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6 Products |  |
0 Diseases | | 6 Products |
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19 Trials |  |
656 News |  |
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- | Galafold (migalastat) / Amicus
Clinical, Journal, Gold Standard: The migalastat GLP-HEK assay is the gold standard for determining amenability in patients with Fabry disease. (Pubmed Central) - Aug 2, 2019
No abstract available
- | Galafold (migalastat) / Amicus
Enrollment closed: MAIORA: Effect of Migalastat on Cardiac Involvement in Fabry Disease (clinicaltrials.gov) - Jul 31, 2019
P=N/A, N=18, Active, not recruiting, Sponsor: Ospedale San Donato
No abstract available Recruiting --> Active, not recruiting
- | Plicera (afegostat tartrate) / Amicus
Journal: Metabolism, transformation and dynamic changes of alkaloids in silkworm during feeding mulberry leaves. (Pubmed Central) - Jul 19, 2019
The experimental results indicated that DNJ and its derivatives turned into some intermediate substances in the metabolic process, and finally they converted back and the content increased. Fagomine and its derivatives interconverted into each other in the process, 4-O-β-d-Glc-fagomine transformed into fagomine, while fagomine transformed into isofagomine.
- | Galafold (migalastat) / Amicus
New P3 trial: A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease (clinicaltrials.gov) - Jul 14, 2019
P3, N=12, Not yet recruiting, Sponsor: Amicus Therapeutics
- | Plicera (afegostat tartrate) / Amicus
Journal: 4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations. (Pubmed Central) - Jun 26, 2019
Compound 1 is clearly capable of restoring β-galactosidase activity and of promoting maturation of the protein, which should result in significant clinical benefit. These properties strongly support the development of compound 1 for the treatment of GM1-gangliosidosis and Morquio disease type B patients harboring β-galactosidase mutations sensitive to pharmacological chaperoning.
- | larazotide (AT1001) / Innovate Biopharmaceuticals, Galafold (migalastat) / Amicus
Clinical, Journal: SERS detection of urea and ammonium sulfate adulterants in milk with coffee ring effect. (Pubmed Central) - Jun 14, 2019
With AuNPs, coefficients of determination (R) of 0.9873 and 0.9859 were achieved for urea and AmS, respectively, while for Au@AgNPs values of 0.9827 and 0.9855 were obtained for urea and AmS, respectively. This study revealed that SERS based on coffee ring effect has the potential to be further exploited for detecting other banned and hazardous adulterants in milk and milk products.
- | scAVV9.CB.CLN6 / Amicus
Trial completion date, Trial primary completion date, Gene therapy: Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (clinicaltrials.gov) - May 31, 2019
P1/2, N=12, Active, not recruiting, Sponsor: Emily de los Reyes
This study revealed that SERS based on coffee ring effect has the potential to be further exploited for detecting other banned and hazardous adulterants in milk and milk products. Trial completion date: Mar 2019 --> Mar 2023 | Trial primary completion date: Mar 2019 --> Mar 2022
- | Galafold (migalastat) / Amicus
Trial completion date, Trial primary completion date, HEOR, Adherence: MALTA-FABRY: MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study (clinicaltrials.gov) - May 28, 2019
P=N/A, N=30, Recruiting, Sponsor: Wuerzburg University Hospital
Trial completion date: Mar 2019 --> Mar 2023 | Trial primary completion date: Mar 2019 --> Mar 2022 Trial completion date: Mar 2019 --> Dec 2020 | Trial primary completion date: Dec 2018 --> Dec 2019
- | Plicera (afegostat tartrate) / Amicus
Journal: Lactate transport facilitates neurite outgrowth. (Pubmed Central) - May 15, 2019
Additionally, GSK-3βSer9 mutation reversed neurite growth failure caused by DAB and isofagomine. Our results suggested that lactate transportation played a critical role in neural development and disruption of lactate shuttle in quiescent condition also affected neurite outgrowth in central nervous system.
- | Galafold (migalastat) / Amicus
New trial: GALAFAB: A Study of Migalastat in Fabry Disease (clinicaltrials.gov) - May 13, 2019
P, N=21, Not yet recruiting, Sponsor: Manchester University NHS Foundation Trust
- | Galafold (migalastat) / Amicus
$FOLD Q1 Galafold revenue $34M. FY19 revenue guidance of $160M-$180M. Cash $438M (Twitter) - May 8, 2019
- | JR-051 (agalsidase beta biosimilar) / JCR, Amicus, GSK
Clinical, Journal: Non-clinical evaluation of JR-051 as a biosimilar to agalsidase beta for the treatment of Fabry disease. (Pubmed Central) - Apr 28, 2019
Furthermore, we identified no safety concerns regarding JR-051 in a 13-week evaluation using cynomolgus monkeys. These findings indicate that JR-051 is similar to agalsidase beta in terms of physicochemical and biological properties.
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus
Enrollment closed: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (clinicaltrials.gov) - Apr 25, 2019
P1/2, N=32, Active, not recruiting, Sponsor: Amicus Therapeutics
These findings indicate that JR-051 is similar to agalsidase beta in terms of physicochemical and biological properties. Recruiting --> Active, not recruiting
- | Galafold (migalastat) / Amicus
Biomarker, Clinical, P3 data, Journal: Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial. (Pubmed Central) - Apr 17, 2019
P3
Migalastat was associated with a clinically meaningful improvement in diarrhea in patients with Fabry disease and amenable mutations. Reductions in kidney globotriaosylceramide may be a useful surrogate endpoint to predict clinical benefit with migalastat in patients with Fabry disease.
- | Galafold (migalastat) / Amicus, CRISPR-Cas9 gene editing therapeutic / CRISPR Therap
Preclinical, Journal: Generation of GLA-Knockout Human Embryonic Stem Cell Lines to Model Autophagic Dysfunction and Exosome Secretion in Fabry Disease-Associated Hypertrophic Cardiomyopathy. (Pubmed Central) - Apr 11, 2019
This caused impairment of autophagic flux and protein turnover, resulting in an increase of reactive oxygen species and apoptosis. To summarize, we established a FD model which can be used as a promising tool to study human hypertrophic cardiomyopathy in a physiologically and pathologically relevant manner and to develop new therapies by targeting Rab GTPases signaling-related exosomal vesicles transportation.
- | Galafold (migalastat) / Amicus
FDA event, Journal: FDA approves Galafold, a triumph for Amicus. (Pubmed Central) - Apr 11, 2019
To summarize, we established a FD model which can be used as a promising tool to study human hypertrophic cardiomyopathy in a physiologically and pathologically relevant manner and to develop new therapies by targeting Rab GTPases signaling-related exosomal vesicles transportation. No abstract available
- |||| Pombiliti (cipaglucosidase alfa-atga) / Amicus
New P3 trial: ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (clinicaltrials.gov) - Apr 11, 2019
P3, N=14, Not yet recruiting, Sponsor: Amicus Therapeutics
- Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Apr 2, 2019
P2, N=8, Completed, Sponsor: Ultragenyx Pharmaceutical Inc
No abstract available Active, not recruiting --> Completed
- | Galafold (migalastat) / Amicus
Journal: Glucosylceramide synthase inhibition with lucerastat lowers globotriaosylceramide and lysosome staining in cultured fibroblasts from Fabry patients with different mutation types. (Pubmed Central) - Mar 29, 2019
Lucerastat's effects in the cell lines, were compared to those with current treatments; agalsidase alfa and migalastat. Consequently, the glucosylceramide synthase inhibitor lucerastat provides a viable mechanism to reduce Gb3 accumulation and lysosome volume, suitable for all Fabry patients regardless of genotype.
- |||||| Pombiliti (cipaglucosidase alfa-atga) / Amicus
New trial: Expanded Access for ATB200/AT2221 for the Treatment of Pompe Disease (clinicaltrials.gov) - Mar 7, 2019
P, N=0, Available, Sponsor: Amicus Therapeutics
- |||||| Galafold (migalastat) / Amicus
New trial: MAIORA: Effect of Migalastat on Cardiac Involvement in Fabry Disease (clinicaltrials.gov) - Feb 11, 2019
P, N=15, Recruiting, Sponsor: Ospedale San Donato
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus
Trial completion date, Trial primary completion date: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (clinicaltrials.gov) - Feb 7, 2019
P1/2, N=32, Recruiting, Sponsor: Amicus Therapeutics
Consequently, the glucosylceramide synthase inhibitor lucerastat provides a viable mechanism to reduce Gb3 accumulation and lysosome volume, suitable for all Fabry patients regardless of genotype. Trial completion date: Sep 2019 --> Sep 2021 | Trial primary completion date: Sep 2019 --> Sep 2021
- | scAVV9.CB.CLN6 / Amicus
Enrollment closed, Gene therapy: Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (clinicaltrials.gov) - Jan 23, 2019
P1/2, N=12, Active, not recruiting, Sponsor: Nationwide Children's Hospital
Trial completion date: Sep 2019 --> Sep 2021 | Trial primary completion date: Sep 2019 --> Sep 2021 Recruiting --> Active, not recruiting
- || Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion: A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) (clinicaltrials.gov) - Jan 18, 2019
P3, N=12, Completed, Sponsor: Ultragenyx Pharmaceutical Inc
Recruiting --> Active, not recruiting Active, not recruiting --> Completed
- Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion date, Trial primary completion date: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Jan 4, 2019
P2, N=8, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Active, not recruiting --> Completed Trial completion date: Dec 2018 --> Mar 2019 | Trial primary completion date: Dec 2018 --> Mar 2019
- |||||| Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
New trial: Expanded Access to Mepsevii (clinicaltrials.gov) - Dec 12, 2018
P, N=0, Available, Sponsor: Ultragenyx Pharmaceutical Inc
- AT-GTX-502 / Amicus
New P1/2 trial, Gene therapy: Gene Therapy for Children With CLN3 Batten Disease (clinicaltrials.gov) - Dec 10, 2018
P1/2, N=7, Recruiting, Sponsor: Nationwide Children's Hospital
- |||| Pombiliti (cipaglucosidase alfa-atga) / Amicus
Enrollment open: A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (clinicaltrials.gov) - Dec 4, 2018
P3, N=100, Recruiting, Sponsor: Amicus Therapeutics
Trial completion date: Dec 2018 --> Mar 2019 | Trial primary completion date: Dec 2018 --> Mar 2019 Not yet recruiting --> Recruiting
- | Galafold (migalastat) / Amicus
Enrollment open, Enrollment change, Trial completion date, Trial initiation date, Trial primary completion date: Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) (clinicaltrials.gov) - Nov 16, 2018
P3, N=20, Recruiting, Sponsor: Amicus Therapeutics
Not yet recruiting --> Recruiting Not yet recruiting --> Recruiting | N=10 --> 20 | Trial completion date: May 2020 --> Sep 2020 | Initiation date: Jun 2018 --> Oct 2018 | Trial primary completion date: May 2020 --> Sep 2020
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus
New P3 trial: A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (clinicaltrials.gov) - Nov 1, 2018
P3, N=100, Not yet recruiting, Sponsor: Amicus Therapeutics
- | Galafold (migalastat) / Amicus
Trial termination: Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study (clinicaltrials.gov) - Oct 3, 2018
P2, N=23, Terminated, Sponsor: Amicus Therapeutics
Not yet recruiting --> Recruiting | N=10 --> 20 | Trial completion date: May 2020 --> Sep 2020 | Initiation date: Jun 2018 --> Oct 2018 | Trial primary completion date: May 2020 --> Sep 2020 Completed --> Terminated; The Sponsor (Amicus Therapeutics) terminated this study for logistical reasons.
- | Galafold (migalastat) / Amicus
Trial termination, Monotherapy: Open-Label Phase 3 Long-Term Safety Study of Migalastat (clinicaltrials.gov) - Oct 2, 2018
P3, N=85, Terminated, Sponsor: Amicus Therapeutics
Completed --> Terminated; The Sponsor (Amicus Therapeutics) terminated this study for logistical reasons. Completed --> Terminated; Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons.
- | Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial primary completion date: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Sep 13, 2018
P2, N=8, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Completed --> Terminated; Amicus Therapeutics discontinued Study AT1001-041 for logistical reasons. Trial primary completion date: Dec 2017 --> Oct 2018
- | Galafold (migalastat) / Amicus
Enrollment change: A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease (clinicaltrials.gov) - Sep 7, 2018
P2, N=9, Completed, Sponsor: Amicus Therapeutics
Trial primary completion date: Dec 2017 --> Oct 2018 N=14 --> 9
- | Galafold (migalastat) / Amicus
Enrollment closed: German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (clinicaltrials.gov) - Aug 31, 2018
P=N/A, N=60, Active, not recruiting, Sponsor: University Hospital Muenster
N=14 --> 9 Enrolling by invitation --> Active, not recruiting
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus
Enrollment open, Enrollment change, Trial completion date, Trial primary completion date: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (clinicaltrials.gov) - Apr 19, 2018
P1/2, N=32, Recruiting, Sponsor: Amicus Therapeutics
Enrolling by invitation --> Active, not recruiting Active, not recruiting --> Recruiting | N=20 --> 32 | Trial completion date: Jun 2019 --> Sep 2019 | Trial primary completion date: Jun 2019 --> Sep 2019
- | Galafold (migalastat) / Amicus
New P3 trial: Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years) (clinicaltrials.gov) - Apr 17, 2018
P3, N=7, Not yet recruiting, Sponsor: Amicus Therapeutics
- Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion date: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Mar 3, 2018
P2, N=8, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Active, not recruiting --> Recruiting | N=20 --> 32 | Trial completion date: Jun 2019 --> Sep 2019 | Trial primary completion date: Jun 2019 --> Sep 2019 Trial completion date: Sep 2021 --> Dec 2018
- Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Enrollment change: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Nov 6, 2017
P2, N=8, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Trial completion date: Sep 2021 --> Dec 2018 N=15 --> 8
- | Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Enrollment change: A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) (clinicaltrials.gov) - Nov 6, 2017
P3, N=12, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
N=15 --> 8 N=20 --> 12
- Galafold (migalastat) / Amicus
Phase classification: Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease (clinicaltrials.gov) - Aug 25, 2017
P=N/A, N=0, Available, Sponsor: Amicus Therapeutics
N=20 --> 12 Phase classification: P3 --> P=N/A
- || Galafold (migalastat) / Amicus
Enrollment status: German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (clinicaltrials.gov) - Jun 14, 2017
P=N/A, N=60, Enrolling by invitation, Sponsor: University Hospital Muenster
Phase classification: P3 --> P=N/A Recruiting --> Enrolling by invitation
- ||| Galafold (migalastat) / Amicus
Enrollment open: German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (clinicaltrials.gov) - Jun 8, 2017
P=N/A, N=60, Recruiting, Sponsor: University Hospital Muenster
Recruiting --> Enrolling by invitation Not yet recruiting --> Recruiting
- ||| Galafold (migalastat) / Amicus
New trial: German Observational Multicenter Study of Patients With Fabry Disease Under Chaperone Therapy With Migalastat-HCl. (clinicaltrials.gov) - Apr 30, 2017
P=N/A, N=60, Not yet recruiting, Sponsor: University Hospital Muenster
- | Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Enrollment closed: A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) (clinicaltrials.gov) - Apr 19, 2017
P3, N=20, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Not yet recruiting --> Recruiting Recruiting --> Active, not recruiting
- | scAVV9.CB.CLN6 / Amicus
Trial primary completion date, Gene therapy: Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease (clinicaltrials.gov) - Mar 30, 2017
P1/2, N=6, Recruiting, Sponsor: Nationwide Children's Hospital
Recruiting --> Active, not recruiting Trial primary completion date: Jul 2018 --> Mar 2019
- | Pombiliti (cipaglucosidase alfa-atga) / Amicus
Enrollment closed: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221 (clinicaltrials.gov) - Mar 16, 2017
P1/2, N=18, Active, not recruiting, Sponsor: Amicus Therapeutics
Trial primary completion date: Jul 2018 --> Mar 2019 Recruiting --> Active, not recruiting
- | Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Enrollment closed: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age (clinicaltrials.gov) - Mar 8, 2017
P2, N=15, Active, not recruiting, Sponsor: Ultragenyx Pharmaceutical Inc
Recruiting --> Active, not recruiting Recruiting --> Active, not recruiting
- || Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion: A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) (clinicaltrials.gov) - Mar 8, 2017
P3, N=12, Completed, Sponsor: Ultragenyx Pharmaceutical Inc
Recruiting --> Active, not recruiting Active, not recruiting --> Completed
- Mepsevii (vestronidase alfa) / Ultragenyx, Amicus
Trial completion, Trial primary completion date: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) (clinicaltrials.gov) - Mar 8, 2017
P1/2, N=3, Completed, Sponsor: Ultragenyx Pharmaceutical Inc
Active, not recruiting --> Completed Active, not recruiting --> Completed | Trial primary completion date: Jun 2015 --> Jul 2016