Type 1 Gaucher DiseasePhase 1NCT00875160
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101
A Phase 1 study testing AT2101 in people with Type 1 Gaucher Disease, run by Amicus Therapeutics at 2 sites in 2 countries.
Trial summary
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Type 1 Gaucher Disease
- What is being tested?
- AT2101
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Apr 2009to Jul 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Have a confirmed diagnosis of type 1 Gaucher disease with a documented gene mutation;
- Clinically stable and on a stable dose of ERT for at least 2 years before study entry, with no changes to dose level or regimen in the last 6 months;
- Willing to stop ERT for the duration of the study;
- Male or female between the ages of 18 to 65 inclusive;
- All subjects of reproductive potential are required to practice an acceptable method of contraception;
- All subjects must have a body mass index of less than 30; and
- Provide written informed consent to participate in the study.
Who cannot join
- 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/wash out period, any clinically significant findings, based on physical exam, medical history review, lab 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;
- History of pulmonary hypertension or Gaucher-related lung disease;
- History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars;
- Screening or Day 1 (before dosing) 12-lead ECG demonstration QTc >450 msec in males or >470 in females;
Show all 12Show fewer
- Pregnant or breast-feeding;
- Current/recent drug or alcohol abuse within the past 12 months;
- Treatment with any investigational product, including investigational forms of ERT, in the 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; or
- Subject is otherwise unsuitable for the study in the opinion of the Investigator.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
Official title: An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days).
The study consists of a screening period (~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.
Read the detailed description
While on the study, subjects will be required to refrain from consuming the following foods and beverages:
- Caffeine: 24 hours before admission and throughout the in-patient stay;
- Alcohol: 48 hours before admission and throughout the in-patient stay. A maximum of 2 units/day will be allowed during the out-patient part of the study (1 unit of alcohol = wind (125mL)=spirits(25mL)=beer (284mL);
- Vitamins: throughout the in-patient periods.
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
- Conditions
- Type 1 Gaucher Disease
- Being tested
- AT2101
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AT2101Drug
225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10)
| Study group | Role | What they receive |
|---|---|---|
| AT2101 | Experimental | Not described Receives: Drug: AT2101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease
Measured: Multiple times for up to three days following Day 1 and Day 10
Secondary outcomes (2)
To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease
Measured: Daily
To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease.
Measured: Days 1, 5 & 7 (optional), 10 and 17
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2009 (estimated)
- Study startApr 2009
- Primary completionJul 2009
- Study completionJul 2009
Why it stopped: Subject Recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Type 1 Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Eugene Schneider, MD, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00875160
- Sponsor's protocol ID
- GAU-CL-104
- Status last verified
- Aug 2010
- First submitted
- 2 Apr 2009
- Last updated
- 19 Aug 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Assess the Pharmacokinetics, Safety, and Pharmacodynamics of Repeated Doses of Orally Administered AT2101 in Adult Patients With Type 1 Gaucher Disease
- ClinicalTrials.gov ID
- NCT00875160
- Acronym / sponsor ID
- GAU-CL-104
- Conditions
- Type 1 Gaucher Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Subject Recruitment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Apr 2009
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 3 Apr 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days).
The study consists of a screening period (~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.
Detailed description
While on the study, subjects will be required to refrain from consuming the following foods and beverages:
- Caffeine: 24 hours before admission and throughout the in-patient stay;
- Alcohol: 48 hours before admission and throughout the in-patient stay. A maximum of 2 units/day will be allowed during the out-patient part of the study (1 unit of alcohol = wind (125mL)=spirits(25mL)=beer (284mL);
- Vitamins: throughout the in-patient periods.
Subjects will also be restricted to taking medications or herbal products during the study except if the Principal Investigator permits.
Subjects will not engage in strenuous activity at any time during the in-patient periods.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AT2101 | Experimental | Drug: AT2101 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Have a confirmed diagnosis of type 1 Gaucher disease with a documented gene mutation;
- Clinically stable and on a stable dose of ERT for at least 2 years before study entry, with no changes to dose level or regimen in the last 6 months;
- Willing to stop ERT for the duration of the study;
- Male or female between the ages of 18 to 65 inclusive;
- All subjects of reproductive potential are required to practice an acceptable method of contraception;
Key exclusion criteria (12 listed)
- 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/wash out period, any clinically significant findings, based on physical exam, medical history review, lab 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;
- History of pulmonary hypertension or Gaucher-related lung disease;
- History of allergy or sensitivity to the study drug or any excipients, including any prior serious allergic reaction to iminosugars;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AT2101225mg (nine 25mg capsules total dose) to be taken by mouth every day for 9 days (Study Days 1, 3-10) | Drug | AT2101 |
Primary outcome
To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease
Time frame: Multiple times for up to three days following Day 1 and Day 10
2 secondary outcomes
To evaluate the safety and tolerability of oral AT2101 administered in patients with type 1 Gaucher disease
Time frame: Daily
To evaluate the pharmacodynamic effect of WBC GCase of oral AT2101 administered in patients with type 1 Gaucher disease.
Time frame: Days 1, 5 & 7 (optional), 10 and 17
Results and safety
The study was stopped early (reason given: Subject Recruitment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2010.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Eugene Schneider, MD study director
Amicus Therapeutics
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AT2101 in people with Type 1 Gaucher Disease, run by Amicus Therapeutics at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Aug 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Subject Recruitment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate single-dose and multiple-dose pharmacokinetics of isofagomine following the oral administration of AT2101 in patients with type 1 Gaucher disease (time frame: Multiple times for up to three days following Day 1 and Day 10). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2010.