Trial Outcomes & Findings for A Phase 2/3 Study of GLASSIA for the Treatment of Acute GvHD (NCT NCT02956122)

NCT ID: NCT02956122

Last Updated: 2021-01-13

Results Overview

OR was defined as graft-versus-host disease (GvHD) complete response (CR) + partial response (PR), defined as: - GvHD CR was complete resolution of all signs and symptoms of acute GvHD in all organs without intervening salvage and GvHD PR was improvement of 1 stage in 1 or more organs involved in GvHD without progression in other organs.

Recruitment status

TERMINATED

Study phase

PHASE2/PHASE3

Target enrollment

1 participants

Primary outcome timeframe

Day 28

Results posted on

2021-01-13

Participant Flow

Study was conducted between 26 April 2017 (first participant first visit) and 03 May 2018 (last participant last visit).

A total of 1 participant was enrolled and completed Part 1 (GLASSIA) of the study, and was analyzed for efficacy and safety with individual PK parameters estimated. Part 2 (GLASSIA versus albumin \[control\]) was not initiated following discontinuation of the study due to operational and business considerations.

Participant milestones

Participant milestones
Measure
GLASSIA
Participants received an intravenous (IV) infusion of GLASSIA at a dose of 90 milligrams per kilogram (mg/kg) on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 milligrams per kilogram per day (mg/kg/day) of methylprednisolone or equivalent steroid.
Overall Study
STARTED
1
Overall Study
COMPLETED
1
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase 2/3 Study of GLASSIA for the Treatment of Acute GvHD

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
Age, Categorical
<=18 years
0 Participants
n=5 Participants
Age, Categorical
Between 18 and 65 years
1 Participants
n=5 Participants
Age, Categorical
>=65 years
0 Participants
n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
1 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
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
1 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

PRIMARY outcome

Timeframe: Day 28

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable.

OR was defined as graft-versus-host disease (GvHD) complete response (CR) + partial response (PR), defined as: - GvHD CR was complete resolution of all signs and symptoms of acute GvHD in all organs without intervening salvage and GvHD PR was improvement of 1 stage in 1 or more organs involved in GvHD without progression in other organs.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Percentage of Participants Achieving Overall Response (OR) At Day 28
100 Percentage of participants

SECONDARY outcome

Timeframe: Day 28

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable.

GI response was defined as complete response (CR) + partial response (PR), defined as: - GI CR was able to eat; not requiring parenteral nutrition, and passing primarily formed stools - GI PR was decrease in need for parenteral nutrition to less than or equal to (\<=) 50% of required calories; and reduction of stool volume by greater than or equal to (\>=) 50%, without ileus.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Percentage of Participants Achieving Gastrointestinal (GI) Response at Day 28
100 Percentage of participants

SECONDARY outcome

Timeframe: Day 56

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable.

Overall response was defined as graft-versus-host disease (GvHD) complete response (CR) + partial response (PR), defined as: - GvHD CR was complete resolution of all signs and symptoms of acute GvHD in all organs without intervening salvage - GvHD PR was improvement of 1 stage in 1 or more organs involved in GvHD without progression in other organs.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Percentage of Participants Achieving Overall Response at Day 56
100 Percentage of participants

SECONDARY outcome

Timeframe: Days 28, 56 and 180

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable.

Grading of GvHD was performed by the investigator according to the modified International Bone Marrow Transplant Registry (IBMTR) grading system which classifies the degree of involvement of each organ system by stage on a scale of 0 to 4. The degree of skin involvement was staged depending upon degree and severity of the lesions: Stage 1: Maculopapular rash over less than (\<) 25% of body area, Stage 2: Maculopapular rash over 25 to 50% of body area, Stage 3: Generalized erythroderma, Stage 4: Generalized erythroderma with bullous formation. Degree of GI involvement was staged based on severity of diarrhoea: Stage 1: 500 to 1000 mL/day,Stage 2: 1000 to 1500 mL/day, Stage 3: 1500 to 2000 mL/day, Stage 4: greater than (\>) 2000 mL/day OR pain OR ileus. Degree of liver involvement was staged based upon serum total bilirubin level as follows: Stage 1: 2 to 3 mg/dL, Stage 2: 3 to 6 mg/dL, Stage 3: 6 to 15 mg/dL, Stage 4: \>15 mg/dL.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of skin Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of skin Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of liver Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of liver Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of liver Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of liver Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of skin Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of skin Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of skin Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of GI Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of GI Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of GI Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of GI Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of GI Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of liver Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of liver Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of liver Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 28: Degree of liver Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of skin Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of skin Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of skin Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of skin Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of skin Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of GI Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of GI Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of GI Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of GI Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of GI Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of liver Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of liver Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of liver Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of liver Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 56: Degree of liver Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of skin Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of skin Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of skin Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of skin Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of skin Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of GI Involvement · Stage 0
1 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of GI Involvement · Stage 1
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of GI Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of GI Involvement · Stage 3
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of GI Involvement · Stage 4
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of liver Involvement · Stage 2
0 Participants
Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180
Day 180: Degree of liver Involvement · Stage 4
0 Participants

SECONDARY outcome

Timeframe: Days 180 and 365

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable.

Incidence of chronic GvHD at Days 180 and 365 was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Incidence of Chronic Graft-versus-host Disease (GvHD)
Day 180
0 Participants
Incidence of Chronic Graft-versus-host Disease (GvHD)
Day 365
0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 365

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable. Here, number of participants analyzed refer to the participants evaluable for this outcome.

OR was defined as GvHD CR + PR, defined as: - GvHD CR was complete resolution of all signs and symptoms of acute GvHD in all organs without intervening salvage - GvHD PR was improvement of 1 stage in 1 or more organs involved in GvHD without progression in other organs. Duration of OR was not assessed due to the termination of the study.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline up to Day 365

Population: Efficacy analysis set included all participants who were evaluated for overall response at Day 28. Participants who received at least 1 dose of study treatment and who had a lower GI biopsy that was consistent with acute GvHD were considered evaluable. Here, number of participants analyzed refer to the participants evaluable for this outcome.

GI response was defined as CR + PR, defined as: - GI CR was able to eat; not requiring parenteral nutrition, and passing primarily formed stools - GI PR was decrease in need for parenteral nutrition to \<= 50% of required calories; and reduction of stool volume by \>= 50%, without ileus. Duration of GI response was not assessed due to the termination of the study.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Days 100, 180 and 365

Population: Safety analysis (SAF) set consisted of all participants who received at least 1 dose of study treatment.

OS was defined as the time from the date of randomization to the date of death due to any cause.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Overall Survival (OS) - Percentage of Participants With an Event
Day 100
NA Percentage of participants
No deaths were observed during the study.
Overall Survival (OS) - Percentage of Participants With an Event
Day 180
NA Percentage of participants
No deaths were observed during the study.
Overall Survival (OS) - Percentage of Participants With an Event
Day 365
NA Percentage of participants
No deaths were observed during the study.

SECONDARY outcome

Timeframe: Days 28, 56, 100 and 180

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant).

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Transplant-related Mortality
Day 28
0 Participants
Transplant-related Mortality
Day 56
0 Participants
Transplant-related Mortality
Day 100
0 Participants
Transplant-related Mortality
Day 180
0 Participants

SECONDARY outcome

Timeframe: Days 100 and 180

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Failure-free survival was defined as the absence of all of the following criteria: Need for second-line treatment for acute GvHD, Non-relapse mortality (death during continuous complete remission) and recurrent malignancy.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Failure-free Survival - Percentage of Participants With an Event
Day 100
100 Percentage of participants
Failure-free Survival - Percentage of Participants With an Event
Day 180
100 Percentage of participants

SECONDARY outcome

Timeframe: Days 28, 56, 100, 180 and 365

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

GVHD-free survival was defined as being alive without previous onset of acute GVHD or chronic GVHD requiring immunosuppressive therapy.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event
Day 365
100 Percentage of participants
Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event
Day 28
100 Percentage of participants
Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event
Day 56
100 Percentage of participants
Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event
Day 100
100 Percentage of participants
Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event
Day 180
100 Percentage of participants

SECONDARY outcome

Timeframe: Days 28, 56, 100 and 180

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Infection-related mortality was determined by the investigator (any deaths considered related to infection \[including infections related to hematopoietic stem cell transplant {HSCT}\]).

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Infection-related Mortality - Percentage of Participants With an Event
Day 28
0 Percentage of participants
Infection-related Mortality - Percentage of Participants With an Event
Day 56
0 Percentage of participants
Infection-related Mortality - Percentage of Participants With an Event
Day 100
0 Percentage of participants
Infection-related Mortality - Percentage of Participants With an Event
Day180
0 Percentage of participants

SECONDARY outcome

Timeframe: Days 28, 56, 100 and 180

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Graft-versus-host disease (GvHD)-related mortality was determined by the investigator (any deaths considered related to GvHD).

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event
Day 28
0 Percentage of participants
Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event
Day 56
0 Percentage of participants
Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event
Day 100
0 Percentage of participants
Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event
Day 180
0 Percentage of participants

SECONDARY outcome

Timeframe: Days 28, 56, 100 and 180

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

All-cause mortality was defined as the time from HSCT to death due to any cause.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
All-cause Mortality - Percentage of Participants With an Event
Day 28
0 Percentage of participants
All-cause Mortality - Percentage of Participants With an Event
Day 56
0 Percentage of participants
All-cause Mortality - Percentage of Participants With an Event
Day 100
0 Percentage of participants
All-cause Mortality - Percentage of Participants With an Event
Day180
0 Percentage of participants

SECONDARY outcome

Timeframe: From start of study drug administration up to 371 days

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

An AE was defined as any untoward medical occurrence in a participant administered an investigational product (IP) that does not necessarily have a causal relationship with the treatment. An SAE was defined as an untoward medical occurrence that at any dose meets one or more of the following criteria: outcome was fatal/results in death, life-threatening, required inpatient hospitalization or resulted in prolongation of an existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs
Any Adverse Event
1 Participants
Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs
Treatment-related AEs
0 Participants
Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs
Serious adverse Events (SAEs)
0 Participants
Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs
Treatment-related SAEs
0 Participants
Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs
Temporally-associated AEs
0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 56

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Clinical laboratory assessments such as hematology, clinical chemistry, lipid and coagulation panels and urinalysis were performed.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Number of Participants With Clinically Significant Changes in Clinical Laboratory Assessments
0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 56

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Vital signs included body temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Number of Participants With Clinically Significant Changes in Vital Signs
0 Participants

SECONDARY outcome

Timeframe: Baseline up to Day 365

Population: SAF set consisted of all participants who received at least 1 dose of study treatment.

Incidence of recurrence of primary malignancies was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Number of Participants With Recurrence of Primary Malignancies
0 Participants

SECONDARY outcome

Timeframe: Day 1: through 48 hours; Day 13: through 48 hours; Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

AUC of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
Area Under the Plasma Concentration Curve (AUC0-inf) From Time Zero to Infinity
61500 Hour*milligrams per deciliter (h*mg/dL)
43500 Hour*milligrams per deciliter (h*mg/dL)
126000 Hour*milligrams per deciliter (h*mg/dL)
117000 Hour*milligrams per deciliter (h*mg/dL)

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

AUC(0-t) of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
Area Under the Plasma Concentration Curve From Time Zero to Time "t" AUC(0-t) of GLASSIA
16300 h*mg/dL
11000 h*mg/dL
40400 h*mg/dL
33400 h*mg/dL

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

CLss of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Systemic Clearance at Steady State (CLss) of GLASSIA
0.297 Deciliters per hour (dL/h)
0.286 Deciliters per hour (dL/h)
0.260 Deciliters per hour (dL/h)

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

Cmax of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
Maximum Observed Plasma Concentration (Cmax) of GLASSIA
339 Milligrams per deciliter (mg/dl)
262 Milligrams per deciliter (mg/dl)
390 Milligrams per deciliter (mg/dl)
409 Milligrams per deciliter (mg/dl)

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

Vss of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
Participants received an IV infusion of 120 mg/kg GLASSIA.
Apparent Volume of Distribution at Steady State (Vss) of GLASSIA
50.9 Deciliter (dL)
123 Deciliter (dL)
91.1 Deciliter (dL)

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected.

Apparent terminal half-life (hour), determined as ln2/lambda-z. lambda-z is the apparent terminal rate constant (one per hour), determined by linear regression of the terminal points of the log-linear concentration-time curve. Visual assessment will be used to identify the terminal linear phase of the concentration-time profile. A minimum of 3 data points will be used for determination. t1/2 of GLASSIA was reported.

Outcome measures

Outcome measures
Measure
GLASSIA
n=1 Participants
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
GLASSIA: Day 13: 30 mg/kg
n=1 Participants
Participants received an IV infusion of 30 mg/kg GLASSIA.
GLASSIA: Day 22: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
GLASSIA: Day 50: 120 mg/kg
n=1 Participants
Participants received an IV infusion of 120 mg/kg GLASSIA.
Apparent Terminal Half-life (t1/2) of GLASSIA
152 Hour (h)
117 Hour (h)
317 Hour (h)
247 Hour (h)

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. Her, the number of participants analyzed refer to the participants evaluable for this outcome at specified time point.

MRT of GLASSIA was not calculated.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours

Population: PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. Here, number of participants analyzed refer to the participants evaluable for this outcome at specified time point.

Ctrough of GLASSIA was not assessed due to the termination of the study.

Outcome measures

Outcome data not reported

Adverse Events

GLASSIA

Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
GLASSIA
n=1 participants at risk
Participants received an IV infusion of GLASSIA at a dose of 90 mg/kg on Day 1 followed by 30 mg/kg every other day (Days 3 to 13), then followed by 120 mg/kg weekly (Days 15 to 50) along with 2 mg/kg/day of methylprednisolone or equivalent steroid.
Investigations
Platelet count decreased/
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Skin and subcutaneous tissue disorders
Pruritus
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hyperglycaemia
100.0%
1/1 • Number of events 6 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypocalcaemia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Eye disorders
Vision blurred
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypokalaemia
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Investigations
Weight decreased
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Investigations
Blood cholesterol increased
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypomagnesaemia
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Musculoskeletal and connective tissue disorders
Arthralgia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Respiratory, thoracic and mediastinal disorders
Cough
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Musculoskeletal and connective tissue disorders
Myalgia
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypoalbuminaemia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Infections and infestations
Sinusitis
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Vascular disorders
Hot flush
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Cardiac disorders
Sinus tachycardia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Investigations
White blood cell count decreased
100.0%
1/1 • Number of events 3 • From the start of drug administration up to 371 days
General disorders
Injection site reaction
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Gastrointestinal disorders
Toothache
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
General disorders
Fatigue
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Infections and infestations
Mucosal infection
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
General disorders
Localised oedema
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypophosphataemia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Metabolism and nutrition disorders
Hypercalcaemia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Blood and lymphatic system disorders
Anaemia
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Infections and infestations
Cellulitis
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Nervous system disorders
Peripheral motor neuropathy
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Nervous system disorders
Amnesia
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Gastrointestinal disorders
Gastrooesophageal reflux disease
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Investigations
Bilirubin conjugated increased
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Gastrointestinal disorders
Dry mouth
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Investigations
Lymphocyte count decreased
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Nervous system disorders
Headache
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Respiratory, thoracic and mediastinal disorders
Nasal congestion
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Respiratory, thoracic and mediastinal disorders
Dyspnoea
100.0%
1/1 • Number of events 2 • From the start of drug administration up to 371 days
Investigations
Neutrophil count decreased
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Gastrointestinal disorders
Tongue ulceration
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Skin and subcutaneous tissue disorders
Dry skin
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days
Skin and subcutaneous tissue disorders
Eczema
100.0%
1/1 • Number of events 1 • From the start of drug administration up to 371 days

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Results disclosure agreements

  • Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonmentor termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
  • Publication restrictions are in place

Restriction type: OTHER