Type I Gaucher Disease MedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's disease
Conditions
Interventions
Product Name: afegostat tartrate
Product Code: AT2101
Pharmaceutical Form: Capsule*
INN or Proposed INN: afegostat tartrate
CAS Number: 919364-56-0
Current Sponsor code: AT2101
Concentration unit: mg
Sponsors
Amicus Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male or female subjects, 18 years of age or older - Completed study GAU-CL-202 with no significant protocol violations or safety concerns - Clinically stable - Has not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) in the past 12 months and is willing not to initiate ERT or SRT during study participation - All subjects of reproductive potential are required to practice an acceptable method of contraception as defined in Section 5.5 of the protocol - Provides written informed consent to participate in the study Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - During the screening period (Visit 1), any clinically significant findings, based on physical and brief neurological examination, ophthalmologic examination, medical history review, laboratory assessment, vital sign assessment and/or other significant finding which would compromise the safety of the subject, or preclude the subject from completing the study as deemed by the investigator - A clinically significant disease, severe complications from Gaucher disease, or serious intercurrent illness that may preclude participation in the study, in the opinion of the Investigator - History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (e.g., miglustat) - Pacemaker or other contraindication for MRI scanning - Pregnant or breast-feeding - Presence or sequelae of gastrointestinal, liver, or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs - Subject is otherwise unsuitable for the study in the opinion of the investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): - Findings on physical, brief neurological, and ophthalmologic examination - Vital signs (blood pressure, heart rate, body temperature, respiratory rate) - Clinical laboratory tests (hematology, serum chemistry, urinalysis) - Electrocardiogram (ECG) - Adverse events - Changes in concomitant medications;Main Objective: The primary objective of the study is to evaluate the long-term safety of orally administered AT2101 in adult patients with type 1 Gaucher disease;Secondary Objective: The secondary objective of the study is to assess the long-term efficacy of orally administered AT2101 in adult patients with type 1 Gaucher disease. The tertiary objective of the study is to assess the pharmacodynamics of orally administered AT2101 in adult patients with type 1 Gaucher disease | — |
Countries
United Kingdom
Outcome results
None listed