Adrenomyeloneuropathy News - LARVOL Sigma

Adrenomyeloneuropathy

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|||||||||| Trial completion, Trial completion date, Trial primary completion date: Newborn Screening for Adrenoleukodystrophy (clinicaltrials.gov) - Jul 12, 2018
P=N/A, N=45796, Completed, Sponsor: National Taiwan University Hospital
Recruiting --> Completed | Trial completion date: Jun 2018 --> Aug 2017 | Trial primary completion date: Nov 2017 --> Aug 2017

|||||||||| Enrollment change, Trial withdrawal: Early Diagnosis Of Childhood Cerebral ALD (clinicaltrials.gov) - Jul 6, 2018
P=N/A, N=0, Withdrawn, Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting --> Completed | Trial completion date: Jun 2018 --> Aug 2017 | Trial primary completion date: Nov 2017 --> Aug 2017 N=100 --> 0 | Not yet recruiting --> Withdrawn

|||||||||| Trial completion: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders (clinicaltrials.gov) - Feb 5, 2018
P2, N=46, Completed, Sponsor: Masonic Cancer Center, University of Minnesota
N=100 --> 0 | Not yet recruiting --> Withdrawn Active, not recruiting --> Completed

|||||||||| Trial initiation date, Trial primary completion date, IO biomarker: Early Diagnosis Of Childhood Cerebral ALD (clinicaltrials.gov) - Dec 5, 2017
P=N/A, N=100, Not yet recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
Active, not recruiting --> Completed Initiation date: Nov 2017 --> Nov 2018 | Trial primary completion date: Jul 2022 --> Jul 2023

||||||||||  Campath (alemtuzumab) / Sanofi
Enrollment change:  Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (clinicaltrials.gov) -  Dec 5, 2017
P=N/A, N=3, Completed,  Sponsor: Masonic Cancer Center, University of Minnesota
Initiation date: Nov 2017 --> Nov 2018 | Trial primary completion date: Jul 2022 --> Jul 2023 N=37 --> 3

|||||||||| Trial initiation date, IO biomarker: Early Diagnosis Of Childhood Cerebral ALD (clinicaltrials.gov) - Oct 25, 2017
P=N/A, N=100, Not yet recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
N=37 --> 3 Initiation date: Aug 2017 --> Nov 2017

||||||||||  Qizenday (biotin) / MedDay
Trial completion:  MD1003-AMN MD1003 in Adrenomyeloneuropathy (clinicaltrials.gov) -  Oct 9, 2017
P2/3, N=67, Completed,  Sponsor: MedDay Pharmaceuticals SA
Initiation date: Aug 2017 --> Nov 2017 Active, not recruiting --> Completed

|||||||||| Enrollment open: Newborn Screening for Adrenoleukodystrophy (clinicaltrials.gov) - Jan 6, 2017
P=N/A, N=60000, Recruiting, Sponsor: National Taiwan University Hospital
Active, not recruiting --> Completed Not yet recruiting --> Recruiting

||||||||||  Qizenday (biotin) / MedDay
New P2/3 trial:  MD1003-AMN MD1003 in Adrenomyeloneuropathy (clinicaltrials.gov) -  Nov 14, 2016
P2/3, N=67, Active, not recruiting,  Sponsor: MedDay Pharmaceuticals SA

|||||||||| New trial: Newborn Screening for Adrenoleukodystrophy (clinicaltrials.gov) - Nov 2, 2016
P=N/A, N=60000, Not yet recruiting, Sponsor: National Taiwan University Hospital

|||||||||| New trial, IO biomarker: Early Diagnosis Of Childhood Cerebral ALD (clinicaltrials.gov) - Oct 28, 2016
P=N/A, N=100, Not yet recruiting, Sponsor: Masonic Cancer Center, University of Minnesota

||||||||||  acetylcysteine solution / Generic mfg.
Biomarker, Enrollment change:  A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants (clinicaltrials.gov) -  Sep 19, 2016
P2, N=13, Completed,  Sponsor: Onofre, Aurora Pujol, M.D.
Not yet recruiting --> Recruiting N=20 --> 13

|||||||||| Enrollment change: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders (clinicaltrials.gov) - Jan 25, 2016
P2, N=46, Active, not recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
N=20 --> 13 N=66 --> 46

|||||||||| Enrollment change, Trial withdrawal, Metastases: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) (clinicaltrials.gov) - Jan 25, 2016
P1, N=0, Withdrawn, Sponsor: Masonic Cancer Center, University of Minnesota
N=66 --> 46 N=18 --> 0 | Not yet recruiting --> Withdrawn

|||||||||| Enrollment closed, Trial primary completion date: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders (clinicaltrials.gov) - Apr 25, 2015
P2, N=66, Active, not recruiting, Sponsor: Masonic Cancer Center, University of Minnesota
N=18 --> 0 | Not yet recruiting --> Withdrawn Recruiting --> Active, not recruiting | Trial primary completion date: Nov 2014 --> Jun 2015

||||||||||  ursodeoxycholic acid / Generic mfg.
Trial termination:  Study of Bile Acids in Patients With Peroxisomal Disorders (clinicaltrials.gov) -  Apr 19, 2015
P=N/A, N=25, Terminated,  Sponsor: Children's Hospital Research Foundation University of Cincinnati
Recruiting --> Active, not recruiting | Trial primary completion date: Nov 2014 --> Jun 2015 Not yet recruiting --> Terminated

|||||||||| New P1 trial, Metastases: MT2014-14 IT-MSC for Advanced Cerebral Adrenoleukodystrophy (cALD) (clinicaltrials.gov) - Apr 7, 2015
P1, N=18, Not yet recruiting, Sponsor: Masonic Cancer Center, University of Minnesota

|||||||||| Trial completion, Trial primary completion date: Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy (clinicaltrials.gov) - Jan 9, 2015
P=N/A, N=31, Completed, Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Not yet recruiting --> Terminated Recruiting --> Completed | Trial primary completion date: Apr 2014 --> Oct 2014

|||||||||| Trial termination: UCBT-002: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases (clinicaltrials.gov) - Jul 8, 2014
P3, N=40, Terminated, Sponsor: Aldagen
Recruiting --> Completed | Trial primary completion date: Apr 2014 --> Oct 2014 Suspended --> Terminated; Terminated - Sponsor Decision

||||||||||  Campath (alemtuzumab) / Sanofi
Trial primary completion date:  Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (clinicaltrials.gov) -  May 14, 2014
P=N/A, N=37, Completed,  Sponsor: Masonic Cancer Center, University of Minnesota
Suspended --> Terminated; Terminated - Sponsor Decision Trial primary completion date: Jun 2020 --> Nov 2013

||||||||||  Campath (alemtuzumab) / Sanofi
Trial completion date:  Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (clinicaltrials.gov) -  May 14, 2014
P=N/A, N=37, Completed,  Sponsor: Masonic Cancer Center, University of Minnesota
Trial primary completion date: Jun 2020 --> Nov 2013 Trial completion date: Jun 2020 --> Nov 2013

||||||||||  Campath (alemtuzumab) / Sanofi
Trial completion:  Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (clinicaltrials.gov) -  May 14, 2014
P=N/A, N=37, Completed,  Sponsor: Masonic Cancer Center, University of Minnesota
Trial completion date: Jun 2020 --> Nov 2013 Recruiting --> Completed

||||||||||  acetylcysteine solution / Generic mfg.
Biomarker, Trial completion:  A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants (clinicaltrials.gov) -  Mar 18, 2014
P2, N=20, Completed,  Sponsor: Onofre, Aurora Pujol, M.D.
Recruiting --> Completed Recruiting --> Completed

||||||||||  sobetirome (QRX 411) / Annovis Bio
Trial withdrawal:  Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) (clinicaltrials.gov) -  Feb 11, 2014
P1, N=0, Withdrawn,  Sponsor: Thomas S. Scanlan
Recruiting --> Completed Recruiting --> Withdrawn

||||||||||  sobetirome (QRX 411) / Annovis Bio
Enrollment change:  Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) (clinicaltrials.gov) -  Feb 11, 2014
P1, N=0, Withdrawn,  Sponsor: Thomas S. Scanlan
Recruiting --> Withdrawn N=10 --> 0

|||||||||| New P3 trial: Betaine and Peroxisome Biogenesis Disorders (clinicaltrials.gov) - Apr 22, 2013
P3, N=12, Recruiting, Sponsor: McGill University Health Center

|||||||||| Enrollment open: Betaine and Peroxisome Biogenesis Disorders (clinicaltrials.gov) - Apr 18, 2013
P3, N=12, Recruiting, Sponsor: McGill University Health Center
N=10 --> 0 Not yet recruiting --> Recruiting

||||||||||  sobetirome (QRX 411) / Annovis Bio
New P1 trial:  Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD) (clinicaltrials.gov) -  Feb 6, 2013
P1, N=0, Withdrawn,  Sponsor: Thomas S. Scanlan

||||||||||  Campath (alemtuzumab) / Sanofi
Enrollment open:  Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (clinicaltrials.gov) -  Jul 17, 2012
P=N/A, N=37, Recruiting,  Sponsor: Masonic Cancer Center, University of Minnesota
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting

|||||||||| Clinical: Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy (clinicaltrials.gov) - May 7, 2012
P=N/A, N=30, Recruiting, Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

|||||||||| Trial termination: A Phase III Trial of Lorenzo's Oil in Adrenomyeloneuropathy (clinicaltrials.gov) - Feb 29, 2012
P3, N=240, Terminated, Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Not yet recruiting --> Recruiting Recruiting --> Terminated; Upon recommendation of DSMB

||||||||||  acetylcysteine solution / Generic mfg.
Biomarker, New P2 trial:  A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants (clinicaltrials.gov) -  Dec 15, 2011
P2, N=20, Completed,  Sponsor: Onofre, Aurora Pujol, M.D.

|||||||||| Trial completion: The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD) (clinicaltrials.gov) - Aug 8, 2011
P=N/A, N=10, Completed, Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Recruiting --> Terminated; Upon recommendation of DSMB Recruiting --> Completed