NCT00433147

Brief 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Mar 2007

Shorter than P25 for phase_2

Geographic Reach
1 country

10 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 7, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

February 9, 2007

Completed
1 month until next milestone

Study Start

First participant enrolled

March 23, 2007

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 19, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 19, 2008

Completed
10.5 years until next milestone

Results Posted

Study results publicly available

August 15, 2018

Completed
Last Updated

September 25, 2018

Status Verified

August 1, 2018

Enrollment Period

11 months

First QC Date

February 7, 2007

Results QC Date

July 23, 2018

Last Update Submit

August 28, 2018

Conditions

Gaucher Disease, Type 1Type 1 Gaucher DiseaseGaucher Disease

Keywords

afegostat tartrateisofagomine tartrateAT2101Amicus Therapeutics

Outcome Measures

Primary Outcomes (1)

  • 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.

    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)

    Baseline, Day 28

Study Arms (4)

Afegostat tartrate 25 milligrams (mg) once per day

EXPERIMENTAL

Afegostat tartrate was administered orally during the 4-week treatment period.

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.

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.

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.

Drug: Afegostat tartrate

Interventions

Afegostat tartrateDRUG
Also known as: isofagomine tartrate, AT2101
Afegostat tartrate 150 mg once every four daysAfegostat tartrate 150 mg once every seven daysAfegostat tartrate 150 mg once per dayAfegostat tartrate 25 milligrams (mg) once per day

Eligibility Criteria

Age18 Years - 74 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Unknown Facility

San Francisco, California, 94143, United States

Location

Unknown Facility

Coral Springs, Florida, 33065, United States

Location

Unknown Facility

Decatur, Georgia, 30033, United States

Location

Unknown Facility

Iowa City, Iowa, 52242, United States

Location

Unknown Facility

New York, New York, 10016, United States

Location

Unknown Facility

Cincinnati, Ohio, 45229, United States

Location

Unknown Facility

Portland, Oregon, 97239, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, 19104, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, 15213, United States

Location

Unknown Facility

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

AT2101

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Results Point of Contact

Title
Amicus Therapeutics
Organization
Patient Advocacy
Clinicaltrials@Alexion.com
+1-609-662-2000

Study Officials

  • Medical Monitor, Clinical Research

    Amicus Therapeutics

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 7, 2007

First Posted

February 9, 2007

Study Start

March 23, 2007

Primary Completion

February 19, 2008

Study Completion

February 19, 2008

Last Updated

September 25, 2018

Results First Posted

August 15, 2018

Record last verified: 2018-08

Locations

US(10)