Trial Outcomes & Findings for Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study (NCT NCT01326845)
NCT ID: NCT01326845
Last Updated: 2017-03-03
Results Overview
Study was prematurely terminated and not powered for efficacy. Frequency of GI AEs during the overall study period is available in the AE tables reported in the safety section.
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
12 participants
Primary outcome timeframe
3 months
Results posted on
2017-03-03
Participant Flow
Participant milestones
| Measure |
Deferasirox am
Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food
|
Deferasirox pm
Deferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal
|
|---|---|---|
|
Overall Study
STARTED
|
7
|
5
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
7
|
5
|
Reasons for withdrawal
| Measure |
Deferasirox am
Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food
|
Deferasirox pm
Deferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal
|
|---|---|---|
|
Overall Study
Administrative Problems
|
5
|
2
|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Misisng
|
1
|
3
|
Baseline Characteristics
Myelodysplastic Syndrome (MDS) Gastrointestinal (GI) Tolerability Study
Baseline characteristics by cohort
| Measure |
Deferasirox am
n=7 Participants
Deferasirox 20 mg/kg/day taken in the morning, 30 minutes before food
|
Deferasirox pm
n=5 Participants
Deferasirox 20 mg/kg/day taken in the evening, no less than 2 hours after the last food intake or at least 30 minutes before the evening meal
|
Total
n=12 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
69.7 Years
STANDARD_DEVIATION 7.5 • n=99 Participants
|
71.6 Years
STANDARD_DEVIATION 8.65 • n=107 Participants
|
70.5 Years
STANDARD_DEVIATION 7.67 • n=206 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
6 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 3 monthsStudy was prematurely terminated and not powered for efficacy. Frequency of GI AEs during the overall study period is available in the AE tables reported in the safety section.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 6 monthsStudy was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: months 3 and 6.Study was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: months 3 and 6.Study was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: months 3 and 6Study was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: months 3 and 6Study was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months, 6 monthsStudy was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months, 6 monthsOutcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 3 months, 6 monthsStudy was prematurely terminated and not powered for efficacy.
Outcome measures
Outcome data not reported
Adverse Events
ICL am
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
ICL pm
Serious events: 2 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ICL am
n=7 participants at risk
ICL am
|
ICL pm
n=5 participants at risk
ICL pm
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Leukaemia
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Vascular disorders
Jugular vein thrombosis
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
Other adverse events
| Measure |
ICL am
n=7 participants at risk
ICL am
|
ICL pm
n=5 participants at risk
ICL pm
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
28.6%
2/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Blood and lymphatic system disorders
Neutropenia
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Gastrointestinal disorders
Nausea
|
28.6%
2/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Gastrointestinal disorders
Vomiting
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
General disorders
Asthenia
|
28.6%
2/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
General disorders
Oedema peripheral
|
28.6%
2/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Infections and infestations
Lyme disease
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Infections and infestations
Staphylococcal infection
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
Infections and infestations
Upper respiratory tract infection
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Investigations
Alanine aminotransferase increased
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Investigations
Aspartate aminotransferase increased
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Investigations
Blood alkaline phosphatase increased
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Investigations
Blood creatinine increased
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Investigations
Gamma-glutamyltransferase increased
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Metabolism and nutrition disorders
Decreased appetite
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/7 • 6 months
|
20.0%
1/5 • 6 months
|
|
Renal and urinary disorders
Renal impairment
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
14.3%
1/7 • 6 months
|
0.00%
0/5 • 6 months
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee Principal Investigators are NOT employed by the organization sponsoring the study. Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial.
- Publication restrictions are in place
Restriction type: OTHER