Trial Outcomes & Findings for Phase II INCB024360 Study for Patients With Myelodysplastic Syndromes (MDS) (NCT NCT01822691)
NCT ID: NCT01822691
Last Updated: 2016-01-18
Results Overview
ORR, measured by Response Criteria for Patients with Myelodysplastic Syndrome (MDS). According International Working Group (IWG) 2006 criteria (Cheson et al, 2006). Complete Remission (CR), Partial Remission (PR), Marrow CR, and Hematological Improvement (HI)(any cell line). Stable Disease (SD): Failure to achieve at least PR, but no evidence of progression for \> 8 weeks.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Up to 12 months
Results posted on
2016-01-18
Participant Flow
Participants were enrolled at Moffitt Cancer Center between August 2013 and January 2014.
Participant milestones
| Measure |
INCB024360 Treatment
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
13
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
INCB024360 Treatment
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Phase II INCB024360 Study for Patients With Myelodysplastic Syndromes (MDS)
Baseline characteristics by cohort
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
13 Participants
n=5 Participants
|
|
Age, Continuous
|
72 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 12 monthsPopulation: All participants
ORR, measured by Response Criteria for Patients with Myelodysplastic Syndrome (MDS). According International Working Group (IWG) 2006 criteria (Cheson et al, 2006). Complete Remission (CR), Partial Remission (PR), Marrow CR, and Hematological Improvement (HI)(any cell line). Stable Disease (SD): Failure to achieve at least PR, but no evidence of progression for \> 8 weeks.
Outcome measures
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Overall Response Rate (ORR)
Stable Disease
|
12 participants
|
|
Overall Response Rate (ORR)
Complete Response
|
0 participants
|
|
Overall Response Rate (ORR)
Marrow Complete Response
|
0 participants
|
|
Overall Response Rate (ORR)
Hematological Improvement
|
0 participants
|
|
Overall Response Rate (ORR)
Progressive Disease
|
3 participants
|
SECONDARY outcome
Timeframe: Up to 12 monthsPopulation: All participants
Disease progression defined as progression to int-2 or high risk International Prognostic Scoring System (IPSS) score or AML, based on World Health Organization (WHO) AML Criteria.
Outcome measures
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Mean Time to Acute Myeloid Leukemia (AML) Progression
|
3.5 months
Interval 2.0 to 5.0
|
SECONDARY outcome
Timeframe: Up to 24 monthsPopulation: All participants
Overall Survival (OS) defined as the time between the start of treatment and death. Treatment Duration is 17 weeks plus optional continuation phase. Study Duration is Treatment Phase followed by survival follow-up.
Outcome measures
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Median Overall Survival (OS)
|
NA months
Interval 0.0 to
Median OS was not calculable because an insufficient number of participants reached the event at the final time point for assessment.
|
SECONDARY outcome
Timeframe: Up to 12 monthsPopulation: All participants
Participants with treatment emergent Grade 3 or 4 SAEs according to the NCI Common Terminology Criteria for Adverse Events Version (CTCAE) V4.0.
Outcome measures
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Number of Participants With Study Related Serious Adverse Events (SAEs)
|
0 participants
|
SECONDARY outcome
Timeframe: Up to 12 monthsPopulation: All participants
Participants with treatment emergent Other (not including serious) Adverse events.
Outcome measures
| Measure |
INCB024360 Treatment
n=15 Participants
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
AE of any category
|
12 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Metabolism and Nutrition Disorders
|
7 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Gastrointestinal Disorders
|
6 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
General Disorders
|
3 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Investigations
|
3 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Immune System Disorders
|
2 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Musculoskeletal Disorders
|
2 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Skin and Subcutaneous Tissue Disorders
|
2 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Infections and Infestations
|
1 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Nervous System Disorders
|
1 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Psychiatric Disorders
|
1 participants
|
|
Number of Participants With Study Treatment Related Adverse Events (AEs)
Respiratory Disorders
|
1 participants
|
Adverse Events
INCB024360 Treatment
Serious events: 3 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
INCB024360 Treatment
n=15 participants at risk
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Immune system disorders
Allergic reaction
|
6.7%
1/15 • Number of events 2 • 11 months
|
|
Infections and infestations
Lung infection
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Infections and infestations
Lymph gland infection
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Vascular disorders
Lymphedema
|
6.7%
1/15 • Number of events 1 • 11 months
|
Other adverse events
| Measure |
INCB024360 Treatment
n=15 participants at risk
Participants were treated with 600 mg orally, twice a day for 16 weeks, unless clear evidence of disease progression or toxicity was evident.
|
|---|---|
|
Gastrointestinal disorders
Nausea
|
33.3%
5/15 • Number of events 5 • 11 months
|
|
Gastrointestinal disorders
Constipation
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Gastrointestinal disorders
Diarrhea
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Gastrointestinal disorders
Vomiting
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Gastrointestinal disorders
Mucositis oral
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Gastrointestinal disorders
Oral hemorrhage
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Investigations
Platelet count decreased
|
26.7%
4/15 • Number of events 8 • 11 months
|
|
Investigations
Weight loss
|
20.0%
3/15 • Number of events 3 • 11 months
|
|
Investigations
Blood prolactin abnormal
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Investigations
Investigations - Other, Increased chloride
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Investigations
Neutrophil count decreased
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Metabolism and nutrition disorders
Anorexia
|
46.7%
7/15 • Number of events 7 • 11 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
13.3%
2/15 • Number of events 3 • 11 months
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders - Other, Decreased chloride
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, Lip ulcer
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Skin and subcutaneous tissue disorders
Skin induration
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
General disorders
Chills
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
General disorders
Fatigue
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
General disorders
Pain
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
General disorders
Irritability
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Infections and infestations
Upper respiratory infection
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Infections and infestations
Bladder infection
|
6.7%
1/15 • Number of events 2 • 11 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Immune system disorders
Immune system disorders - Other, Decreased testosterone
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Immune system disorders
Immune system disorders - Other, Autoimmune endocrinopathy
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Injury, poisoning and procedural complications
Fall
|
13.3%
2/15 • Number of events 2 • 11 months
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications - Other, Hand wound
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Nervous system disorders
Dizziness
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Nervous system disorders
Dysgeusia
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Cardiac disorders
Palpitations
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Endocrine disorders
Adrenal insufficiency
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Endocrine disorders
Hypothyroidism
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Bladder cancer
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Psychiatric disorders
Confusioin
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Psychiatric disorders
Depression
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Renal and urinary disorders
Hematuria
|
6.7%
1/15 • Number of events 1 • 11 months
|
|
Renal and urinary disorders
Renal and urinary disorders - Other, Kidney injury
|
6.7%
1/15 • Number of events 2 • 11 months
|
Additional Information
Dr. Rami Komrokji
H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-4692
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place