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6 Companies |
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6 Products |
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6 Products |
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6 Mechanisms of Action |
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0 Trials |
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28 News |
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- | NN1213 / Novo Nordisk
Trial completion date, Trial termination, Trial primary completion date: Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - Sep 13, 2018
P=N/A, N=30, Terminated, Sponsor: Alexion Pharmaceuticals
Trial completion date: Apr 2018 --> Jul 2017 | Active, not recruiting --> Terminated | Trial primary completion date: Apr 2018 --> Jul 2017; The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103.
- | lesinidase alfa (SBC-103) / AstraZeneca
Trial completion, Trial primary completion date: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Jul 12, 2018
P1/2, N=11, Completed, Sponsor: Alexion Pharmaceuticals
Trial completion date: Apr 2018 --> Jul 2017 | Active, not recruiting --> Terminated | Trial primary completion date: Apr 2018 --> Jul 2017; The Sponsor's decision to terminate the SBC-103 program was reached after review of the data from all interventional clinical studies of SBC-103. Active, not recruiting --> Completed | Trial primary completion date: Jul 2018 --> Oct 2017
- | 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
Active, not recruiting --> Completed | Trial primary completion date: Jul 2018 --> Oct 2017 N=37 --> 3
- SAF-301 / Lysogene, Alcyone
Enrollment closed, Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Jan 18, 2017
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
N=37 --> 3 Completed --> Active, not recruiting | Trial primary completion date: May 2016 --> May 2017
- SAF-301 / Lysogene, Alcyone
Trial completion, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Sep 7, 2016
P1/2, N=4, Completed, Sponsor: LYSOGENE
Completed --> Active, not recruiting | Trial primary completion date: May 2016 --> May 2017 Active, not recruiting --> Completed
- Trial completion, MRI: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging (clinicaltrials.gov) - Jun 27, 2016
P=N/A, N=5, Completed, Sponsor: Alexion Pharmaceuticals
Active, not recruiting --> Completed Active, not recruiting --> Completed
- | Trial completion: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - Jun 27, 2016
P=N/A, N=30, Completed, Sponsor: Alexion Pharmaceuticals
Active, not recruiting --> Completed Recruiting --> Completed
- SAF-301 / Lysogene, Alcyone
Trial initiation date, Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Mar 24, 2016
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
Recruiting --> Completed Initiation date: Oct 2013 --> May 2013 | Trial primary completion date: Jun 2017 --> May 2016
- Trial completion: Natural History Studies of Mucopolysaccharidosis III (clinicaltrials.gov) - Nov 16, 2015
P=N/A, N=25, Completed, Sponsor: Nationwide Children's Hospital
Initiation date: Oct 2013 --> May 2013 | Trial primary completion date: Jun 2017 --> May 2016 Recruiting --> Completed
- Enrollment closed: Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - Oct 6, 2015
P=N/A, N=30, Active, not recruiting, Sponsor: Synageva BioPharma Corp.
Recruiting --> Completed Recruiting --> Active, not recruiting
- lesinidase alfa (SBC-103) / AstraZeneca
Enrollment closed: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Aug 11, 2015
P1/2, N=9, Active, not recruiting, Sponsor: Synageva BioPharma Corp.
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- SAF-301 / Lysogene, Alcyone
Trial primary completion date, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Apr 14, 2015
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
Recruiting --> Active, not recruiting Trial primary completion date: Jun 2014 --> Jun 2017
- Trial initiation date, Trial primary completion date: Natural History Studies of Mucopolysaccharidosis III (clinicaltrials.gov) - Feb 12, 2015
P=N/A, N=30, Recruiting, Sponsor: Nationwide Children's Hospital
Trial primary completion date: Jun 2014 --> Jun 2017 Initiation date: Aug 2013 --> Feb 2014 | Trial primary completion date: Jul 2015 --> Oct 2015
- lesinidase alfa (SBC-103) / AstraZeneca
Enrollment open: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Jan 23, 2015
P1/2, N=9, Recruiting, Sponsor: Synageva BioPharma Corp.
Initiation date: Aug 2013 --> Feb 2014 | Trial primary completion date: Jul 2015 --> Oct 2015 Not yet recruiting --> Recruiting
- lesinidase alfa (SBC-103) / AstraZeneca
New P1/2 trial: Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (clinicaltrials.gov) - Dec 25, 2014
P1/2, N=9, Not yet recruiting, Sponsor: Synageva BioPharma Corp.
- Review: Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - Nov 18, 2014
P=N/A, N=30, Recruiting, Sponsor: Synageva BioPharma Corp.
- Review: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB (clinicaltrials.gov) - Nov 18, 2014
P=N/A, N=30, Recruiting, Sponsor: Synageva BioPharma Corp.
- 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
Not yet recruiting --> Recruiting Suspended --> Terminated; Terminated - Sponsor Decision
- Enrollment closed, MRI: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging (clinicaltrials.gov) - Jun 18, 2014
P=N/A, N=6, Active, not recruiting, Sponsor: Synageva BioPharma Corp.
Suspended --> Terminated; Terminated - Sponsor Decision Recruiting --> Active, not recruiting
- 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
Recruiting --> Active, not recruiting 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
- MRI: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging (clinicaltrials.gov) - Mar 16, 2014
P=N/A, N=6, Recruiting, Sponsor: Synageva BioPharma Corp.
- SAF-301 / Lysogene, Alcyone
New P1/2 trial, Gene therapy: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy (clinicaltrials.gov) - Jan 30, 2014
P1/2, N=4, Active, not recruiting, Sponsor: LYSOGENE
- Enrollment open: Natural History Studies of Mucopolysaccharidosis III (clinicaltrials.gov) - Jan 14, 2014
P=N/A, N=30, Recruiting, Sponsor: Nationwide Children's Hospital
Recruiting --> Completed Not yet recruiting --> Recruiting
- SAF-301 / Lysogene, Alcyone
Trial completion, Gene therapy, Viral vector: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome (clinicaltrials.gov) - Jul 8, 2013
P1/2, N=4, Completed, Sponsor: LYSOGENE
Not yet recruiting --> Recruiting Active, not recruiting --> Completed
- 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
Active, not recruiting --> Completed Not yet recruiting --> Recruiting
- SAF-301 / Lysogene, Alcyone
New P1/2 trial, Gene therapy, Viral vector: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome (clinicaltrials.gov) - Nov 16, 2011
P1/2, N=4, Completed, Sponsor: LYSOGENE