Trial Outcomes & Findings for Study of Ruxolitinib in Sclerotic Chronic Graft-Versus-Host Disease After Failure of Systemic Glucocorticoids (NCT NCT03616184)

Last Updated: 2023-10-05

Results Overview

The primary endpoint of the study was complete or partial response in skin and/or joint defined according to the 2014 NIH cGVHD Consensus Criteria. Partial response for skin would be a decrease in NIH Skin Score by 1 or more points. Partial response in joint would be a decrease in NIH Joint and Fascia Score by 1 or more points or increase in P-ROM score by 1 point for any site.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

49 participants

Primary outcome timeframe

6 months

Results posted on

2023-10-05

Participant Flow

Participant milestones

Participant milestones
Measure	Ruxolitinib Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Overall Study STARTED	47
Overall Study COMPLETED	34
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of Ruxolitinib in Sclerotic Chronic Graft-Versus-Host Disease After Failure of Systemic Glucocorticoids

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ruxolitinib n=47 Participants Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Age, Continuous	62 Years n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	28 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 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	2 Participants n=5 Participants
Race (NIH/OMB) White	43 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	47 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Ruxolitinib n=34 Participants Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Percentage of Participants With Complete and Partial Responses in Skin and/or Joint	49 percentage of participants

SECONDARY outcome

Timeframe: 6 months

As determined by 2014 NIH Criteria which is a clinician overall severity score of 0 for complete response and clinician overall severity score decreased by 2 or more points on a 0-10 scale for partial response.

Outcome measures

Outcome measures
Measure	Ruxolitinib n=34 Participants Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Percentage of Participants With Complete or Partial Responses Overall	47 percentage of participants

Adverse Events

Ruxolitinib

Serious events: 13 serious events

Other events: 29 other events

Deaths: 4 deaths

Serious adverse events

Serious adverse events
Measure	Ruxolitinib n=47 participants at risk Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Gastrointestinal disorders Abdominal Pain	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Blood and lymphatic system disorders Blood and lymphatic system disorders	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Infections and infestations Catheter related infection	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Cardiac disorders Chest pain-cardiac	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Hepatobiliary disorders Cholecystitis	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Gastrointestinal disorders Enterocolitis	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
General disorders Fever	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Injury, poisoning and procedural complications Fracture	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Psychiatric disorders Hallucinations	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Respiratory, thoracic and mediastinal disorders Hypoxia	2.1% 1/47 • Number of events 2 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Infections and infestations Infections and infestations	2.1% 1/47 • Number of events 2 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Investigations Investigations	8.5% 4/47 • Number of events 5 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Infections and infestations Lung Infection	4.3% 2/47 • Number of events 2 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Gastrointestinal disorders Nausea	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Infections and infestations Sepsis	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
General disorders Sudden death NOS	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Nervous system disorders Syncope	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Vascular disorders Thromboembolic event	2.1% 1/47 • Number of events 1 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.

Other adverse events

Other adverse events
Measure	Ruxolitinib n=47 participants at risk Patients will receive oral ruxolitinib at a dose of 10 mg twice daily. Ruxolitinib: Patients with sclerotic chronic graft-versus-host disease (GVHD) will receive oral ruxolitinib at a dose of 10 mg twice daily. Doses may not exceed 10 mg twice daily. Ruxolitinib will be continued for 6 months. Patients who continue to have stable disease, mixed responses or partial/complete responses at the end of 6 months may continue the drug for a total of 12 months.
Blood and lymphatic system disorders Anemia	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Investigations Aspartate aminotransferase increased	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Gastrointestinal disorders Diarrhea	10.6% 5/47 • Number of events 8 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Nervous system disorders Dizziness	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Eye disorders Dry Eye	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
General disorders Fatigue	14.9% 7/47 • Number of events 8 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Vascular disorders Hypertension	8.5% 4/47 • Number of events 4 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Psychiatric disorders Insomnia	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Gastrointestinal disorders Nausea	17.0% 8/47 • Number of events 9 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	6.4% 3/47 • Number of events 3 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Infections and infestations Upper respiratory infection	17.0% 8/47 • Number of events 14 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Gastrointestinal disorders Vomiting	12.8% 6/47 • Number of events 6 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.
Investigations weight gain	10.6% 5/47 • Number of events 8 • 2 years Adverse Events were monitored/assessed/classified without regard to the specific Adverse Event Term.

Additional Information

Vijaya Bhatt

University of Nebraska Medical Center

Phone: 402-559-8008

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place