Trial Outcomes & Findings for Efalizumab in Treating Patients With Graft-Versus-Host Disease of the Skin That Did Not Respond to Previous Steroids (NCT NCT00489216)

Results Overview

The primary objective of this exploratory study is to evaluate the general tolerability of efalizumab in patients suffering from steroid refractory GVHD Subjects will be evaluated for drug toxicity each visit. Toxicity will be graded using the Common Terminology Criteria for Adverse Events (CTCAE) common criteria.

Recruitment status

TERMINATED

Study phase

NA

Target enrollment

2 participants

Primary outcome timeframe

120 days

Results posted on

2017-05-23

Participant Flow

Patients with evidence of an inflammatory, erythematous rash and skin biopsy consistent with cutaneous Graft-Versus-Host disease who had failed to improve after at least 4 weeks of methylprednisolone at 0.5mg/kg were eligible for enrollment.

Participant milestones

Participant milestones
Measure	Efalizumab All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Overall Study STARTED	2
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Efalizumab All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Overall Study Adverse Event	1

Baseline Characteristics

Efalizumab in Treating Patients With Graft-Versus-Host Disease of the Skin That Did Not Respond to Previous Steroids

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efalizumab n=2 Participants All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	1 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	2 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	2 participants n=5 Participants

PRIMARY outcome

Timeframe: 120 days

Population: Of the two patients, one patient completed all 8 doses of study medication according to schedule. The other patient received 6/8 scheduled efalizumab doses, but was subsequently taken off the study after developing transient coagulase negative staphylococcal bacteremia.

The primary objective of this exploratory study is to evaluate the general tolerability of efalizumab in patients suffering from steroid refractory GVHD Subjects will be evaluated for drug toxicity each visit. Toxicity will be graded using the Common Terminology Criteria for Adverse Events (CTCAE) common criteria.

Outcome measures

Outcome measures
Measure	Efalizumab n=2 Participants All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Number of Subjects Experiencing Adverse Events	2 participants

PRIMARY outcome

Timeframe: 120 days

Population: Data was not collected for this objective due to limited number of participants. A minimum of 12 patients were needed for analysis.

Estimate of the percentage of body surface area involved by GVHD using two digital photography techniques and computerized image analyses: 1. Digital photography and body surface area calculations: A total of 12 digital photographs were obtained from different body regions using systematic digital imaging and computerized image analysis. 2. Body surface area calculations: Using National Institutes of Health image software, each body region will be manually traced and the total area of the traced area determined. Using a similar technique, each part of the region that is involved by a GVHD rash will also be traced and its area measured. The areas involved by rash will then be summed, and finally divided by the total area of the region. In so doing, the percentage of each region that is involved by GVHD will be determined.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 57 days

Population: There is no data for this outcome measure because of the small number of patients and inability to make meaningful conclusions

Numerical scoring system for both LFA-1 and ICAM-1 expression which could then be used in a larger phase II trial to correlate clinical response rates to pathological findings.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 57 days

Population: Of the two patients, one patient completed all 8 doses of study medication according to schedule. The other patient received 6/8 scheduled efalizumab doses, but was subsequently taken off the study after developing transient coagulase negative staphylococcal bacteremia. The patient demonstrated a complete response (CR).

To assess the overall complete response rate on study days 29 and 57 after 4 and 8 doses of weekly subcutaneous efalizumab. A complete response (CR) was defined as the total resolution of skin disease, and a partial response was defined as \>=50% reduction in the proportion of total body surface area involved by rash.

Outcome measures

Outcome measures
Measure	Efalizumab n=2 Participants All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Overall Complete Response Rate	1 Participants

SECONDARY outcome

Timeframe: On study days 29 and 57 after 4 and 8 doses of weekly subcutaneous efalizumab

The complete disappearance of all signs of cutaneous graft-versus-host disease. Complete cutaneous response rate + partial cutaneous response rate

Outcome measures

Outcome measures
Measure	Efalizumab n=2 Participants All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Complete Cutaneous Response Rate	1 participants

SECONDARY outcome

Timeframe: On study days 29 and 57 after 4 and 8 doses of weekly subcutaneous efalizumab

Population: There is no data for this outcome measure as no enrolled patients had an elevated bilirubin level.

The normalization of the total serum bilirubin to \<2mg/dL

Outcome measures

Outcome data not reported

Adverse Events

Efalizumab

Serious events: 1 serious events

Other events: 1 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Efalizumab n=2 participants at risk All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Infections and infestations Transient Coagulase Negative Staphylococcal Bacteremia	50.0% 1/2 • Final efalizumab dose administered on day 57. Patients observed for 8 additional weeks.

Other adverse events

Other adverse events
Measure	Efalizumab n=2 participants at risk All patients on study will receive a total of 8 injections of efalizumab efalizumab: Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at 1mg/kg
Gastrointestinal disorders Nausea	50.0% 1/2 • Final efalizumab dose administered on day 57. Patients observed for 8 additional weeks.

Additional Information

Robin V. Johnson

UNC Lineberger Comprehensive Cancer Center

Phone: 919-966-1125

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place