Trial Outcomes & Findings for A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease (NCT NCT00283933)
NCT ID: NCT00283933
Last Updated: 2018-09-07
Results Overview
TEAEs were defined as any adverse event 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 adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
COMPLETED
PHASE2
5 participants
Day 1 (after dosing) through Week 48
2018-09-07
Participant Flow
Participant milestones
| Measure |
Migalastat
Migalastat 150 milligrams (mg) was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
|
|---|---|
|
Treatment Period
STARTED
|
5
|
|
Treatment Period
Safety Population
|
5
|
|
Treatment Period
Pharmacodynamic (PD) Population
|
5
|
|
Treatment Period
COMPLETED
|
5
|
|
Treatment Period
NOT COMPLETED
|
0
|
|
Extension Period
STARTED
|
5
|
|
Extension Period
COMPLETED
|
5
|
|
Extension Period
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Baseline characteristics by cohort
| Measure |
Migalastat
n=5 Participants
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
|
|---|---|
|
Age, Continuous
|
41.6 years
STANDARD_DEVIATION 9.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 (after dosing) through Week 48Population: Safety Population: All participants who received at least 1 dose of study drug.
TEAEs were defined as any adverse event 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 adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Migalastat
n=5 Participants
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
|
|---|---|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
|
0 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24 (end of treatment period), Week 48 (end of extension period)Population: PD Population: All participants who received at least 1 dose of study drug and had at least 1 non-missing postbaseline PD parameter recorded.
PBMC were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole \[nmol\] 4-MU/hour \[hr\]) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.
Outcome measures
| Measure |
Migalastat
n=5 Participants
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
|
|---|---|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 1: Baseline
|
0.05 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 1: Week 24
|
0.36 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 1: Week 48
|
0.1 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 2: Baseline
|
0.06 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 2: Week 24
|
0.13 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 2: Week 48
|
0.1 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 3: Baseline
|
0.14 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 3: Week 24
|
0.3 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 3: Week 48
|
0.33 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 4: Baseline
|
3.4 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 4: Week 24
|
10.9 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 4: Week 48
|
7.4 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 5: Baseline
|
0.18 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 5: Week 24
|
1.32 nmol 4-MU/hr/mg protein
|
|
α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Participant 5: Week 48
|
0.13 nmol 4-MU/hr/mg protein
|
Adverse Events
Migalastat
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Migalastat
n=5 participants at risk
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
|
|---|---|
|
Cardiac disorders
Ventricular tachycardia
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Gastroenteritis
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Infections and infestations
Nasopharyngitis
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Investigations
Gamma-glutamyltransferase increased
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Metabolism and nutrition disorders
Gout
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Renal and urinary disorders
Haematuria
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Renal and urinary disorders
Proteinuria
|
40.0%
2/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Reproductive system and breast disorders
Scrotal mass
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Injury, poisoning and procedural complications
Contusion
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Injury, poisoning and procedural complications
Muscle strain
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Dizziness
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Nervous system disorders
Headache
|
40.0%
2/5 • Day 1 after dosing through Week 48 (end of extension period)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
1/5 • Day 1 after dosing through Week 48 (end of extension period)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The investigator can only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, 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