These trials completed more than 12 months ago and should have reported results. Some have, some have not.

These trials are not yet due to report results: some are ongoing, some completed within the past 12 months.

These trials have problematic data on the registry. Details why »

Status Trial ID Title Completion date Category
Reported results 2005-004384-33 A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease 2008-03-13 due-trials
Reported results 2006-000181-36 A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients with Fabry Disease 2008-05-09 due-trials
Reported results 2007-001838-13 Open Label Extension Study to Evaluate the Long-term Safety Tolerability and Pharmacodynamics of AT1001 in Patients with Fabry Disease 2012-10-04 due-trials
Reported results 2007-003359-35 A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE 2009-11-16 due-trials
Reported results 2008-002302-18 An open-label, multicenter, study to evaluate the safety, tolerability, pharmacodynamics, and pharmacokinetics of three dosing regimens of oral AT2220 in patients with Pompe disease 2009-11-09 due-trials
Completed, but no date, and reported results Terminated 2008-007158-36 An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. bad-data
Reported results 2009-013459-31 A DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY AND PHARMACODYNAMICS OF AT1001 IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS 2014-01-29 due-trials
Reported results 2010-022636-37 A RANDOMIZED, OPEN-LABEL STUDY TO COMPARE THE EFFICACY AND SAFETY OF AT1001 AND ENZYME REPLACEMENT THERAPY (ERT) IN PATIENTS WITH FABRY DISEASE AND AT1001-RESPONSIVE GLA MUTATIONS, WHO WERE PREVIOUSLY... 2015-05-28 due-trials
Reported results 2010-022709-16 AN OPEN-LABEL PHASE 2A STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT1001 (MIGALASTAT HYDROCHLORIDE) AND AGALSIDASE IN SUBJECTS WITH FABRY DISEASE 2012-10-09 due-trials
Reported results 2011-002154-32 AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE 2013-01-04 due-trials
Reported results 2011-004800-40 An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease. Uno studio di estensione in aperto per valutare... 2016-02-17 due-trials
Not reported Terminated 2013-002257-30 AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID α-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE 2013-12-06 due-trials
Listed as ongoing, but also has a completion date and reported results 2014-002288-14 A Phase 3, Multi-center, Randomized, Double-Blind, Placebo Controlled Study of the Efficacy and Safety of SD-101 Cream in Patients with Epidermolysis Bullosa 2017-07-05 bad-data
Reported results 2014-002701-38 An Open-Label, Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in subjects with Fabry Disease 2019-10-23 due-trials
Reported results 2014-005679-96 An Open Label Multi-Center Extension Study to Evaluate the Long-term Safety of ZorblisaTM (SD-101-6.0) in Patients with Epidermolysis Bullosa An Open Label Multi-Center Extension Study to Evaluate ... 2018-09-03 due-trials
Exempt 2015-004798-34 AN OPEN-LABEL, FIXED-SEQUENCE, ASCENDING-DOSE, FIRST-IN-HUMAN STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF INTRAVENOUS INFUSIONS OF ATB200 CO-ADMINISTE... 2024-08-20 not-yet-due
Listed as ongoing, but also has a completion date and reported results 2017-000146-21 AN OPEN-LABEL STUDY OF THE SAFETY, PHARMACOKINETICS, PHARMACODYNAMICS, AND EFFICACY OF 12-MONTH TREATMENT WITH MIGALASTAT IN PEDIATRIC SUBJECTS (AGED 12 TO <18 YEARS) WITH FABRY DISEASE AND AMENABLE G... 2021-02-02 bad-data
Reported results Terminated 2017-004370-34 A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy 2018-09-03 due-trials
Listed as ongoing, but also has a completion date and reported results 2018-000755-40 A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Alglucosi... 2020-12-15 bad-data
Other 2018-001133-40 An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Fabry Subjects with Amenable GLA Variants and Severe Renal Impairment not-yet-due
Other 2019-000222-21 A LONG-TERM, OPEN-LABEL STUDY TO EVALUATE THE SAFETY, PHARMACODYNAMICS, AND EFFICACY OF MIGALASTAT IN SUBJECTS > 12 YEARS OF AGE WITH FABRY DISEASE AND AMENABLE GLA VARIANTS not-yet-due
Ongoing 2019-000954-67 A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease not-yet-due
Exempt, with results Terminated 2021-000706-21 LONG-TERM FOLLOW-UP OF AT-GTX-501 SCAAV9 GENE TRANSFER IN SUBJECTS WITH CLN6 BATTEN DISEASE 2021-12-09 not-yet-due