Trial Outcomes & Findings for Extracorporeal Photopheresis and Low Dose Aldesleukin in Treating Patients With Steroid Refractory Chronic Graft-Versus-Host Disease (NCT NCT03007238)

NCT ID: NCT03007238

Last Updated: 2024-04-08

Results Overview

Defined as the proportion of response-evaluable participates that achieve a CR/PR or SD at Week 16 (4 weeks after the end of treatment).

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: At Week 16 (4 weeks after the end of treatment)
• Results posted on: 2024-04-08

Participant Flow

Participant milestones

Measure	Overall Study Patients received ECP twice weekly for weeks 1 through 4, then two treatment every other week until week 12 of protocol therapy. Daily IL-2 was given subcutaneously for 12 weeks. GVHD assessments were done at the baseline, then at 3-weeks intervals at 3, 6, 9,12 and 16 weeks.
Overall Study STARTED	10
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Extracorporeal Photopheresis and Low Dose Aldesleukin in Treating Patients With Steroid Refractory Chronic Graft-Versus-Host Disease

Baseline characteristics by cohort

Measure	Treatment (ECP Plus Low Dose IL-2) n=10 Participants Patients also undergo ECP twice weekly on weeks 1-4 and then receive 2 ECP treatments every other week until week 12 of protocol therapy. Patients receive aldesleukin subcutaneously (SC) daily for 12 weeks. Patients responding to upfront therapy with aldesleukin and ECP have the option to continue combination therapy per the discretion of the treating physician until clinical benefit is maintained or toxicities develop. After completion of study treatment, patients are followed up periodically.
Age, Continuous	46 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 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	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	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	1 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: At Week 16 (4 weeks after the end of treatment)

Population: At week 16, nine of the ten patients were alive and evaluable for response assessment. ORR was calculated for nine patients who were alive at Week 16 and was also calculated by ITT for ten patients.

Defined as the proportion of response-evaluable participates that achieve a CR/PR or SD at Week 16 (4 weeks after the end of treatment).

Outcome measures

Measure	Treatment (ECP+IL-2) n=10 Participants Patients received ECP twice weekly for weeks 1 through 4, then two treatment every other week until week 12 of protocol therapy. Daily IL-2 was given subcutaneously for 12 weeks. GVHD assessments were done at the baseline, then at 3-weeks intervals at 3, 6, 9,12 and 16 weeks.
Overall Response Rate at Week 16 (4 Weeks After the End of Treatment) Overall response rate in patients who were alive at Week 16 (4 weeks after the end of treatment)	88.9 percentage of participants Interval 51.8 to 99.7
Overall Response Rate at Week 16 (4 Weeks After the End of Treatment) Overall response rate by ITT	80.0 percentage of participants Interval 44.4 to 97.5

SECONDARY outcome

Timeframe: From date of first dose of study drug to first documented cGVHD progression (necessitating change of treatment), malignancy relapse or progression or death from any cause, whichever occurs first, assessed up to 1 year

Failure-free survival will be estimated using the product-limit method of Kaplan and Meier.

Outcome measures

Measure	Treatment (ECP+IL-2) n=10 Participants Patients received ECP twice weekly for weeks 1 through 4, then two treatment every other week until week 12 of protocol therapy. Daily IL-2 was given subcutaneously for 12 weeks. GVHD assessments were done at the baseline, then at 3-weeks intervals at 3, 6, 9,12 and 16 weeks.
Failure-free Survival	40 percentage of participants Interval 18.7 to 85.5

SECONDARY outcome

Timeframe: From date of first dose of study drug to date of death from any cause, assessed up to 1 year

Overall survival was estimated using the product-limit method of Kaplan and Meier.

Outcome measures

Measure	Treatment (ECP+IL-2) n=10 Participants Patients received ECP twice weekly for weeks 1 through 4, then two treatment every other week until week 12 of protocol therapy. Daily IL-2 was given subcutaneously for 12 weeks. GVHD assessments were done at the baseline, then at 3-weeks intervals at 3, 6, 9,12 and 16 weeks.
Overall Survival	60 percentage of participants Interval 36.2 to 99.5

Adverse Events

Overall Study

Serious events: 3 serious events

Other events: 8 other events

Deaths: 4 deaths

Serious adverse events

Measure	Overall Study n=10 participants at risk To evaluate the anti-cGVHD activity of extracorporeal photopheresis (ECP) when combined with low dose IL-2 (interleukin 2) (aldesleukin), in patients with steroid refractory cGVHD, as assessed by overall cGVHD response rate (complete response \[CR\]+partial response \[PR\]+stable disease \[SD\]).
Cardiac disorders Atrial flutter	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Infections and infestations Bronchial infection	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Infections and infestations Sepsis	20.0% 2/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.

Other adverse events

Measure	Overall Study n=10 participants at risk To evaluate the anti-cGVHD activity of extracorporeal photopheresis (ECP) when combined with low dose IL-2 (interleukin 2) (aldesleukin), in patients with steroid refractory cGVHD, as assessed by overall cGVHD response rate (complete response \[CR\]+partial response \[PR\]+stable disease \[SD\]).
Blood and lymphatic system disorders Anemia	30.0% 3/10 • Number of events 4 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Gastrointestinal disorders Anal pain	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Gastrointestinal disorders Nausea	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Gastrointestinal disorders Oral pain	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
General disorders Non-cardiac chest pain	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Infections and infestations Catheter related infection	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Injury, poisoning and procedural complications Vascular access complication	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Investigations Alanine aminotransferase increased	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Investigations CD4 lymphocytes decreased	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Investigations Lymphocyte count decreased	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Investigations Platelet count decreased	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Anorexia	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Hypercalcemia	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Hyperglycemia	20.0% 2/10 • Number of events 3 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Hypokalemia	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Hyponatremia	10.0% 1/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Metabolism and nutrition disorders Hypophosphatemia	20.0% 2/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Nervous system disorders Headache	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Respiratory, thoracic and mediastinal disorders Dyspnea	20.0% 2/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Respiratory, thoracic and mediastinal disorders Pneumonitis	10.0% 1/10 • Number of events 1 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Vascular disorders Hypertension	20.0% 2/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.
Vascular disorders Hypotension	10.0% 1/10 • Number of events 2 • Adverse events were assessed from date of first dose of study drug up to 16 weeks. All-Cause Mortality was assessed from first dose until death, assessed up to 1 year.

Additional Information

Dr. Amandeep Salhotra

City of Hope Medical Center

Phone: 626-359-8111

Email: asalhotra@coh.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place