Trial Outcomes & Findings for LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS) (NCT NCT01034657)

Last Updated: 2017-08-11

Results Overview

HI-E was assessed according to the modified international working group (IWG) criteria for HI. Erythroid response (pretreatment, \<11 g/dL): Hgb increase by ≥ 1.5 g/dL, relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk, and only RBC transfusions given for a Hgb of ≤ 9.0 g/dL pretreatment were counted in the RBC transfusion response evaluation; Platelet response (pretreatment, \< 100 x 109/L): absolute increase of ≥ 30 x 109/L for participants starting with \> 20 x 109/L and platelets Increase from \< 20 x 109/L to \> 20 x 109/L and by at least 100%; Neutrophil response (pretreatment, \< 1.0 x 109/L): at least 100% increase and an absolute increase \> 0.5 x 109/L; Progression or relapse after HI: At least 1 of the following: At least 50% decrement from maximum response levels in granulocytes or platelets, reduction in Hgb by ≥1.5 g/dL, or transfusion dependence.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

34 participants

Primary outcome timeframe

16 weeks

Results posted on

2017-08-11

Participant Flow

The study was divided into a core phase and a randomized phase. The core phase had an open-label single arm study design. The randomized phase was open-label with two parallel treatment arms for participants with stable disease. Participants with Hematological response of the erythropoietin system remained on single agent oral LBH589.

Participants with stable disease were eligible for randomization to single agent LBH589 or LBH589 + epoetin alfa. Participants with progressive disease were discontinued. Seven participants who completed the core phase withdrew before the randomization phase.

Participant milestones

Participant milestones
Measure	LBH589 During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Core Phase STARTED	34	0	0
Core Phase COMPLETED	20	0	0
Core Phase NOT COMPLETED	14	0	0
Randomized Phase STARTED	6	6	1
Randomized Phase COMPLETED	1	0	0
Randomized Phase NOT COMPLETED	5	6	1

Reasons for withdrawal

Reasons for withdrawal
Measure	LBH589 During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Core Phase Withdrawal by Subject	1	0	0
Core Phase Lack of Efficacy	2	0	0
Core Phase Abnormal laboratory value	1	0	0
Core Phase Adverse Event	8	0	0
Core Phase Protocol Violation	2	0	0
Randomized Phase Withdrawal by Subject	0	2	0
Randomized Phase Lack of Efficacy	5	1	0
Randomized Phase Abnormal laboratory value	0	1	0
Randomized Phase Adverse Event	0	2	1

Baseline Characteristics

LBH589 Alone or in Combination With Erythropoietin Stimulating Agents (ESA) in Patients With Low or Int-1 Risk Myelodysplastic Syndromes (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LBH589 n=34 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.
Age, Continuous	65.4 Years STANDARD_DEVIATION 7.70 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants
Sex: Female, Male Male	24 Participants n=5 Participants

PRIMARY outcome

Timeframe: 16 weeks

Population: Participants from the core phase, who had valid response data, were analyzed.

Outcome measures

Outcome measures
Measure	LBH589 n=20 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Percentage of Participants With Hematological Response of the Erythropoetic System (HI-E) - Core Phase	0.0 Percentage of participants	—	—

SECONDARY outcome

Timeframe: 32 weeks, 52 weeks

Population: Participants from the randomized phase, who had valid response data, were analyzed.

Outcome measures

Outcome measures
Measure	LBH589 n=6 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa n=6 Participants During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 Participants Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Percentage of Participants With HI-E - Randomized Phase 32 weeks (n=5,1,1)	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants
Percentage of Participants With HI-E - Randomized Phase 52 weeks (n=4,3,1)	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants

SECONDARY outcome

Timeframe: 16 weeks

Population: Participants from the core phase, who had valid data, were analyzed.

Objective response (complete remission (CR) + partial remission (PR) and HI-platelet (HI-P) response + HI-neutrophil (HI-N) response) was assessed according to the modified IWG criteria: CR bone marrow with 5% myeloblasts with normal maturation of al cell lines (persistent dysplasia is noted) and peripheral blood with Hgb \>= 11 g/dL platelets \>=100 X 10\^9/L, neutrophils \>= 1.0 x 10\^9/L and blasts 0%. PR = All CR if abnormal before treatment except bone marrow blasts decreased by\>=50% over pretreatment but still \>5% (ellularity and morphology not relevant). HI-P (pretreatment, \< 100 x 109/L) = absolute increase of ≥ 30 x 109/L for participants starting with \> 20 x 109/L and platelets Increase from \< 20 x 109/L to \> 20 x 109/L and by at least 100%; HI-N (pretreatment, \< 1.0 x 109/L) = at least 100% increase and an absolute increase \> 0.5 x 10\^9/L.

Outcome measures

Outcome measures
Measure	LBH589 n=20 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Percentage of Participants With Objective Response During Core Phase	0.0 Percentage of participants	—	—

SECONDARY outcome

Timeframe: 32 weeks, 48 weeks

Population: Participants from the randomized phase, who had valid data, were analyzed.

Outcome measures

Outcome measures
Measure	LBH589 n=6 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa n=6 Participants During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 Participants Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Percentage of Participants With Objective Response During the Randomized Phase Week 32 ( n=5,1,1)	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants
Percentage of Participants With Objective Response During the Randomized Phase Week 48 (n=6,5,1)	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants

SECONDARY outcome

Timeframe: baseline

Population: All participants from the core phase were analyzed.

The IPSS score values were calculated based on the results of bone marrow analysis. A score value of 0 has bone marrow blast \<5%, karyotype of normal, sole: -Y, del 5Q, del 20q and cytopenias (lineages affected) of 0 to 1. Score value of 0.5 has 5-10 bone marrow blasts, karyotype of Others and cytopenias of 2 to 3. A score value of 1.0 has complex \>= 3 chromosomal abnormalities and/or chromosome 7 anomalies. A score of 1.5 has 11-20 bone marrow blasts and a score of 2.0 has 21-30 bone marrow blasts. The prognostic score is determined by the sum of the single scoring values. The risk groups are determined as follows: Low = 0 points (5.7 years of median survival); intermediate -1 (INT-1) = 0.5-1.0 points (3.5 years of median survival); INT-2 = 1.5-2.0 points (1.2 years of median survival); and high \>=2.5 points (6 months of median survival).

Outcome measures

Outcome measures
Measure	LBH589 n=34 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Frequency Distribution of IPSS Score Status - Core Phase Baseline (core phase), Low	32.4 Percentage of participants	—	—
Frequency Distribution of IPSS Score Status - Core Phase Baseline (core phase),, INT-1	67.6 Percentage of participants	—	—
Frequency Distribution of IPSS Score Status - Core Phase Baseline (core phase),, INT-2	0.0 Percentage of participants	—	—
Frequency Distribution of IPSS Score Status - Core Phase Baseline (core phase), High	0.0 Percentage of participants	—	—

SECONDARY outcome

Timeframe: 52 weeks

Population: Only participants from the randomized phase, who had values at week 52, were included in the analysis.

Outcome measures

Outcome measures
Measure	LBH589 n=6 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa n=6 Participants During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 Participants Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Frequency Distribution of IPSS Score Status - Randomized Phase Week 52 (randomized phase), Low	0.0 Percentage of participants	0.0 Percentage of participants	100.0 Percentage of participants
Frequency Distribution of IPSS Score Status - Randomized Phase Week 52 (randomized phase), , INT-1	16.7 Percentage of participants	16.7 Percentage of participants	0.0 Percentage of participants
Frequency Distribution of IPSS Score Status - Randomized Phase Week 52 (randomized phase), INT-2	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants
Frequency Distribution of IPSS Score Status - Randomized Phase Week 52 (randomized phase), HIgh	0.0 Percentage of participants	0.0 Percentage of participants	0.0 Percentage of participants

SECONDARY outcome

Timeframe: baseline

Population: All participants from the core phase were analyzed.

Outcome measures

Outcome measures
Measure	LBH589 n=34 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Mean Single Scoring Values of the IPSS - Core Phase	0.43 units on a scale Standard Deviation 0.351	—	—

SECONDARY outcome

Timeframe: 52 weeks

Population: Participants from the randomized phase, who had values at week 52, were included in the analysis.

Outcome measures

Outcome measures
Measure	LBH589 n=1 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa n=1 Participants During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 Participants Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Mean Single Scoring Values of the IPSS - Randomized Phase	1.0 units on a scale Standard Deviation NA Standard deviation cannot be calculated because only 1 participant was analyzed.	1.0 units on a scale Standard Deviation NA Standard deviation cannot be calculated because only 1 participant was analyzed.	0.0 units on a scale Standard Deviation NA Standard deviation cannot be calculated because only 1 participant was analyzed.

SECONDARY outcome

Timeframe: 48 weeks

Population: All participants from the core phase were analyzed.

OS was defined as the time from start of treatment to death from any cause.

Outcome measures

Outcome measures
Measure	LBH589 n=34 Participants During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 + Epoetin Alfa During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Overall Survival (OS) - Overall Period	NA months The median could not be calculated because only 1 event occurred.	—	—

Adverse Events

LBH589 - Core Phase

Serious events: 11 serious events

Other events: 34 other events

Deaths: 0 deaths

LBH589 - Randomized Phase

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

LBH589 + ESA - Randomized Phase

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Not Randomized

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	LBH589 - Core Phase n=34 participants at risk During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 - Randomized Phase n=6 participants at risk During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	LBH589 + ESA - Randomized Phase n=6 participants at risk During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 participants at risk Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Cardiac disorders Coronary artery stenosis	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Cardiac disorders Myocardial infarction	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Ear and labyrinth disorders Sudden hearing loss	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Eye disorders Optic ischaemic neuropathy	0.00% 0/34	0.00% 0/6	0.00% 0/6	100.0% 1/1
Gastrointestinal disorders Diarrhoea	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Faecaloma	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Vomiting	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
General disorders Chest pain	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
General disorders Impaired healing	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
General disorders Pyrexia	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Abscess limb	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Infections and infestations Lymph node abscess	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Pneumonia	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Postoperative wound infection	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Sepsis	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Septic shock	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Staphylococcal infection	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Infections and infestations Subcutaneous abscess	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Metabolism and nutrition disorders Dehydration	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Intervertebral disc protrusion	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Rhabdomyolysis	0.00% 0/34	0.00% 0/6	0.00% 0/6	100.0% 1/1
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Chronic myelomonocytic leukaemia	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Renal and urinary disorders Glomerulonephritis	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Renal and urinary disorders Haematuria	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Renal and urinary disorders Urinary incontinence	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Dyspnoea	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Skin and subcutaneous tissue disorders Alopecia	2.9% 1/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Vascular disorders Phlebitis	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Vascular disorders Vascular pseudoaneurysm	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1

Other adverse events

Other adverse events
Measure	LBH589 - Core Phase n=34 participants at risk During the core phase, all participants received oral LBH589 40 mg (30 mg after a protocol amendment) for 4 months. During the randomization phase, participants with hematological improvement of the erythropoetic system (HI-E) and participants with stable disease, who were randomized to single agent LBH589, continued on single agent LBH589 40mg/30mg for an additional 4 months.	LBH589 - Randomized Phase n=6 participants at risk During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	LBH589 + ESA - Randomized Phase n=6 participants at risk During the randomized phase, participants randomized to LBH589 + Epoetin Alfa (ESA) received oral LBH589 40mg/30mg + ESA 30000 international units (IU)/week injected subcutaneously for 4 months.	Not Randomized n=1 participants at risk Participant, who was eligible for randomization, was not randomized. The participant had stable disease and should have been randomized, but in error, was considered a responder and therefore continued on single agent LBH589.
Blood and lymphatic system disorders Anaemia	8.8% 3/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Blood and lymphatic system disorders Leukopenia	11.8% 4/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Blood and lymphatic system disorders Neutropenia	35.3% 12/34	16.7% 1/6	16.7% 1/6	0.00% 0/1
Blood and lymphatic system disorders Thrombocytopenia	41.2% 14/34	16.7% 1/6	16.7% 1/6	0.00% 0/1
Ear and labyrinth disorders Vertigo	14.7% 5/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Endocrine disorders Hypothyroidism	17.6% 6/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Abdominal pain	8.8% 3/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Abdominal pain upper	8.8% 3/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Cheilitis	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Constipation	8.8% 3/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Diarrhoea	70.6% 24/34	16.7% 1/6	50.0% 3/6	0.00% 0/1
Gastrointestinal disorders Dyspepsia	14.7% 5/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Nausea	52.9% 18/34	16.7% 1/6	16.7% 1/6	0.00% 0/1
Gastrointestinal disorders Stomach discomfort	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Stomatitis	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Gastrointestinal disorders Teeth brittle	2.9% 1/34	0.00% 0/6	0.00% 0/6	100.0% 1/1
Gastrointestinal disorders Vomiting	26.5% 9/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
General disorders Asthenia	20.6% 7/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
General disorders Fatigue	47.1% 16/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
General disorders Gait disturbance	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
General disorders Influenza like illness	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
General disorders Oedema peripheral	5.9% 2/34	50.0% 3/6	16.7% 1/6	0.00% 0/1
General disorders Pyrexia	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Immune system disorders Hypersensitivity	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Abdominal wall abscess	0.00% 0/34	0.00% 0/6	0.00% 0/6	100.0% 1/1
Infections and infestations Bronchitis	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Infections and infestations Nasopharyngitis	8.8% 3/34	16.7% 1/6	16.7% 1/6	0.00% 0/1
Infections and infestations Urinary tract infection	5.9% 2/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Injury, poisoning and procedural complications Post procedural haematoma	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Injury, poisoning and procedural complications Transfusion reaction	0.00% 0/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Investigations Blood bilirubin increased	2.9% 1/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Investigations Blood creatinine increased	5.9% 2/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Investigations Blood iron increased	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Investigations Blood thyroid stimulating hormone increased	8.8% 3/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Investigations Weight decreased	23.5% 8/34	50.0% 3/6	0.00% 0/6	0.00% 0/1
Metabolism and nutrition disorders Anorexia	26.5% 9/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Metabolism and nutrition disorders Haemochromatosis	8.8% 3/34	0.00% 0/6	16.7% 1/6	0.00% 0/1
Metabolism and nutrition disorders Hyperglycaemia	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Metabolism and nutrition disorders Hypophosphataemia	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Arthralgia	2.9% 1/34	16.7% 1/6	16.7% 1/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Back pain	5.9% 2/34	33.3% 2/6	16.7% 1/6	100.0% 1/1
Musculoskeletal and connective tissue disorders Joint swelling	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Muscle spasms	14.7% 5/34	0.00% 0/6	33.3% 2/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Myalgia	11.8% 4/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Musculoskeletal and connective tissue disorders Pain in extremity	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Nervous system disorders Dysgeusia	11.8% 4/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Nervous system disorders Headache	8.8% 3/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Psychiatric disorders Depression	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Dyspnoea	5.9% 2/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	2.9% 1/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Respiratory, thoracic and mediastinal disorders Pulmonary hypertension	0.00% 0/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Skin and subcutaneous tissue disorders Alopecia	11.8% 4/34	0.00% 0/6	0.00% 0/6	0.00% 0/1
Skin and subcutaneous tissue disorders Skin fissures	2.9% 1/34	0.00% 0/6	0.00% 0/6	100.0% 1/1
Vascular disorders Haematoma	5.9% 2/34	16.7% 1/6	0.00% 0/6	0.00% 0/1
Vascular disorders Hypertension	2.9% 1/34	16.7% 1/6	0.00% 0/6	0.00% 0/1

Additional Information

Study Director

Novartis

Phone: 862-778-8300

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 or disclosure of trial results in their entirety.
Publication restrictions are in place

Restriction type: OTHER