Trial Outcomes & Findings for Open-Label Study to Evaluate the Efficacy of ECP in Secondary Progressive Multiple Sclerosis (NCT NCT02296346)

Last Updated: 2019-08-14

Results Overview

MSFC score is a composite score calculated from 3 tests: 1) Timed 25-Foot walk (leg function); 2) 9-Hole Peg Test (arm function); and 3) Paced Auditory Serial Addition Test (cognitive function). The results are combined to create a single score (the MSFC Z-Score) to measure performance and change over time in subjects with MS. MSFC Z-score = {Z arm + Z leg + Z cognitive} / 3.0. A positive score indicates that, on average, an individual performed better than the reference population and a negative score indicates that, on average, an individual performed worse than the reference population. The Expanded Disability Status Scale (EDSS) is used to quantify disability due to symptoms of MS and to track changes in disability status over time. Scores range from 0 (normal neurological exam) to 10 (death due to multiple sclerosis). Scores are determined by performing a neurological exam and given in increments of 0.5.

Recruitment status

TERMINATED

Study phase

Target enrollment

13 participants

Primary outcome timeframe

1 year

Results posted on

2019-08-14

Participant Flow

Participant milestones

Participant milestones
Measure	Corticosteriod Arm Patients will receive 1 gram of IV SoluMedrol over 1 hour, once every month for 12 months. SoluMedrol: Infusion of drug subcutaneously, once a month.	Extracorporeal Photopheresis Patient will receive an Extracorporeal Photopheresis treatment at a set frequency over the course of 1 year. The treatment takes about 2-3 hours per session to complete. The treatment schedule is as follows: ECP will be administered according to the following schedule: Study Arm: Weeks 1-8: 3 times per week Weeks 9-16: Twice per week Weeks 17-36: Treatment on two consecutive days every 2 weeks (or optionally, one treatment per week) Weeks 37-43: Once every 2 weeks Weeks 44-52: Once every 4 Weeks Extracorporeal Photopheresis: This intervention is the placement of up to two IV's to extract your blood as a set volume, separate out the white cells and return the red cells to your body. Then the white cells are treated with a drug called UVADEX (methoxsalen), excited by a UV light and returned to your body. Once IV is to withdraw your blood and the other is to return your blood to your body.
Overall Study STARTED	7	6
Overall Study COMPLETED	2	0
Overall Study NOT COMPLETED	5	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Open-Label Study to Evaluate the Efficacy of ECP in Secondary Progressive Multiple Sclerosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Corticosteriod Arm n=7 Participants Patients will receive 1 gram of IV SoluMedrol over 1 hour, once every month for 12 months. SoluMedrol: Infusion of drug subcutaneously, once a month.	Extracorporeal Photopheresis n=6 Participants Patient will receive an Extracorporeal Photopheresis treatment at a set frequency over the course of 1 year. The treatment takes about 2-3 hours per session to complete. The treatment schedule is as follows: ECP will be administered according to the following schedule: Study Arm: Weeks 1-8: 3 times per week Weeks 9-16: Twice per week Weeks 17-36: Treatment on two consecutive days every 2 weeks (or optionally, one treatment per week) Weeks 37-43: Once every 2 weeks Weeks 44-52: Once every 4 Weeks Extracorporeal Photopheresis: This intervention is the placement of up to two IV's to extract your blood as a set volume, separate out the white cells and return the red cells to your body. Then the white cells are treated with a drug called UVADEX (methoxsalen), excited by a UV light and returned to your body. Once IV is to withdraw your blood and the other is to return your blood to your body.	Total n=13 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=113 Participants	5 Participants n=163 Participants	11 Participants n=160 Participants
Age, Categorical >=65 years	1 Participants n=113 Participants	1 Participants n=163 Participants	2 Participants n=160 Participants
Sex: Female, Male Female	4 Participants n=113 Participants	2 Participants n=163 Participants	6 Participants n=160 Participants
Sex: Female, Male Male	3 Participants n=113 Participants	4 Participants n=163 Participants	7 Participants n=160 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=113 Participants	6 Participants n=163 Participants	13 Participants n=160 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Race (NIH/OMB) Asian	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Race (NIH/OMB) Black or African American	1 Participants n=113 Participants	0 Participants n=163 Participants	1 Participants n=160 Participants
Race (NIH/OMB) White	6 Participants n=113 Participants	6 Participants n=163 Participants	12 Participants n=160 Participants
Race (NIH/OMB) More than one race	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=113 Participants	0 Participants n=163 Participants	0 Participants n=160 Participants
Multiple Sclerosis Functional Composite (MSFC)	0.2410 MSFC z-score n=113 Participants	-0.2335 MSFC z-score n=163 Participants	0.0022 MSFC z-score n=160 Participants
Expanded Disability Status Scale (EDSS)	6 EDSS Score n=113 Participants	5.92 EDSS Score n=163 Participants	5.96 EDSS Score n=160 Participants

PRIMARY outcome

Timeframe: 1 year

Population: Baseline MSFC and EDSS scores were captured for all 13 subjects, however some subjects withdrew immediately after baseline. Withdrawal of additional subjects continued from that point. Only 1 patient from the ECP arm made it to the 12 month visit. Four patients from the CS arm made it to 12 mos, however 1 of the 4 declined the MSFC tests.

Outcome measures

Outcome measures
Measure	ECP Arm n=1 Participants Study participants assigned to ECP treatment	Corticosteroid Arm n=4 Participants Study participants assigned to corticosteroid treatment
Multiple Sclerosis Functional Composite (MSFC) Z-score and Expanded Disability Status Scale (EDSS) EDSS Score	6 score on a scale Interval 6.0 to 6.0	6.25 score on a scale Interval 6.0 to 6.5
Multiple Sclerosis Functional Composite (MSFC) Z-score and Expanded Disability Status Scale (EDSS) MSFC Z-Score	1.1031 score on a scale Interval 1.1031 to 1.1031	0.6751 score on a scale Interval 0.4914 to 0.9504

SECONDARY outcome

Timeframe: 2 year

Population: Due to low enrollment numbers and a high dropout rate, there was insufficient data to analyze and correlate treatment with clinical outcomes. Only 2 subjects continued to the 24 month completion, both were corticosteroid patients. No ECP subjects made it to 24 mos. Therefore, there was no analysis performed on the 2 year data.

Changes in immunological parameters that occur following initiation of ECP or corticosteroids and any relevant correlation with clinical outcomes

Outcome measures

Outcome data not reported

Adverse Events

Corticosteriod Arm

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Extracorporeal Photopheresis

Serious events: 1 serious events

Other events: 3 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Corticosteriod Arm n=7 participants at risk Patients will receive 1 gram of IV SoluMedrol over 1 hour, once every month for 12 months. SoluMedrol: Infusion of drug subcutaneously, once a month.	Extracorporeal Photopheresis n=6 participants at risk Patient will receive an Extracorporeal Photopheresis treatment at a set frequency over the course of 1 year. The treatment takes about 2-3 hours per session to complete. The treatment schedule is as follows: ECP will be administered according to the following schedule: Study Arm: Weeks 1-8: 3 times per week Weeks 9-16: Twice per week Weeks 17-36: Treatment on two consecutive days every 2 weeks (or optionally, one treatment per week) Weeks 37-43: Once every 2 weeks Weeks 44-52: Once every 4 Weeks Extracorporeal Photopheresis: This intervention is the placement of up to two IV's to extract your blood as a set volume, separate out the white cells and return the red cells to your body. Then the white cells are treated with a drug called UVADEX (methoxsalen), excited by a UV light and returned to your body. Once IV is to withdraw your blood and the other is to return your blood to your body.
Psychiatric disorders Suicide	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.

Other adverse events

Additional Information

Dr. Daniel Couriel

Huntsman Cancer Institute, University of Utah

Phone: 801-213-3082

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Corticosteriod Arm n=7 participants at risk Patients will receive 1 gram of IV SoluMedrol over 1 hour, once every month for 12 months. SoluMedrol: Infusion of drug subcutaneously, once a month.	Extracorporeal Photopheresis n=6 participants at risk Patient will receive an Extracorporeal Photopheresis treatment at a set frequency over the course of 1 year. The treatment takes about 2-3 hours per session to complete. The treatment schedule is as follows: ECP will be administered according to the following schedule: Study Arm: Weeks 1-8: 3 times per week Weeks 9-16: Twice per week Weeks 17-36: Treatment on two consecutive days every 2 weeks (or optionally, one treatment per week) Weeks 37-43: Once every 2 weeks Weeks 44-52: Once every 4 Weeks Extracorporeal Photopheresis: This intervention is the placement of up to two IV's to extract your blood as a set volume, separate out the white cells and return the red cells to your body. Then the white cells are treated with a drug called UVADEX (methoxsalen), excited by a UV light and returned to your body. Once IV is to withdraw your blood and the other is to return your blood to your body.
Respiratory, thoracic and mediastinal disorders Pneumonia	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 5 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Vascular disorders Poor Venous Access	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Respiratory, thoracic and mediastinal disorders Nocturnal Hypoxia (on PSG)	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Blood and lymphatic system disorders Iron Deficiency	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Blood and lymphatic system disorders Low Hematocrit Requiring Transfusion of 1 unit PRBC	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Blood and lymphatic system disorders Acute blood loss not requiring transfusion	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Vascular disorders Catheter Line Crack	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
General disorders "Jittery" Sensation during treatment	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
General disorders Dizziness	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Renal and urinary disorders Urinary tract infection	42.9% 3/7 • Number of events 5 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Renal and urinary disorders Acute Cystitis without Hematuria	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
General disorders Mild Hives	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Musculoskeletal and connective tissue disorders Knee Pain and Swelling	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
General disorders Chest pain	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Respiratory, thoracic and mediastinal disorders Obstructive Sleep Apnea	28.6% 2/7 • Number of events 2 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Nervous system disorders Mild Concussion	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Blood and lymphatic system disorders Low Hemoglobin	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Skin and subcutaneous tissue disorders Petechial Rash	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Vascular disorders Infiltrated IV with treatment	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	28.6% 2/7 • Number of events 2 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Musculoskeletal and connective tissue disorders Spinal Vertebral Lesions	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Musculoskeletal and connective tissue disorders Swelling	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Psychiatric disorders Anxiety Attacks	0.00% 0/7 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	16.7% 1/6 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Reproductive system and breast disorders Groin Pain	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Renal and urinary disorders Hematuria	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Skin and subcutaneous tissue disorders Pressure Ulcer	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
Ear and labyrinth disorders Ear Congestion	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.
General disorders Infusion Reaction	14.3% 1/7 • Number of events 1 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.	0.00% 0/6 • Adverse event data were collected on study participants from baseline visit to study completion, approximately 2 years.