Trial Outcomes & Findings for Study of Clinical Efficacy and Safety of Tosedostat in MDS (NCT NCT02452346)
NCT ID: NCT02452346
Last Updated: 2018-06-12
Results Overview
Survival following treatment to the date of death, assessed up to a period of 3-4 years.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
from start of treatment until death, assessed up to a period of 3-4 years.
Results posted on
2018-06-12
Participant Flow
Participant milestones
| Measure |
All Patients
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
9
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Clinical Efficacy and Safety of Tosedostat in MDS
Baseline characteristics by cohort
| Measure |
All Patients
n=12 Participants
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
|
Age, Continuous
|
72 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: from start of treatment until death, assessed up to a period of 3-4 years.Survival following treatment to the date of death, assessed up to a period of 3-4 years.
Outcome measures
| Measure |
All Patients
n=12 Participants
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Over All Survival
|
15.9 months
Interval 2.5 to 30.7
|
SECONDARY outcome
Timeframe: Approximately 3 yearsOverall response according to IWG 2006 criteira
Outcome measures
| Measure |
All Patients
n=12 Participants
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Overall Response
Stable Disease
|
8 Participants
|
|
Overall Response
Non-evaluable
|
3 Participants
|
|
Overall Response
Complete Remission
|
1 Participants
|
SECONDARY outcome
Timeframe: from start of treatment to 1 year and 2 years post treatment initiationOutcome measures
| Measure |
All Patients
n=12 Participants
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
One Year and Two Year Survival
One year survival
|
8 Participants
|
|
One Year and Two Year Survival
two year survival
|
4 Participants
|
Adverse Events
All Patients
Serious events: 8 serious events
Other events: 12 other events
Deaths: 9 deaths
Serious adverse events
| Measure |
All Patients
n=12 participants at risk
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Other - Decline in performance status
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Syncope
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Infections and infestations
Sepsis
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Infections and infestations
Rash pustular
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
General disorders
Fever
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
Other adverse events
| Measure |
All Patients
n=12 participants at risk
Tosedostat 120 mg PO once daily will be administered.
Tosedostat: 120 mg PO once daily continuously for each 28 day treatment cycle
|
|---|---|
|
Investigations
Other - BNP increased
|
58.3%
7/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Electrocardiogram QT corrected interval prolonged
|
50.0%
6/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
General disorders
Fatigue
|
50.0%
6/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Injury, poisoning and procedural complications
Bruising
|
41.7%
5/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Diarrhea
|
41.7%
5/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
General disorders
Edema limbs
|
41.7%
5/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Hypomagnesemia
|
33.3%
4/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Alanine transferase increased
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Dizziness
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Ejection fraction decreased
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Platelet count decreased
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
25.0%
3/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
GGT increased
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Psychiatric disorders
Insomnia
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Neutrophil count decreased
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Toothache
|
16.7%
2/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Abdominal pain
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Alkaline phosphatase increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Immune system disorders
Allergic reaction
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Blood and lymphatic system disorders
Anemia
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Aspartate aminotransferase increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Cardiac disorders
Atrial fibrillation
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Blood bilirubin increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Eye disorders
Blurred vision
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Cardiac troponin I increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Cognitive disturbance
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Psychiatric disorders
Confusion
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Constipation
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Creatinine increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Psychiatric disorders
Delirium
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Psychiatric disorders
Depression
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Dry mouth
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
General disorders
Edema face
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Injury, poisoning and procedural complications
Fracture
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Gastroenteritis
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Hemorrhoidal hemorrhage
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Hemorrhoids
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
INR increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Lethargy
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Mucositis
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Nausea
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Neuralgia
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
General disorders
Non-cardiac chest pain
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Oral hemorrhage
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Cardiac disorders
Other - Demand ischemia
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Other - Ferritin increased
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Psychiatric disorders
Other - Forgetfulness
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Eye disorders
Other - left subconjunctival hemorrhage
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Injury, poisoning and procedural complications
Other - Lip excoriation
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Other - Muscle cramps
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Musculoskeletal and connective tissue disorders
Other - Muscle spasm
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Other - Pneumonia
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Nervous system disorders
Paresthesia, foot
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary edema
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary hypertension
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Renal and urinary disorders
Urinary incontinence
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Infections and infestations
Urinary tract infection
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Gastrointestinal disorders
Vomiting
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
|
Investigations
Weight loss
|
8.3%
1/12 • The adverse event data was collected for a period of approx. 3 years from the date of enrollment.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place