 | EUCTR2008-003059-56-IT | 29/07/2008 | 19 March 2012 | Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND | Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ? ICAND ? Trial - ND | PKAN MedDRA version: 9.1 Level: HLT Classification code 10042259 Term: Structural brain disorders NEC | Trade Name: FERRIPROX*100CPR RIV 500MG Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Deferiprone Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 25- | ISTITUTO NEUROLOGICO CARLO BESTA | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy |