Trial Outcomes & Findings for Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias (NCT NCT02443545)
NCT ID: NCT02443545
Last Updated: 2024-01-10
Results Overview
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
134 participants
Primary outcome timeframe
From the first day of the study until the last study visit (Week 104 or early termination)
Results posted on
2024-01-10
Participant Flow
Participant milestones
| Measure |
Group 1: Deferiprone 3 Years
Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411 (NCT02041299), and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study)..
Deferiprone
|
Group 2: Deferiprone 2 Years
Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411 (NCT02041299), and hence will receive deferiprone for 2 years (both of them in the extension study).
Deferiprone
|
|---|---|---|
|
Overall Study
STARTED
|
89
|
45
|
|
Overall Study
COMPLETED
|
50
|
26
|
|
Overall Study
NOT COMPLETED
|
39
|
19
|
Reasons for withdrawal
| Measure |
Group 1: Deferiprone 3 Years
Patients in this group are those who were randomized to the deferiprone arm in study LA38-0411 (NCT02041299), and hence will receive deferiprone for a total of 3 years (1 year in the initial study plus 2 years in the extension study)..
Deferiprone
|
Group 2: Deferiprone 2 Years
Patients in this group are those who were randomized to the deferoxamine arm in study LA38-0411 (NCT02041299), and hence will receive deferiprone for 2 years (both of them in the extension study).
Deferiprone
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Lost to Follow-up
|
0
|
2
|
|
Overall Study
Physician Decision
|
3
|
0
|
|
Overall Study
Protocol Violation
|
8
|
1
|
|
Overall Study
Pregnancy
|
1
|
1
|
|
Overall Study
Withdrawal by Subject
|
6
|
3
|
|
Overall Study
Termination of study
|
20
|
11
|
Baseline Characteristics
Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias
Baseline characteristics by cohort
| Measure |
Deferiprone
n=134 Participants
All patients in this study received deferiprone
|
|---|---|
|
Age, Continuous
|
16.2 years
STANDARD_DEVIATION 8.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
53 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
81 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
133 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
20 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
114 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Saudi Arabia
|
5 participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
2 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
14 participants
n=5 Participants
|
|
Region of Enrollment
Egypt
|
109 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
4 participants
n=5 Participants
|
|
Liver iron concentration at baseline
|
14.93 mg iron per gram of liver dry weight
STANDARD_DEVIATION 7.61 • n=5 Participants
|
|
Cardiac iron at baseline
|
32.69 milliseconds
STANDARD_DEVIATION 17.66 • n=5 Participants
|
|
Serum ferritin level at baseline
|
3894 micrograms per liter
STANDARD_DEVIATION 2591 • n=5 Participants
|
PRIMARY outcome
Timeframe: From the first day of the study until the last study visit (Week 104 or early termination)Population: Safety population (all enrolled patients who took at least one dose of study drug)
Number of patients with at least one adverse event (AE) of any type; number of patients with at least one serious adverse event, and number of patients who withdrew from the study due to an AE
Outcome measures
| Measure |
Deferiprone
n=134 Participants
All patients in this study received deferiprone
|
|---|---|
|
Number of Patients With Adverse Events
Number of patients with any type of adverse event
|
104 Participants
|
|
Number of Patients With Adverse Events
Number of patients with a serious adverse event
|
35 Participants
|
|
Number of Patients With Adverse Events
Number of patients who discontinued due to an AE
|
2 Participants
|
SECONDARY outcome
Timeframe: One year, two years, and three years after the start of deferiprone therapyPopulation: Intent-to-Treat (ITT) population (patients who received at least one dose of study drug and had a baseline and at least one post-baseline efficacy assessment). LIC data for one year of exposure to deferiprone were obtained from 129 patients, data for two years from 112 patients, and data for three years from 59 patients.
LIC was measured by MRI, in units of mg of iron per gram of liver (dry weight). The change from baseline in LIC was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Outcome measures
| Measure |
Deferiprone
n=129 Participants
All patients in this study received deferiprone
|
|---|---|
|
Change From Baseline in Liver Iron Concentration (LIC)
Change from baseline in LIC after 1 year
|
-2.64 mg of iron per gram of liver dry weight
Standard Deviation 4.64
|
|
Change From Baseline in Liver Iron Concentration (LIC)
Change from baseline in LIC after 2 years
|
-3.91 mg of iron per gram of liver dry weight
Standard Deviation 6.38
|
|
Change From Baseline in Liver Iron Concentration (LIC)
Change from baseline in LIC after 3 years
|
-6.64 mg of iron per gram of liver dry weight
Standard Deviation 7.72
|
SECONDARY outcome
Timeframe: One year, two years, and three years after the start of deferiprone therapyPopulation: ITT population. Cardiac MRI T2\* data for one year of exposure to deferiprone were obtained from 121 patients, data for two years from 110 patients, and data for three years from 58 patients.
The change from baseline in cardiac MRI T2\* was determined for three different periods of exposure to deferiprone: one year, two years, and three years
Outcome measures
| Measure |
Deferiprone
n=121 Participants
All patients in this study received deferiprone
|
|---|---|
|
Change From Baseline in Cardiac MRI T2*
Change from baseline in cardiac iron after 1 year
|
1.02 milliseconds
Standard Deviation 19.65
|
|
Change From Baseline in Cardiac MRI T2*
Change from baseline in cardiac iron after 2 years
|
1.00 milliseconds
Standard Deviation 21.18
|
|
Change From Baseline in Cardiac MRI T2*
Change from baseline in cardiac iron after 3 years
|
0.98 milliseconds
Standard Deviation 24.53
|
SECONDARY outcome
Timeframe: One year, two years, and three years after the start of deferiprone therapyPopulation: ITT population. Serum ferritin data for one year of exposure to deferiprone were obtained from 125 patients, data for two years from 96 patients, and data for three years from 55 patients.
The change from baseline in serum ferritin (SF) was determined for three different periods of exposure to deferiprone: one year, two years, and three years.
Outcome measures
| Measure |
Deferiprone
n=125 Participants
All patients in this study received deferiprone
|
|---|---|
|
Change From Baseline in Serum Ferritin
Change from baseline in SF after 1 year
|
-1 micrograms per liter
Standard Deviation 1986
|
|
Change From Baseline in Serum Ferritin
Change from baseline in SF after 2 years
|
-771 micrograms per liter
Standard Deviation 2171
|
|
Change From Baseline in Serum Ferritin
Change from baseline in SF after 3 years
|
-1016 micrograms per liter
Standard Deviation 3617
|
Adverse Events
Deferiprone
Serious events: 35 serious events
Other events: 99 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Deferiprone
n=134 participants at risk
All patients in this study received deferiprone
|
|---|---|
|
Blood and lymphatic system disorders
Agranulocytosis
|
1.5%
2/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
|
Blood and lymphatic system disorders
Neutropenia
|
9.0%
12/134 • Number of events 15 • Baseline to end of study (up to 2 years)
|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
14.2%
19/134 • Number of events 42 • Baseline to end of study (up to 2 years)
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Endocrine disorders
Adrenal insufficiency
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Gastrointestinal disorders
Gastritis
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Gastrointestinal disorders
Nausea
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Gastrointestinal disorders
Vomiting
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
General disorders
Generalised oedema
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
General disorders
Pain
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
General disorders
Pyrexia
|
3.7%
5/134 • Number of events 6 • Baseline to end of study (up to 2 years)
|
|
Hepatobiliary disorders
Cholecystitis chronic
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Device related sepsis
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Osteomyelitis
|
0.75%
1/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Pneumonia
|
1.5%
2/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Propionibacterium infection
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Sepsis
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Staphylococcal infection
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Upper respiratory tract infection
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Investigations
Transaminases increased
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
1.5%
2/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Nervous system disorders
Hypoaesthesia
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Product Issues
Device malfunction
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.75%
1/134 • Number of events 3 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Hypoventilation
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Surgical and medical procedures
Cholecystectomy
|
2.2%
3/134 • Number of events 3 • Baseline to end of study (up to 2 years)
|
|
Surgical and medical procedures
Medical device repositioning
|
0.75%
1/134 • Number of events 1 • Baseline to end of study (up to 2 years)
|
|
Surgical and medical procedures
Splenectomy
|
1.5%
2/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
|
Vascular disorders
Hypotension
|
1.5%
2/134 • Number of events 2 • Baseline to end of study (up to 2 years)
|
Other adverse events
| Measure |
Deferiprone
n=134 participants at risk
All patients in this study received deferiprone
|
|---|---|
|
Blood and lymphatic system disorders
Sickle cell anaemia with crisis
|
12.7%
17/134 • Number of events 39 • Baseline to end of study (up to 2 years)
|
|
Gastrointestinal disorders
Abdominal pain
|
19.4%
26/134 • Number of events 44 • Baseline to end of study (up to 2 years)
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
9/134 • Number of events 12 • Baseline to end of study (up to 2 years)
|
|
General disorders
Pyrexia
|
24.6%
33/134 • Number of events 50 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Nasopharyngitis
|
12.7%
17/134 • Number of events 28 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Pharyngitis
|
7.5%
10/134 • Number of events 15 • Baseline to end of study (up to 2 years)
|
|
Infections and infestations
Upper respiratory tract infection
|
6.7%
9/134 • Number of events 12 • Baseline to end of study (up to 2 years)
|
|
Investigations
Neutrophil count decreased
|
11.9%
16/134 • Number of events 29 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.5%
10/134 • Number of events 15 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
12.7%
17/134 • Number of events 51 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
26.1%
35/134 • Number of events 97 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
6.7%
9/134 • Number of events 11 • Baseline to end of study (up to 2 years)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
13.4%
18/134 • Number of events 33 • Baseline to end of study (up to 2 years)
|
|
Nervous system disorders
Headache
|
9.0%
12/134 • Number of events 23 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.2%
7/134 • Number of events 10 • Baseline to end of study (up to 2 years)
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
13.4%
18/134 • Number of events 28 • Baseline to end of study (up to 2 years)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60