Gaucher Disease, Type 1Phase 2NCT00433147
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
A Phase 2 study testing Afegostat tartrate in people with Gaucher Disease, Type 1 and Type 1 Gaucher Disease, run by Amicus Therapeutics at 10 sites in United States.
Trial summary
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy. Read more about this study
- Who is it for?
- Ages 18 to 74 years
- Conditions the study is for
- Gaucher Disease, Type 1, Type 1 Gaucher Disease, Gaucher Disease
- What is being tested?
- Afegostat tartrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 10 sitesUnited States
- Study dates
- 23 Mar 2007to 19 Feb 2008
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 74 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Had a confirmed diagnosis of type 1 Gaucher disease with a known documented missense gene mutation in at least 1 of the 2 gene-encoding β-glucosidase alleles
- Clinically stable
- Male or female participants, 18 to 74 years old inclusive
- All participants of childbearing potential used adequate birth control
- Provided written informed consent to participate in the study
Who cannot join
- Clinically significant disease, severe complications from Gaucher disease, or serious illness that precluded participation in the study in the opinion of the Investigator that compromised the safety of the participant or precluded the participant from completing the study
- During the screening period, any clinically significant findings, as deemed by the Investigator
- Partial or total splenectomy (removal of spleen) within the 2 years prior to study entry
- 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 adverse reaction to iminosugars (for example, N-butyldeoxynojirimycin or miglustat)
- Pregnant or breast-feeding
Show all 10Show fewer
- Current/recent drug or alcohol abuse
- Treatment with any investigational product 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 symptoms of gastrointestinal, liver or kidney disease, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Study site
Cincinnati, Ohio, 45229
Study site
Coral Springs, Florida, 33065
Study site
Decatur, Georgia, 30033
Study site
Houston, Texas, 77030
Study site
Iowa City, Iowa, 52242
Study site
New York, New York, 10016
Study site
Philadelphia, Pennsylvania, 19104
Study site
Pittsburgh, Pennsylvania, 15213
Study site
Portland, Oregon, 97239
Study site
San Francisco, California, 94143
About this study
Official title: A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
Read the detailed description
This was a Phase 2, open-label study in participants with Gaucher disease, a lysosomal storage disorder. Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. The study consisted of a 14-day screening period, a 28-day treatment period, and a 7-day wash-out period. Participants received 1 of 4 dosing regimens for afegostat tartrate.
- Being tested
- Afegostat tartrate
- Keywords
- afegostat tartrate, isofagomine tartrate, AT2101, Amicus Therapeutics
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Afegostat tartrateDrug
Also called isofagomine tartrate, AT2101
| Study group | Role | What they receive |
|---|---|---|
| Afegostat tartrate 25 milligrams (mg) once per day | Experimental | Afegostat tartrate was administered orally during the 4-week treatment period. Receives: Drug: Afegostat tartrate |
| Afegostat tartrate 150 mg once per day | Experimental | Afegostat tartrate was administered orally once per day during the 4-week treatment period. Receives: Drug: Afegostat tartrate |
| Afegostat tartrate 150 mg once every four days | Experimental | Afegostat tartrate was administered orally once every 4 days during the 4-week treatment period. Receives: Drug: Afegostat tartrate |
| Afegostat tartrate 150 mg once every seven days | Experimental | Afegostat tartrate was administered orally once every 7 days during the 4-week treatment period. Receives: Drug: Afegostat tartrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
TEAEs were defined as any adverse event (AE) with a start date on or after administration of the study drug (on Day 1). A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through 7 days after the last dose of study drug (Day 35) is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Measured: Day 1 (after dosing) through Day 35
Secondary outcomes (1)
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)
GCase is a biomarker used to assess the PD effects of afegostat tartrate. Blood samples were collected to assess GCase levels in WBC. The baseline value was defined as the last non-missing value before the start of study drug.
Measured: Baseline, Day 28
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Feb 2007 (estimated)
- Study start23 Mar 2007
- Primary completion19 Feb 2008
- Study completion19 Feb 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Gaucher Disease, Type 1Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Amicus Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Clinical Research, Study director, Amicus Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00433147
- Sponsor's protocol ID
- GAU-CL-201
- Status last verified
- Aug 2018
- First submitted
- 7 Feb 2007
- Last updated
- 25 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Open-label Study to Assess the Safety and Tolerability of Multiple Dose Levels and Multiple Dosing Regimens of AT2101 in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
- ClinicalTrials.gov ID
- NCT00433147
- Acronym / sponsor ID
- GAU-CL-201
- Keywords
- afegostat tartrate, isofagomine tartrate, AT2101, Amicus Therapeutics
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Amicus TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 Mar 2007 (actual)
- Primary completion
- 19 Feb 2008 (actual)
- Study completion
- 19 Feb 2008 (actual)
- First posted
- 9 Feb 2007 (estimated)
- Results
- Posted 15 Aug 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study was conducted to test the safety and tolerability of afegostat tartrate in participants with type 1 Gaucher disease already receiving enzyme replacement therapy.
Detailed description
This was a Phase 2, open-label study in participants with Gaucher disease, a lysosomal storage disorder. Afegostat tartrate (also known as AT2101 or isofagomine tartrate) is designed to act as a pharmacological chaperone by selectively binding to misfolded β-glucocerebrosidase (GCase) and helping it fold correctly, intended to restore GCase activity. The study consisted of a 14-day screening period, a 28-day treatment period, and a 7-day wash-out period. Participants received 1 of 4 dosing regimens for afegostat tartrate.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Afegostat tartrate 25 milligrams (mg) once per day | Experimental | Drug: Afegostat tartrateAfegostat tartrate was administered orally during the 4-week treatment period. |
| Afegostat tartrate 150 mg once per day | Experimental | Drug: Afegostat tartrateAfegostat tartrate was administered orally once per day during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every four days | Experimental | Drug: Afegostat tartrateAfegostat tartrate was administered orally once every 4 days during the 4-week treatment period. |
| Afegostat tartrate 150 mg once every seven days | Experimental | Drug: Afegostat tartrateAfegostat tartrate was administered orally once every 7 days during the 4-week treatment period. |
Who may qualify
- Ages
- 18 to 74 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Had a confirmed diagnosis of type 1 Gaucher disease with a known documented missense gene mutation in at least 1 of the 2 gene-encoding β-glucosidase alleles
- Clinically stable
- Male or female participants, 18 to 74 years old inclusive
- All participants of childbearing potential used adequate birth control
- Provided written informed consent to participate in the study
Key exclusion criteria (10 listed)
- Clinically significant disease, severe complications from Gaucher disease, or serious illness that precluded participation in the study in the opinion of the Investigator that compromised the safety of the participant or precluded the participant from completing the study
- During the screening period, any clinically significant findings, as deemed by the Investigator
- Partial or total splenectomy (removal of spleen) within the 2 years prior to study entry
- 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 adverse reaction to iminosugars (for example, N-butyldeoxynojirimycin or miglustat)
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 |
|---|---|---|
| Afegostat tartrateAlso: isofagomine tartrate, AT2101 | Drug | Afegostat tartrate 150 mg once every four days, Afegostat tartrate 150 mg once every seven days, Afegostat tartrate 150 mg once per day, Afegostat tartrate 25 milligrams (mg) once per day |
Primary outcome
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 1 (after dosing) through Day 35
TEAEs were defined as any adverse event (AE) with a start date on or after administration of the study drug (on Day 1). A severe AE was defined as an AE that was incapacitating and required medical intervention. The number of participants who experienced 1 or more severe TEAEs after dosing on Day 1 through 7 days after the last dose of study drug (Day 35) is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
1 secondary outcome
Change From Baseline To End Of Treatment In Beta-glucocerebrosidase (GCase) Levels In White Blood Cells (WBC)
Time frame: Baseline, Day 28
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Medical Monitor, Clinical Research study director
Amicus Therapeutics
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Afegostat tartrate in people with Gaucher Disease, Type 1 and Type 1 Gaucher Disease, run by Amicus Therapeutics at 10 sites in United States.
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 5 inclusion and 10 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: finished, as of the sponsor's last update on 25 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Aug 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) (time frame: Day 1 (after dosing) through Day 35). 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 25 Sep 2018.