Trial Outcomes & Findings for Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules. (NCT NCT01857362)
NCT ID: NCT01857362
Last Updated: 2021-10-12
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
12 participants
Primary outcome timeframe
Day 1 predose and at 15, 30, 45 minutes and 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8, 10, 12, 24, 36, 48 and 72 hours postdose
Results posted on
2021-10-12
Participant Flow
Participant milestones
| Measure |
Nitisinone 1 x 20 mg, Then Nitisinone 2 x 10 mg
Participants first received one nitisinone capsule of 20 mg. After washout of 3 weeks participants then received two nitisinone capsules of 10 mg.
|
Nitisinone 2 x 10 mg, Then Nitisinone 1 x 20 mg
Participants first received two nitisinone capsules of 10 mg. After washout of 3 weeks participants then received one nitisinone capsule of 20 mg.
|
|---|---|---|
|
Overall Study
STARTED
|
6
|
6
|
|
Overall Study
COMPLETED
|
6
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.
Baseline characteristics by cohort
| Measure |
Overall Study
n=12 Participants
All treatment arms
|
|---|---|
|
Age, Continuous
|
31 years
STANDARD_DEVIATION 11 • n=5 Participants
|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
12 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 1 predose and at 15, 30, 45 minutes and 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8, 10, 12, 24, 36, 48 and 72 hours postdosePopulation: Full analysis set was used; subjects with available PK data for at least one of the treatments.
Outcome measures
| Measure |
Nitisinone 2 x 10 mg Capsules
n=12 Participants
Nitisinone 2 x 10 mg capsules
|
Nitisinone 1 x 20 mg Capsules
n=12 Participants
Nitisinone 1 x 20 mg capsules
|
|---|---|---|
|
The Area Under the Serum Concentration vs. Time Profile During 72 Hours After Dose (AUC72h).
|
262 uM*h
Interval 209.0 to 336.0
|
267 uM*h
Interval 210.0 to 341.0
|
PRIMARY outcome
Timeframe: Day 1 predose and at 15, 30, 45 minutes and 1, 1.5, 2, 2.5, 3, 3.5, 4, 6, 8, 10, 12, 24, 36, 48 and 72 hours postdosePopulation: Full analysis set was used; subjects with available PK data for at least one of the treatments.
Outcome measures
| Measure |
Nitisinone 2 x 10 mg Capsules
n=12 Participants
Nitisinone 2 x 10 mg capsules
|
Nitisinone 1 x 20 mg Capsules
n=12 Participants
Nitisinone 1 x 20 mg capsules
|
|---|---|---|
|
The Maximum Serum Concentration (Cmax).
|
6438 nM
Interval 4510.0 to 8950.0
|
6366 nM
Interval 4690.0 to 9330.0
|
Adverse Events
Nitisinone 2 x 10 mg Capsules
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Nitisinone 1 x 20 mg Capsules
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Nitisinone 2 x 10 mg Capsules
n=12 participants at risk
Nitisinone 2 x 10 mg capsules
|
Nitisinone 1 x 20 mg Capsules
n=12 participants at risk
Nitisinone 1 x 20 mg capsules
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
8.3%
1/12 • Number of events 1
|
0.00%
0/12
|
|
General disorders
Catheter site related reaction
|
0.00%
0/12
|
8.3%
1/12 • Number of events 1
|
|
General disorders
Hunger
|
0.00%
0/12
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/12
|
8.3%
1/12 • Number of events 1
|
|
Metabolism and nutrition disorders
Decreased appetite
|
8.3%
1/12 • Number of events 1
|
0.00%
0/12
|
|
Metabolism and nutrition disorders
Increased appetite
|
8.3%
1/12 • Number of events 1
|
0.00%
0/12
|
|
Nervous system disorders
Dizziness
|
0.00%
0/12
|
16.7%
2/12 • Number of events 2
|
|
Nervous system disorders
Headache
|
8.3%
1/12 • Number of events 1
|
16.7%
2/12 • Number of events 2
|
|
Nervous system disorders
Somnolence
|
8.3%
1/12 • Number of events 1
|
0.00%
0/12
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/12
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
0.00%
0/12
|
8.3%
1/12 • Number of events 1
|
Additional Information
Dr Erik Brouwer, Medical Director
Swedish Orphan Biovitrum
Phone: +4686972000
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60