Trial Outcomes & Findings for Safety and Acceptability of Deferiprone Delayed Release Tablets in Patients With Systemic Iron Overload (NCT NCT03802916)
NCT ID: NCT03802916
Last Updated: 2021-07-16
Results Overview
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
Day 28
Results posted on
2021-07-16
Participant Flow
Participant milestones
| Measure |
Low Dosage
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (Low dosage): Deferiprone DR tablets 1000 mg
|
High Dosage
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (High dosage): Deferiprone DR tablets 1000 mg
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
15
|
|
Overall Study
COMPLETED
|
15
|
13
|
|
Overall Study
NOT COMPLETED
|
0
|
2
|
Reasons for withdrawal
| Measure |
Low Dosage
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (Low dosage): Deferiprone DR tablets 1000 mg
|
High Dosage
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (High dosage): Deferiprone DR tablets 1000 mg
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
The results for this baseline measure are presented separately for each group.
Baseline characteristics by cohort
| Measure |
Low Dosage
n=15 Participants
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 75 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (Low dosage): Deferiprone DR tablets 1000 mg
|
High Dosage
n=14 Participants
Patients in this group will receive a total daily dosage of deferiprone DR tablets that is closer to 100 mg/kg/day. The total dosage will be divided into two equal parts, taken about 12 hours apart.
Deferiprone DR tablets 1000 mg (High dosage): Deferiprone DR tablets 1000 mg
|
Total
n=29 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
41.9 years
STANDARD_DEVIATION 9.9 • n=15 Participants
|
40.4 years
STANDARD_DEVIATION 6.8 • n=14 Participants
|
41.1 years
STANDARD_DEVIATION 8.4 • n=29 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=15 Participants
|
7 Participants
n=14 Participants
|
13 Participants
n=29 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=15 Participants
|
7 Participants
n=14 Participants
|
16 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=15 Participants
|
1 Participants
n=14 Participants
|
1 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
15 Participants
n=15 Participants
|
13 Participants
n=14 Participants
|
28 Participants
n=29 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
2 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=15 Participants
|
13 Participants
n=14 Participants
|
26 Participants
n=29 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=15 Participants
|
0 Participants
n=14 Participants
|
0 Participants
n=29 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=15 Participants
|
1 Participants
n=14 Participants
|
1 Participants
n=29 Participants
|
|
Region of Enrollment
Greece
|
3 participants
n=15 Participants
|
5 participants
n=14 Participants
|
8 participants
n=29 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=15 Participants
|
0 participants
n=14 Participants
|
3 participants
n=29 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=15 Participants
|
2 participants
n=14 Participants
|
3 participants
n=29 Participants
|
|
Region of Enrollment
Italy
|
8 participants
n=15 Participants
|
7 participants
n=14 Participants
|
16 participants
n=29 Participants
|
|
Level of liver enzymes at baseline for low-dosage group
Baseline ALT
|
29.60 units per liter
STANDARD_DEVIATION 19.88 • n=15 Participants • The results for this baseline measure are presented separately for each group.
|
—
|
29.60 units per liter
STANDARD_DEVIATION 19.88 • n=15 Participants • The results for this baseline measure are presented separately for each group.
|
|
Level of liver enzymes at baseline for low-dosage group
Baseline AST
|
27.53 units per liter
STANDARD_DEVIATION 10.00 • n=15 Participants • The results for this baseline measure are presented separately for each group.
|
—
|
27.53 units per liter
STANDARD_DEVIATION 10.00 • n=15 Participants • The results for this baseline measure are presented separately for each group.
|
|
Level of liver enzymes at baseline for high-dosage group
Baseline ALT
|
—
|
38.29 units per liter
STANDARD_DEVIATION 22.43 • n=14 Participants • The results for this baseline measure are presented separately for each group.
|
38.29 units per liter
STANDARD_DEVIATION 22.43 • n=14 Participants • The results for this baseline measure are presented separately for each group.
|
|
Level of liver enzymes at baseline for high-dosage group
Baseline AST
|
—
|
28.50 units per liter
STANDARD_DEVIATION 11.39 • n=14 Participants • The results for this baseline measure are presented separately for each group.
|
28.50 units per liter
STANDARD_DEVIATION 11.39 • n=14 Participants • The results for this baseline measure are presented separately for each group.
|
PRIMARY outcome
Timeframe: Day 28Population: One patient in the high-dosage group withdrew before providing any evaluable data
Levels of the liver enzymes alanine aminotransferase (ALT) and aspartate aminotransferase (AST) will be assessed throughout the study to determine if any patients have post-dose increases that are considered to be a safety concern. The criteria for being considered a safety concern are meeting one of the following: * For a patient whose level was within the normal range at baseline, the criterion is reaching a value of 5 times the upper limit of normal (ULN) * For a patient whose level was above the ULN at baseline, the criterion is reaching either 5 times the baseline value or 10 x ULN
Outcome measures
| Measure |
Low Dosage
n=15 Participants
Evaluable patients who received the lower dosage of deferiprone
|
High Dosage
n=14 Participants
Evaluable patients who received the higher dosage of deferiprone
|
|---|---|---|
|
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Patients with elevated ALT of clinical concern
|
0 Participants
|
0 Participants
|
|
The Percentage of Patients in Each Treatment Group Who Experience Post-dose Increases in Liver Enzyme Levels That Are Considered a Safety Concern.
Patients with elevated AST of clinical concern
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Day 28Patients will be asked to report any events of GI distress during the study, such as nausea, vomiting, diarrhea, abdominal pain, and dyspepsia.
Outcome measures
| Measure |
Low Dosage
n=15 Participants
Evaluable patients who received the lower dosage of deferiprone
|
High Dosage
n=14 Participants
Evaluable patients who received the higher dosage of deferiprone
|
|---|---|---|
|
The Percentage of Patients in Each Treatment Group Who Report Post-dose Occurrences of Gastrointestinal (GI) Distress.
|
3 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: Day 28Population: One of the evaluable patients withdrew before completing the questionnaire
At the end of the study, patients will complete a questionnaire to indicate which formulation they prefer.
Outcome measures
| Measure |
Low Dosage
n=15 Participants
Evaluable patients who received the lower dosage of deferiprone
|
High Dosage
n=13 Participants
Evaluable patients who received the higher dosage of deferiprone
|
|---|---|---|
|
The Percentage of Patients in Each Group Who Indicate That They Prefer the Deferiprone DR Formulation Over the Immediate-release Formulation.
|
13 Participants
|
13 Participants
|
Adverse Events
Low Dosage
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
High Dosage
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Low Dosage
n=15 participants at risk
Evaluable patients who received the lower dosage of deferiprone
|
High Dosage
n=14 participants at risk
Evaluable patients who received the higher dosage of deferiprone
|
|---|---|---|
|
Ear and labyrinth disorders
Ear pain
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Eye disorders
Blepharitis
|
0.00%
0/15 • Baseline to Day 28
|
14.3%
2/14 • Number of events 2 • Baseline to Day 28
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/15 • Baseline to Day 28
|
21.4%
3/14 • Number of events 3 • Baseline to Day 28
|
|
Gastrointestinal disorders
Dyspepsia
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Gastrointestinal disorders
Nausea
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
General disorders
Pyrexia
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Injury, poisoning and procedural complications
Joint injury
|
13.3%
2/15 • Number of events 2 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Investigations
Alanine aminotransferase increased
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 2 • Baseline to Day 28
|
|
Investigations
Neutrophil count decreased
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
20.0%
3/15 • Number of events 5 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Nervous system disorders
Headache
|
20.0%
3/15 • Number of events 4 • Baseline to Day 28
|
21.4%
3/14 • Number of events 5 • Baseline to Day 28
|
|
Nervous system disorders
Migraine
|
6.7%
1/15 • Number of events 2 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Nervous system disorders
Sciatica
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Psychiatric disorders
Anxiety
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
|
Renal and urinary disorders
Renal colic
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/15 • Baseline to Day 28
|
7.1%
1/14 • Number of events 1 • Baseline to Day 28
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.7%
1/15 • Number of events 1 • Baseline to Day 28
|
0.00%
0/14 • Baseline to Day 28
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60