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.

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2008-007158-36-GB
• Date of registration: 29/04/2009
• Prospective Registration: No
• Primary sponsor: Amicus Therapeutics, Inc.
• Public title: 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.
• Scientific title: 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.
• Date of first enrolment: 25/02/2009
• Target sample size: 16
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007158-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: no Open: yes Single blind: no Double blind: no Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

United Kingdom

Key inclusion & exclusion 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:
- 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Type I Gaucher Disease
MedDRA version: 9.1 Level: LLT Classification code 10018048 Term: Gaucher's disease

Intervention(s)

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 milligram(s)
Concentration type: equal
Concentration number: 25-

Primary Outcome(s)

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

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

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

Secondary ID(s)

GAU-CL-202X