Trial Outcomes & Findings for Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (NCT NCT04854096)
NCT ID: NCT04854096
Last Updated: 2025-05-23
Results Overview
Proportion of subjects who achieve ≥35% reduction in spleen volume from baseline compared to Week 24 as measured by MRI (or by CT for applicable subjects)
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
7 participants
Primary outcome timeframe
baseline and week 24
Results posted on
2025-05-23
Participant Flow
Participant milestones
| Measure |
NS-018
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Overall Study
STARTED
|
5
|
2
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
5
|
2
|
Reasons for withdrawal
| Measure |
NS-018
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Study terminated by Sponsor due to business reasons.
|
2
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Other reasons
|
1
|
0
|
Baseline Characteristics
Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis
Baseline characteristics by cohort
| Measure |
NS-018
n=5 Participants
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 Participants
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
Total
n=7 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Age, Continuous
|
65.0 Years
STANDARD_DEVIATION 16.48 • n=5 Participants
|
70.5 Years
STANDARD_DEVIATION 0.71 • n=7 Participants
|
66.6 Years
STANDARD_DEVIATION 13.72 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: baseline and week 24Population: The primary outcome measure for an efficacy endpoint to compare the efficacy of NS-018 to the Best Available Therapy (BAT) was analyzed among the limited participants enrolled before the study was terminated early for business reasons.
Proportion of subjects who achieve ≥35% reduction in spleen volume from baseline compared to Week 24 as measured by MRI (or by CT for applicable subjects)
Outcome measures
| Measure |
NS-018
n=5 Participants
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 Participants
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Change in Spleen Volume
|
0 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline and week 24Population: This co-primary outcome measure for an efficacy endpoint to compare NS-018 to the Best Available Therapy (BAT) was analyzed among the limited participants enrolled before the study was terminated early for business reasons. .
Proportion of subjects who achieve ≥50% reduction in total symptom score from baseline compared to Week 24 as measured by the MF-SAF v4.0
Outcome measures
| Measure |
NS-018
n=5 Participants
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 Participants
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Change in Total Symptom Score (TSS)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: from baseline to anytime before or at week 24Population: This secondary outcome measure for an efficacy endpoint to compare the efficacy of NS-018 to the Best Available Therapy (BAT) was analyzed among the limited participants enrolled before the study was terminated early for business reasons.
Proportion of subjects in NS-018 vs BAT arm who achieve ≥35% reduction in spleen volume from baseline at any time up to Week 24 as measured by MRI (or by CT for applicable subjects)
Outcome measures
| Measure |
NS-018
n=5 Participants
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 Participants
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Change in Spleen Volume
|
1 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: from baseline to week 24Population: TEAE's were evaluated in the participants who received NS-018 or BAT.
Laboratory events graded by the NCI CTCAE v5.0 will be assessed in both arms, to compare the safety profile of NS-018 versus BAT.
Outcome measures
| Measure |
NS-018
n=5 Participants
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 Participants
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Comparison of Treatment-emergent AEs Between NS-018 and BAT
|
5 Participants
|
2 Participants
|
Adverse Events
NS-018
Serious events: 1 serious events
Other events: 5 other events
Deaths: 0 deaths
Best Available Therapy (BAT)
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
NS-018
n=5 participants at risk
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 participants at risk
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Infections and infestations
Tracheobronchitis
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
Other adverse events
| Measure |
NS-018
n=5 participants at risk
Self-administered NS-018 300 mg orally, twice daily, preferably at the same time each day in consecutive 4-week (28-day) cycles
NS-018: Experimental
|
Best Available Therapy (BAT)
n=2 participants at risk
Single agent per Investigator discretion or no therapy
Best Available Therapy: Active Comparator
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
80.0%
4/5 • Number of events 8 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Investigations
Platelet Count Decrease
|
40.0%
2/5 • Number of events 2 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Eye disorders
Conjunctival haemorrhage
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Gastrointestinal disorders
Hematochezia
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Influenza
|
40.0%
2/5 • Number of events 2 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Injury, poisoning and procedural complications
Allergic transfusion reaction
|
20.0%
1/5 • Number of events 2 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
20.0%
1/5 • Number of events 3 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary Disease
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Eye disorders
Eyelid function disorder
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Gastrointestinal disorders
Abdominal Pain
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Gastrointestinal disorders
Diarrhoea
|
40.0%
2/5 • Number of events 2 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Gastrointestinal disorders
Constipation
|
20.0%
1/5 • Number of events 3 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Gastrointestinal disorders
Nausea
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
General disorders
Fatigue
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
General disorders
Influenza-like illness
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
General disorders
Oedema peripheral
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Hepatobiliary disorders
Cholestasis
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Upper respiratory tract infection
|
40.0%
2/5 • Number of events 2 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Bronchitis
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 3 • from baseline to week 24
|
|
Infections and infestations
Tracheobronchitis
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Urinary tract infection
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Injury, poisoning and procedural complications
Periorbital haemorrhage
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Injury, poisoning and procedural complications
Transfusion reaction
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Investigations
C-reactive protein increased
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Investigations
Eastern Cooperative Oncology Group performance status worsened
|
20.0%
1/5 • Number of events 3 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
40.0%
2/5 • Number of events 2 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Musculoskeletal and connective tissue disorders
Arthalgia
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Musculoskeletal and connective tissue disorders
Spinal pain
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Nervous system disorders
Lethargy
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Nervous system disorders
Neuropathy peripheral
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Psychiatric disorders
Affective disorder
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Renal and urinary disorders
Nocturia
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
20.0%
1/5 • Number of events 4 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Skin and subcutaneous tissue disorders
Skin haemorrhage
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Skin and subcutaneous tissue disorders
Skin ulcer
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Vascular disorders
Haematoma
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Vascular disorders
Venous haemorrhage
|
20.0%
1/5 • Number of events 1 • from baseline to week 24
|
0.00%
0/2 • from baseline to week 24
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/5 • from baseline to week 24
|
50.0%
1/2 • Number of events 1 • from baseline to week 24
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee UK: The Sponsor recognizes that the PI may have a responsibility to ensure that results of scientific interest arising from the Clinical Trial are appropriately published. Germany: Details of study and its results shall not be publicized or published in any form without prior written consent of the sponsor. Malaysia: Institution and Investigator may publish or otherwise publicly disclose, for non-commercial purposes.
- Publication restrictions are in place
Restriction type: OTHER