Trial Outcomes & Findings for A Rollover Study to Provide Continued Treatment With Eltrombopag (NCT NCT01957176)

NCT ID: NCT01957176

Last Updated: 2022-12-30

Results Overview

The distribution of adverse events was done via the analysis of frequencies for Adverse Events (AEs) and Serious Adverse Events (SAEs), through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

22 participants

Primary outcome timeframe

From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.

Results posted on

2022-12-30

Participant Flow

22 subjects were enrolled in 14 centers in 12 countries (Belgium, China, France, Greece, Hong Kong, Ireland, Korea, Republic of., Netherlands, Peru, Poland, Romania, Tunisia).

Participant milestones

Participant milestones
Measure	Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects) All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects) All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.
Overall Study STARTED	8	14
Overall Study COMPLETED	3	13
Overall Study NOT COMPLETED	5	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects) All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects) All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.
Overall Study Adverse Event	2	0
Overall Study Physician Decision	2	1
Overall Study Withdrawal by Subject	1	0

Baseline Characteristics

A Rollover Study to Provide Continued Treatment With Eltrombopag

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects) n=8 Participants All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects) n=14 Participants All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Total n=22 Participants Total of all reporting groups
Age, Continuous	73.8 Years STANDARD_DEVIATION 7.57 • n=5 Participants	59.5 Years STANDARD_DEVIATION 13.18 • n=7 Participants	64.7 Years STANDARD_DEVIATION 13.26 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants	12 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Race/Ethnicity, Customized American Indian or Alaskan Native	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian - East Asian Heritage	1 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized White - Arabic/North African Heritage	0 Participants n=5 Participants	6 Participants n=7 Participants	6 Participants n=5 Participants
Race/Ethnicity, Customized White - White/Caucasian/European Heritage	7 Participants n=5 Participants	1 Participants n=7 Participants	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months.

Population: All treated subjects (ATS): All subjects who received at least one dose of eltrombopag on this study were included.

Outcome measures

Outcome measures
Measure	Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects) n=8 Participants All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects) n=14 Participants All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.
Number of Participants With Adverse Events (AEs) Adverse Events (AEs)	7 Participants	12 Participants
Number of Participants With Adverse Events (AEs) Serious Adverse Events (SAEs)	6 Participants	7 Participants

Adverse Events

Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects)

Serious events: 6 serious events

Other events: 7 other events

Deaths: 3 deaths

Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects)

Serious events: 7 serious events

Other events: 12 other events

Deaths: 2 deaths

All Subjects

Serious events: 13 serious events

Other events: 19 other events

Deaths: 5 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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 the pooled data (i.e., data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER

Measure	Cohort A (Myelodysplastic Syndrome (MDS)/ Acute Myeloid Leukemia (AML) Adult Subjects) n=8 participants at risk All subjects in this cohort received eltrombopag (ELT) at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 50 to 300 mg once daily (OD) for subjects of non-East Asian heritage. The dose ranges for subjects of East Asian heritage (i.e., Japanese, Chinese, Taiwanese, Thai and Korean) were 25 to 150 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	Cohort B (Idiopathic Thrombocytopenic Purpura (ITP) Adult Subjects) n=14 participants at risk All subjects in this cohort received ELT at the dose that they were receiving at the time of the transition visit, except in the case where the subject required a dose modification. The range of doses of ELT that were used in this cohort were from 12.5 to 75 mg. Dose adjustments (if required) were done depending on each subject's platelet counts.	All Subjects n=22 participants at risk All enrolled subjects in the study
Blood and lymphatic system disorders Anaemia	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Cardiac disorders Atrial fibrillation	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Gastrointestinal disorders Ascites	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
General disorders Multiple organ dysfunction syndrome	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
General disorders Pyrexia	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Hepatobiliary disorders Cholecystitis acute	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Infections and infestations Appendicitis	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Infections and infestations Encephalitis	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Infections and infestations Encephalitis viral	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Infections and infestations Pneumonia	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	9.1% 2/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Infections and infestations Sepsis	25.0% 2/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	9.1% 2/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Investigations Alanine aminotransferase increased	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Metabolism and nutrition disorders Hypernatraemia	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Metabolism and nutrition disorders Hypocalcaemia	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Metabolism and nutrition disorders Hypoglycaemia	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Endometrial cancer	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Nervous system disorders Cerebral haemorrhage	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Nervous system disorders Haemorrhagic stroke	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Nervous system disorders Subdural hygroma	12.5% 1/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	0.00% 0/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/8 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	7.1% 1/14 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.	4.5% 1/22 • From the time of transition visit until 30 days after last dose of study treatment, assessed up to approximately 100 months. Any sign or symptom that occurs during the treatment period plus 30 days post-treatment.