Trial Outcomes & Findings for A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients (NCT NCT00813865)
NCT ID: NCT00813865
Last Updated: 2018-08-15
Results Overview
A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE 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 the end of follow-up is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
COMPLETED
PHASE2
8 participants
Day 1 (after dosing) through end of follow-up (6 months after EOT)
2018-08-15
Participant Flow
Participants were screened for evaluation of eligibility to participate in the study. Eight participants received at least 1 dose of study drug.
Participant milestones
| Measure |
Afegostat Tartrate
Afegostat tartrate was administered orally at a dose of 225 milligram (mg) once daily (QD) on Monday, Wednesday, and Friday, with no study medication on Tuesday, Thursday, Saturday, and Sunday (MWF 3-days-on/4-days-off). Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after the end of treatment (EOT).
|
|---|---|
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Overall Study
STARTED
|
8
|
|
Overall Study
Safety Population
|
8
|
|
Overall Study
Pharmacodynamic (PD) Population
|
8
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
Afegostat Tartrate
Afegostat tartrate was administered orally at a dose of 225 milligram (mg) once daily (QD) on Monday, Wednesday, and Friday, with no study medication on Tuesday, Thursday, Saturday, and Sunday (MWF 3-days-on/4-days-off). Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after the end of treatment (EOT).
|
|---|---|
|
Overall Study
Failure to Respond
|
3
|
|
Overall Study
Lost to Follow-up
|
3
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
Baseline characteristics by cohort
| Measure |
Afegostat Tartrate
n=8 Participants
Afegostat tartrate was administered orally at a dose of 225 mg QD MWF 3-days-on/4-days-off. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
|
|---|---|
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Age, Continuous
|
41.3 years
STANDARD_DEVIATION 20.74 • n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 (after dosing) through end of follow-up (6 months after EOT)Population: Safety Population: all participants who received at least 1 dose of study drug.
A TEAE was defined as any adverse event (AE) with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe AE 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 the end of follow-up is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Afegostat Tartrate
n=8 Participants
Afegostat tartrate was administered orally at a dose of 225 mg QD MWF 3-days-on/4-days-off. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
|
|---|---|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Month 30Population: PD Population: all participants in the Safety Population who had a baseline and at least 1 post-baseline PD measurement. Seven of 8 participants did not complete the study and did not contribute data to this endpoint.
Standard MRI procedures were used to measure the volume of the spleen. The baseline value was defined as the value recorded on Days 1 to 3 of the study (certain values could have been carried over from the last assessment in the lead-in study, GAU-CL-202). A negative change from Baseline indicates that spleen volume decreased.
Outcome measures
| Measure |
Afegostat Tartrate
n=1 Participants
Afegostat tartrate was administered orally at a dose of 225 mg QD MWF 3-days-on/4-days-off. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
|
|---|---|
|
Change From Baseline To EOT In Volume Of Spleen As Assessed By Magnetic Resonance Imaging (MRI)
|
-138 cubic centimeters (cm^3)
|
SECONDARY outcome
Timeframe: Baseline, Month 30Population: PD Population: all participants in the Safety Population who had a baseline and at least 1 post-baseline PD measurement. Seven of 8 participants did not complete the study and did not contribute data to this endpoint.
Standard MRI procedures were used to measure the volume of the liver. The baseline value was defined as the value recorded on Days 1 to 3 of the study (certain values could have been carried over from the last assessment in the lead-in study, GAU-CL-202). A negative change from Baseline indicates that liver volume decreased.
Outcome measures
| Measure |
Afegostat Tartrate
n=1 Participants
Afegostat tartrate was administered orally at a dose of 225 mg QD MWF 3-days-on/4-days-off. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
|
|---|---|
|
Change From Baseline To EOT In Volume Of Liver As Assessed By MRI
|
57 cm^3
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Adverse Events
Afegostat Tartrate
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Afegostat Tartrate
n=8 participants at risk
Afegostat tartrate was administered orally at a dose of 225 mg QD MWF 3-days-on/4-days-off. Participants were to receive afegostat tartrate for 30 months and be followed for 6 months after EOT.
|
|---|---|
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Blood and lymphatic system disorders
Splenomegaly
|
25.0%
2/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Blood and lymphatic system disorders
Splenic infarction
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Eye disorders
Blepharitis
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Eye disorders
Conjunctivitis
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Eye disorders
Lacrimation increased
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Eye disorders
Macular scar
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Eye disorders
Vision blurred
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Gastrointestinal disorders
Abdominal pain
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
General disorders
Chest pain
|
25.0%
2/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
General disorders
Gait disturbance
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Hepatobiliary disorders
Hepatomegaly
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Injury, poisoning and procedural complications
Muscle strain
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Skin and subcutaneous tissue disorders
Skin exfoliation
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
12.5%
1/8 • Day 1 (after dosing) through end of follow-up (6 months after EOT)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
- Publication restrictions are in place
Restriction type: OTHER