Trial Outcomes & Findings for Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases (NCT NCT00115349)
NCT ID: NCT00115349
Last Updated: 2018-03-01
Results Overview
The primary outcome variable is change in left ventricular ejection fraction (blood ejected from the heart into the body) as measured by MRI from baseline to one year. The unit of primary outcome (left ventricular ejection fraction) is the percent of the blood in left ventricle.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
Baseline to one year
Results posted on
2018-03-01
Participant Flow
First patient enrolled in August, 2005 and study closed due to low enrollment in June, 2008. 8 participating sites.
Patients were evaluated for eligibility based on the following criterial. If not eligible, they were not randomized to a treatment arm.
Participant milestones
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
9
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
11
|
9
|
Reasons for withdrawal
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
|---|---|---|
|
Overall Study
Study closed due to low enrollment.
|
11
|
9
|
Baseline Characteristics
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
Baseline characteristics by cohort
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
n=11 Participants
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
n=9 Participants
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
11 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
26.5 years
STANDARD_DEVIATION 5.1 • n=5 Participants
|
25.8 years
STANDARD_DEVIATION 7.6 • n=7 Participants
|
26.15 years
STANDARD_DEVIATION 6.18 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=5 Participants
|
4 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
Region of Enrollment
Lebanon
|
2 participants
n=5 Participants
|
2 participants
n=7 Participants
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to one yearThe primary outcome variable is change in left ventricular ejection fraction (blood ejected from the heart into the body) as measured by MRI from baseline to one year. The unit of primary outcome (left ventricular ejection fraction) is the percent of the blood in left ventricle.
Outcome measures
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
n=11 Participants
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
n=9 Participants
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
|---|---|---|
|
Change in Left Ventricular Ejection Fraction (LVEF).
|
7.2 Percent of the blood in left ventricle
Standard Error 1.9
|
6.3 Percent of the blood in left ventricle
Standard Error 3.3
|
SECONDARY outcome
Timeframe: one yearOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: one yearOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: one yearOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: one yearOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: continuousOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: continousOutcome measures
Outcome data not reported
Adverse Events
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
Serious events: 6 serious events
Other events: 8 other events
Deaths: 0 deaths
Deferoxamine (DFO) + Monotherapy
Serious events: 3 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
n=11 participants at risk
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
n=9 participants at risk
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
|---|---|---|
|
Hepatobiliary disorders
Abnormal LFTs
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Cardiac disorders
Congestive heart failure
|
9.1%
1/11 • Number of events 1 • 1 year
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Blood and lymphatic system disorders
Clot
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Cellulitis
|
0.00%
0/11 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Meningitis (unkown viral vs. bacterial)
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Renal and urinary disorders
Renal Disease
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Cardiac disorders
Palpitation
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 2 • 1 year
|
|
Infections and infestations
high grade fever and chills
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Hepatobiliary disorders
Abdominal Pain
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Pneumonia
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
Splenectomy
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Cardiac disorders
Arrhythmia
|
18.2%
2/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Metabolism and nutrition disorders
Syncope
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
General disorders
Watery diarrhea & hypotension
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Eye disorders
Retinal toxicity- drug induced
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Infections and infestations
Central venous line infection
|
9.1%
1/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
Other adverse events
| Measure |
Deferoxamine (DFO) and Deferiprone (L1) Combination Therapy
n=11 participants at risk
DFO + L1 DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
L1 Administered daily at 75 mg/kg in 3 divided doses taken orally and timed so that 2 of 3 doses will be simultaneous with DFO infusion.
|
Deferoxamine (DFO) + Monotherapy
n=9 participants at risk
DFO + Placebo DFO Administered daily at 50-60 mg/kg for 12-24 hr/day 7 days a week either subcutaneous or intravenous.
Placebo Administered orally three times daily.
|
|---|---|---|
|
Blood and lymphatic system disorders
Abnormal LFTs
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Bilerateral Hip Pain
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Chest Ache
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Social circumstances
Common Cold
|
9.1%
1/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
General disorders
Dizziness
|
18.2%
2/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Gastrointestinal disorders
Dry mouth
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Skin and subcutaneous tissue disorders
Epistaxis
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Pregnancy, puerperium and perinatal conditions
Fanconi
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
General disorders
Fever
|
18.2%
2/11 • Number of events 2 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Social circumstances
Kicked in chest
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
General disorders
Low Grade Fever and Sore Throay
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Gastrointestinal disorders
Nausea
|
27.3%
3/11 • Number of events 4 • 1 year
|
0.00%
0/9 • 1 year
|
|
General disorders
Nausea and Vomiting
|
18.2%
2/11 • Number of events 2 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Blood and lymphatic system disorders
Neutropenia
|
9.1%
1/11 • Number of events 2 • 1 year
|
11.1%
1/9 • Number of events 2 • 1 year
|
|
Nervous system disorders
Otitis media
|
18.2%
2/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Cardiac disorders
Palpitations with dizziness
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Injury, poisoning and procedural complications
Phlebotic pain at blood infusion
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Sternocleidomastoid muscle spasm
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
9.1%
1/11 • Number of events 1 • 1 year
|
22.2%
2/9 • Number of events 2 • 1 year
|
|
Infections and infestations
Strep Throat
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infection
|
18.2%
2/11 • Number of events 4 • 1 year
|
0.00%
0/9 • 1 year
|
|
Vascular disorders
Vertigo
|
18.2%
2/11 • Number of events 2 • 1 year
|
0.00%
0/9 • 1 year
|
|
Musculoskeletal and connective tissue disorders
Vomiting
|
9.1%
1/11 • Number of events 1 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Allergic transfusion reactions hives
|
9.1%
1/11 • Number of events 1 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
sore throat and cough
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
stomach flu
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Injury, poisoning and procedural complications
tooth ache
|
9.1%
1/11 • Number of events 1 • 1 year
|
0.00%
0/9 • 1 year
|
|
Psychiatric disorders
Anxiety attack
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 2 • 1 year
|
|
Cardiac disorders
Heart palpatations
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
stomach discomfort
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Renal and urinary disorders
Urinary Tract Infection
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Shortness of Breath/fatigue
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Injury, poisoning and procedural complications
Ankle pain
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
|
Gastrointestinal disorders
Abdominal Bloating
|
0.00%
0/11 • 1 year
|
11.1%
1/9 • Number of events 1 • 1 year
|
Additional Information
John Porter, MD, Principal Investigator
UCL Cancer Institute
Phone: +011 (44) 207 679 6224
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place