Trial Outcomes & Findings for A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome (NCT NCT02400463)
NCT ID: NCT02400463
Last Updated: 2021-01-25
Results Overview
Number of Patients Alive at 2 Months after the first administration of ruxolitinib.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
2 Months
Results posted on
2021-01-25
Participant Flow
Participant milestones
| Measure |
Ruxolitinib
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Pilot Study of Ruxolitinib in Secondary Hemophagocytic Syndrome
Baseline characteristics by cohort
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Age, Continuous
|
38 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 MonthsNumber of Patients Alive at 2 Months after the first administration of ruxolitinib.
Outcome measures
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Overall Survival at 2 Months
|
6 Participants
|
SECONDARY outcome
Timeframe: 2 MonthsComplete response is defined as complete normalization of all quantifiable symptoms and laboratory abnormalities. A partial response is defined as at least a 25% improvement in two or more quantifiable symptoms/laboratory markers.
Outcome measures
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Percentage of Patients With a Response to Treatment With Ruxolitinib
|
100 percentage of participants
|
SECONDARY outcome
Timeframe: Up to 3 years (Due to funding and other constraints, participant follow-up was discontinued in 2020. Thus, not all participants were followed for a full three years.)Duration will be calculated from the date of the determination of partial response or better until the date of progression, death, or additional non-protocol therapy.
Outcome measures
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Duration of Response
|
17.25 months
Interval 3.5 to 35.5
|
SECONDARY outcome
Timeframe: Up to 3 years (Due to funding and other constraints, participant follow-up was discontinued in 2020. Thus, not all participants were followed for a full three years.)Progressive Disease is defined as at least a 50% worsening in two or more quantifiable laboratory markers. Calculated from the first administration of ruxolitinib until the date of progression or death.
Outcome measures
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Progression Free Survival Time
|
17.25 months
Interval 3.5 to 35.5
|
SECONDARY outcome
Timeframe: Up to 30 days after last treatment administrationIncidence and grade of adverse events (AEs) unlikely, possibly or probably related to treatment (tx) with ruxolitinib. Graded per Common Terminology Criteria for Adverse Events (CTCAE) v.4. Grade refers to the severity of the AE, from mild (grade 1) to life-threatening (grade 4).
Outcome measures
| Measure |
Ruxolitinib
n=6 Participants
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Regimen Related Toxicities
Grade 1
|
5 Adverse Events
|
|
Regimen Related Toxicities
Grade 2
|
3 Adverse Events
|
|
Regimen Related Toxicities
Grade 3
|
1 Adverse Events
|
|
Regimen Related Toxicities
Grade 4
|
1 Adverse Events
|
Adverse Events
Ruxolitinib
Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Ruxolitinib
n=6 participants at risk
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
Other adverse events
| Measure |
Ruxolitinib
n=6 participants at risk
Ruxolitinib 15 mg by mouth twice daily. For patients unable to ingest tablets, ruxolitinib suspended in water may be administered through a nasogastric (NG) or percutaneous endoscopy gastrostomy (PEG) tube.
|
|---|---|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders - Other, specify
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Endocrine disorders
Cushingoid
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Gastrointestinal disorders
Abdominal pain
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Gastrointestinal disorders
Constipation
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Gastrointestinal disorders
Dysphagia
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Gastrointestinal disorders
Nausea
|
33.3%
2/6 • Number of events 3 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
Fatigue
|
50.0%
3/6 • Number of events 4 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
Fever
|
16.7%
1/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
33.3%
2/6 • Number of events 4 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
Localized edema
|
50.0%
3/6 • Number of events 3 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
Non-cardiac chest pain
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
General disorders
Pain
|
16.7%
1/6 • Number of events 3 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Infections and infestations - Other, specify
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Sinusitis
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Skin infection
|
16.7%
1/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Tooth infection
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Upper respiratory infection
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Infections and infestations
Vaginal infection
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Investigations
Cholesterol high
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
16.7%
1/6 • Number of events 3 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
16.7%
1/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Nervous system disorders
Dizziness
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Nervous system disorders
Headache
|
66.7%
4/6 • Number of events 6 • Up to 30 days after last treatment administration (13.5 months)
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Psychiatric disorders
Agitation
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Psychiatric disorders
Anxiety
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Psychiatric disorders
Depression
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Psychiatric disorders
Insomnia
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Psychiatric disorders
Psychiatric disorders - Other, specify
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Reproductive system and breast disorders
Irregular menstruation
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
33.3%
2/6 • Number of events 2 • Up to 30 days after last treatment administration (13.5 months)
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Skin and subcutaneous tissue disorders
Erythroderma
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
33.3%
2/6 • Number of events 4 • Up to 30 days after last treatment administration (13.5 months)
|
|
Vascular disorders
Hot flashes
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • Number of events 1 • Up to 30 days after last treatment administration (13.5 months)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place