A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 16

Inclusion Criteria

Subjects must meet all of the following criteria in order to be considered for enrollment into the study:
•Confirmed diagnosis of type 1 Gaucher disease with a known genotype and a documented missense gene mutation in at least one of the two mutated GBA alleles
•Clinically stable
•Treatment naïve to ERT/SRT or has not received ERT/SRT in the 12 months before screening
•Willing not to initiate ERT or SRT treatment during study participation
•Male or female subjects, 18 to 74 years old inclusive
•At the screening period visit (Day -21 to Day -1), subjects must meet at least two of the following criteria:
- Platelet count of =150,000 per microliter (=150 x 109 per liter)
- Hemoglobin =12 g/dL for females and =13 g/dL for males
- Liver volume =1.25 multiples of normal
- Spleen volume =2 multiples of normal
•All subjects of reproductive potential are required to practice an acceptable method of contraception as defined in Section 5.5 of the protocol
•Provide 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

Subjects presenting with any of the following will not be included in the study:
•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
•During the screening period, any clinically significant findings, based on physical and brief neurological 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
•Partial or total splenectomy
•Documentation of moderate or severe pulmonary hypertension, defined as pulmonary arterial pressure (PAP) >35 mmHg or significant Gaucher related lung disease
•History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars (e.g., miglustat)
•Screening period visit (Day -21 to Day -1) or Day 1 12-lead ECG demonstrating a QTc >450 msec for males or >470 msec for females, prior to dosing
•Pacemaker or other contraindication for MRI scanning
•Pregnant or breast-feeding
•Current/recent drug or alcohol abuse
•Treatment with any investigational product in the last 90 days before study entry
•Treatment in the previous 90 days with any drug known to have a well defined potential for toxicity to a major organ
•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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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