|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
12 December 2020 |
|
Main ID: |
NCT00813865 |
|
Date of registration:
|
22/12/2008 |
|
Prospective Registration:
|
Yes |
|
Primary sponsor: |
|
|
Public title:
|
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
|
|
Scientific title:
|
An Open-Label, Multicenter, Long-Term Extension Study To Assess The Safety, Efficacy, And Pharmacodynamics Of AT2101 In Adult Patients With Type 1 Gaucher Disease |
|
Date of first enrolment:
|
May 11, 2009 |
|
Target sample size:
|
8 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00813865 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
Paraguay
|
United Kingdom
|
United States
| | | | | |
|
Contacts
|
|
Name:
|
Medical Director |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
Amicus Therapeutics, Inc. |
| | |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Male or female participants, 18 years of age or older
- Completed study GAU-CL-202 with no significant protocol violations or safety concerns
- Clinically stable
- Had not received enzyme replacement therapy (ERT) or substrate reduction therapy (SRT)
in the past 12 months and was willing not to initiate ERT or SRT during study
participation
- Agreed to practice an acceptable method of contraception
- Provided written informed consent to participate in the study
Exclusion Criteria:
- During the screening period, had any clinically significant findings which would
compromise the safety of the participant, or preclude the participant from completing
the study as deemed by the investigator
- Had 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
- Had a history of allergy or sensitivity to the study drug or any excipients, including
any prior serious allergic reaction to iminosugars (for example, miglustat)
- Had a pacemaker or other contraindication for magnetic resonance imaging scanning
- Was pregnant or breast-feeding
- Had current gastrointestinal, liver, or kidney disease, sequelae of these diseases, or
other conditions known to interfere with the absorption, distribution, metabolism, or
excretion of drugs
- Participant was otherwise unsuitable for the study in the opinion of the Investigator
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Type 1 Gaucher Disease
|
|
Gaucher Disease
|
|
Gaucher Disease, Type 1
|
|
Intervention(s)
|
|
Drug: afegostat tartrate
|
|
Primary Outcome(s)
|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
[Time Frame: Day 1 (after dosing) through end of follow-up (6 months after EOT)]
|
|
Secondary Outcome(s)
|
|
Change From Baseline To EOT In Volume Of Liver As Assessed By MRI
[Time Frame: Baseline, Month 30]
|
|
Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI)
[Time Frame: Baseline, Month 30]
|
|
Secondary ID(s)
|
|
GAU-CL-202X
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|