Trial Outcomes & Findings for Long-Term Side Effects of Ruxolitinib in Treating Patients With Myelofibrosis (NCT NCT02784496)
NCT ID: NCT02784496
Last Updated: 2025-08-29
Results Overview
A review of the most common adverse events related to study medication will be performed. Will follow standard reporting guidelines for adverse events. Safety data will be summarized by the number of participants affected.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
8 participants
Primary outcome timeframe
to the date of failure or death from any cause, Up to 7 years, 3 months and 24 days
Results posted on
2025-08-29
Participant Flow
Participant milestones
| Measure |
Treatment (Ruxolitinib, Follow-up)
Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.
Long-term Follow-up: Undergo follow-up assessment
Quality-of-Life Assessment: Ancillary studies
Ruxolitinib: Given PO
|
|---|---|
|
Overall Study
STARTED
|
8
|
|
Overall Study
COMPLETED
|
8
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Long-Term Side Effects of Ruxolitinib in Treating Patients With Myelofibrosis
Baseline characteristics by cohort
| Measure |
Treatment (Ruxolitinib, Follow-up)
n=8 Participants
Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.
Long-term Follow-up: Undergo follow-up assessment
Quality-of-Life Assessment: Ancillary studies
Ruxolitinib: Given PO
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
8 Participants
n=5 Participants
|
|
Age, Continuous
|
73 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 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
|
8 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
|
|
Region of Enrollment
United States
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: to the date of failure or death from any cause, Up to 7 years, 3 months and 24 daysA review of the most common adverse events related to study medication will be performed. Will follow standard reporting guidelines for adverse events. Safety data will be summarized by the number of participants affected.
Outcome measures
| Measure |
Treatment (Ruxolitinib, Follow-up)
n=8 Participants
Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.
Long-term Follow-up: Undergo follow-up assessment
Quality-of-Life Assessment: Ancillary studies
Ruxolitinib: Given PO
|
|---|---|
|
Most Common Treatment Related Adverse Events
Infections
|
1 participants
|
|
Most Common Treatment Related Adverse Events
Headache
|
1 participants
|
|
Most Common Treatment Related Adverse Events
Fatigue
|
2 participants
|
|
Most Common Treatment Related Adverse Events
Dizziness
|
1 participants
|
Adverse Events
Treatment (Ruxolitinib, Follow-up)
Serious events: 6 serious events
Other events: 8 other events
Deaths: 2 deaths
Serious adverse events
| Measure |
Treatment (Ruxolitinib, Follow-up)
n=8 participants at risk
Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.
Long-term Follow-up: Undergo follow-up assessment
Quality-of-Life Assessment: Ancillary studies
Ruxolitinib: Given PO
|
|---|---|
|
Cardiac disorders
Atrial fibrillation
|
12.5%
1/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Injury, poisoning and procedural complications
Fall
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Headache
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Vascular disorders
Hypertension
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Intracranial hemorrhage
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Lower gastrointestinal hemorrhage
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Lung infection
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
|
12.5%
1/8 • Number of events 5 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Nervous system disorders
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Non-cardiac chest pain
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Renal and urinary disorders
Renal calculi
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Sepsis
|
25.0%
2/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Skin infection
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Surgical and medical procedures
Surgical and medical procedures
|
37.5%
3/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Renal and urinary disorders
Urinary tract infection
|
37.5%
3/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
Other adverse events
| Measure |
Treatment (Ruxolitinib, Follow-up)
n=8 participants at risk
Patients continue to receive ruxolitinib PO QD or BID. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo follow-up assessment for safety over 10 minutes every 3 cycles.
Long-term Follow-up: Undergo follow-up assessment
Quality-of-Life Assessment: Ancillary studies
Ruxolitinib: Given PO
|
|---|---|
|
Investigations
Alanine aminotransferase increased
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Investigations
Alkaline phosphatase increased
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Allergic rhinitis
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Anorexia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
62.5%
5/8 • Number of events 5 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Investigations
Aspartate aminotransferase increased
|
37.5%
3/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Cardiac disorders
Atrial fibrillation
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Bloating
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Injury, poisoning and procedural complications
Bruising
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Chills
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Conjunctivitis
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Investigations
Creatinine increased
|
25.0%
2/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Dehydration
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Diarrhea
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Dizziness
|
12.5%
1/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Dysarthria
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Dysphagia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
37.5%
3/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Edema
|
62.5%
5/8 • Number of events 6 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Injury, poisoning and procedural complications
Fall
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Fatigue
|
37.5%
3/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Fever
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Gait disturbance
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
37.5%
3/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
General disorders and administration site conditions
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
37.5%
3/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Injury, poisoning and procedural complications
Hip fracture
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
25.0%
2/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Hypernatremia
|
12.5%
1/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Infection
|
25.0%
2/8 • Number of events 4 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Infections and infestations
Infections and infestations
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Injury, poisoning and procedural complications
Injury, poisoning and procedural complications
|
37.5%
3/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Psychiatric disorders
Insomnia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Investigations
Investigations
|
62.5%
5/8 • Number of events 9 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Localized edema
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
25.0%
2/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Musculoskeletal and connective tissue disorders
Myositis
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Nausea
|
12.5%
1/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Eye disorders
Night blindness
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
General disorders
Pain
|
50.0%
4/8 • Number of events 9 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Paresthesia
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Nervous system disorders
Peripheral motor neuropathy
|
37.5%
3/8 • Number of events 3 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
25.0%
2/8 • Number of events 6 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Surgical and medical procedures
Surgical and medical procedures
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Renal and urinary disorders
Urinary frequency
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Vascular disorders
Vascular disorders
|
12.5%
1/8 • Number of events 1 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
1/8 • Number of events 2 • Up to the date of failure or death from any cause, up to 7 years, 3 months and 24 days.
|
Additional Information
Prithviraj Bose, MD/Professor
The University of MD Anderson Cancer Center
Phone: 713-792-7747
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place