Not provided
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The decision to discontinue the study was based on business reasons.
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| Name | Class |
|---|---|
| Kamada, Ltd. | INDUSTRY |
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The purpose of the study is to evaluate the safety and efficacy of GLASSIA as an add-on biopharmacotherapy to standard-of-care steroid treatment as the first-line treatment in participants with acute GvHD with lower GI involvement.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Study Part 1 - All Participants - GLASSIA | Experimental | Participants to receive GLASSIA (intravenously) and methylprednisolone or equivalent steroid (either IV or oral per investigator discretion) |
|
| Study Part 2 - GLASSIA | Experimental | Participants to receive GLASSIA (intravenously) and methylprednisolone or equivalent steroid (either IV or oral per investigator discretion) |
|
| Study Part 2 - Albumin (Control) | Placebo Comparator | Participants to receive control (intravenously) and methylprednisolone or equivalent steroid (either IV or oral per investigator discretion) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GLASSIA | Biological | GLASSIA [Alpha1-Proteinase Inhibitor (Human)] |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Achieving Overall Response (OR) At Day 28 | 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. | Day 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Achieving Gastrointestinal (GI) Response at Day 28 | 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Shire | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Georgia Cancer Center | Augusta | Georgia | 30912 | United States |
De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the low number of study participants).
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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.
Study was conducted between 26 April 2017 (first participant first visit) and 03 May 2018 (last participant last visit).
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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| ID | Title | Description |
|---|---|---|
| BG000 | GLASSIA | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of Participants Achieving Overall Response (OR) At Day 28 | 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. | 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. | Posted | Number | Percentage of participants | Day 28 |
|
From the start of drug administration up to 371 days
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GLASSIA | 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Platelet count decreased/ | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 19, 2017 | Mar 21, 2019 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 13, 2017 | Mar 21, 2019 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006086 | Graft vs Host Disease |
| ID | Term |
|---|---|
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| D008775 | Methylprednisolone |
| D000418 | Albumins |
| D000075462 | Serum Albumin, Human |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
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| methylprednisolone or equivalent steroid | Drug | The conventional steroid treatment (methylprednisolone or equivalent steroid) will be supplied by the investigators per their institutional practice. |
|
| Albumin | Biological | The control vials contain human albumin 20% in 50 mL normal saline solution in glass vials (for non-United States (US) Countries), or Flexbumin 25% in 50 mL in normal saline solution in plastic IV bags (for US). |
|
|
| Day 28 |
| Percentage of Participants Achieving Overall Response at Day 56 | 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. | Day 56 |
| Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180 | 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. | Days 28, 56 and 180 |
| Incidence of Chronic Graft-versus-host Disease (GvHD) | Incidence of chronic GvHD at Days 180 and 365 was reported. | Days 180 and 365 |
| Duration of Overall Response (OR) | 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. | Baseline up to Day 365 |
| Duration of Gastrointestinal (GI) Response | 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. | Baseline up to Day 365 |
| Overall Survival (OS) - Percentage of Participants With an Event | OS was defined as the time from the date of randomization to the date of death due to any cause. | Days 100, 180 and 365 |
| Transplant-related Mortality | Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant). | Days 28, 56, 100 and 180 |
| Failure-free Survival - Percentage of Participants With an Event | 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. | Days 100 and 180 |
| Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event | GVHD-free survival was defined as being alive without previous onset of acute GVHD or chronic GVHD requiring immunosuppressive therapy. | Days 28, 56, 100, 180 and 365 |
| Infection-related Mortality - Percentage of Participants With an Event | Infection-related mortality was determined by the investigator (any deaths considered related to infection [including infections related to hematopoietic stem cell transplant {HSCT}]). | Days 28, 56, 100 and 180 |
| Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event | Graft-versus-host disease (GvHD)-related mortality was determined by the investigator (any deaths considered related to GvHD). | Days 28, 56, 100 and 180 |
| All-cause Mortality - Percentage of Participants With an Event | All-cause mortality was defined as the time from HSCT to death due to any cause. | Days 28, 56, 100 and 180 |
| Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs | 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. | From start of study drug administration up to 371 days |
| Number of Participants With Clinically Significant Changes in Clinical Laboratory Assessments | Clinical laboratory assessments such as hematology, clinical chemistry, lipid and coagulation panels and urinalysis were performed. | Baseline up to Day 56 |
| Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs included body temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure. | Baseline up to Day 56 |
| Number of Participants With Recurrence of Primary Malignancies | Incidence of recurrence of primary malignancies was reported. | Baseline up to Day 365 |
| Area Under the Plasma Concentration Curve (AUC0-inf) From Time Zero to Infinity | AUC of GLASSIA was reported. | Day 1: through 48 hours; Day 13: through 48 hours; Day 22 and Day 50: through approximately 168 hours |
| Area Under the Plasma Concentration Curve From Time Zero to Time "t" AUC(0-t) of GLASSIA | AUC(0-t) of GLASSIA was reported. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Systemic Clearance at Steady State (CLss) of GLASSIA | CLss of GLASSIA was reported. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Maximum Observed Plasma Concentration (Cmax) of GLASSIA | Cmax of GLASSIA was reported. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Apparent Volume of Distribution at Steady State (Vss) of GLASSIA | Vss of GLASSIA was reported. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Apparent Terminal Half-life (t1/2) of GLASSIA | 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. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Mean Residence Time (MRT) of GLASSIA | MRT of GLASSIA was not calculated. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Trough Plasma Concentration at Steady State (Ctrough) of GLASSIA | Ctrough of GLASSIA was not assessed due to the termination of the study. | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
|
|
| Secondary | Percentage of Participants Achieving Gastrointestinal (GI) Response at Day 28 | 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. | 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. | Posted | Number | Percentage of participants | Day 28 |
|
|
|
| Secondary | Percentage of Participants Achieving Overall Response at Day 56 | 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. | 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. | Posted | Number | Percentage of participants | Day 56 |
|
|
|
| Secondary | Acute Graft-versus-host Disease (GvHD) Grading at Days 28, 56 and 180 | 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. | 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. | Posted | Count of Participants | Participants | Days 28, 56 and 180 |
|
|
|
| Secondary | Incidence of Chronic Graft-versus-host Disease (GvHD) | Incidence of chronic GvHD at Days 180 and 365 was reported. | 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. | Posted | Number | Participants | Days 180 and 365 |
|
|
|
| Secondary | Duration of Overall Response (OR) | 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. | 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. | Posted | Baseline up to Day 365 |
|
|
| Secondary | Duration of Gastrointestinal (GI) Response | 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. | 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. | Posted | Baseline up to Day 365 |
|
|
| Secondary | Overall Survival (OS) - Percentage of Participants With an Event | OS was defined as the time from the date of randomization to the date of death due to any cause. | Safety analysis (SAF) set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 100, 180 and 365 |
|
|
|
| Secondary | Transplant-related Mortality | Transplant-related mortality was determined by the investigator (any deaths considered related to the transplant). | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | Days 28, 56, 100 and 180 |
|
|
|
| Secondary | Failure-free Survival - Percentage of Participants With an Event | 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. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 100 and 180 |
|
|
|
| Secondary | Graft-versus-host Disease (GvHD)-Free Survival - Percentage of Participants With an Event | GVHD-free survival was defined as being alive without previous onset of acute GVHD or chronic GVHD requiring immunosuppressive therapy. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 28, 56, 100, 180 and 365 |
|
|
|
| Secondary | Infection-related Mortality - Percentage of Participants With an Event | Infection-related mortality was determined by the investigator (any deaths considered related to infection [including infections related to hematopoietic stem cell transplant {HSCT}]). | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 28, 56, 100 and 180 |
|
|
|
| Secondary | Graft-versus-host Disease (GvHD)-Related Mortality - Percentage of Participants With an Event | Graft-versus-host disease (GvHD)-related mortality was determined by the investigator (any deaths considered related to GvHD). | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 28, 56, 100 and 180 |
|
|
|
| Secondary | All-cause Mortality - Percentage of Participants With an Event | All-cause mortality was defined as the time from HSCT to death due to any cause. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Number | Percentage of participants | Days 28, 56, 100 and 180 |
|
|
|
| Secondary | Number of Participants With Adverse Events (AEs), Treatment-related AEs, Serious Adverse Events (SAEs), Treatment-related SAEs and Temporally-associated AEs | 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. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | From start of study drug administration up to 371 days |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Clinical Laboratory Assessments | Clinical laboratory assessments such as hematology, clinical chemistry, lipid and coagulation panels and urinalysis were performed. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | Baseline up to Day 56 |
|
|
|
| Secondary | Number of Participants With Clinically Significant Changes in Vital Signs | Vital signs included body temperature, respiratory rate, pulse rate and systolic and diastolic blood pressure. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | Baseline up to Day 56 |
|
|
|
| Secondary | Number of Participants With Recurrence of Primary Malignancies | Incidence of recurrence of primary malignancies was reported. | SAF set consisted of all participants who received at least 1 dose of study treatment. | Posted | Count of Participants | Participants | Baseline up to Day 365 |
|
|
|
| Secondary | Area Under the Plasma Concentration Curve (AUC0-inf) From Time Zero to Infinity | AUC of GLASSIA was reported. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | Hour*milligrams per deciliter (h*mg/dL) | Day 1: through 48 hours; Day 13: through 48 hours; Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Area Under the Plasma Concentration Curve From Time Zero to Time "t" AUC(0-t) of GLASSIA | AUC(0-t) of GLASSIA was reported. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | h*mg/dL | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Systemic Clearance at Steady State (CLss) of GLASSIA | CLss of GLASSIA was reported. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | Deciliters per hour (dL/h) | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Maximum Observed Plasma Concentration (Cmax) of GLASSIA | Cmax of GLASSIA was reported. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | Milligrams per deciliter (mg/dl) | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Apparent Volume of Distribution at Steady State (Vss) of GLASSIA | Vss of GLASSIA was reported. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | Deciliter (dL) | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Apparent Terminal Half-life (t1/2) of GLASSIA | 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. | PK Analysis set included all participants in the SAF set who have at least 1 PK or stool sample collected. | Posted | Number | Hour (h) | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
|
| Secondary | Mean Residence Time (MRT) of GLASSIA | MRT of GLASSIA was not calculated. | 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. | Posted | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
| Secondary | Trough Plasma Concentration at Steady State (Ctrough) of GLASSIA | Ctrough of GLASSIA was not assessed due to the termination of the study. | 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. | Posted | Day 1: through 48 hours, Day 13: through 48 hours, Day 22 and Day 50: through approximately 168 hours |
|
|
| 0 |
| 1 |
| 0 |
| 1 |
| 1 |
| 1 |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypocalcaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Vision blurred | Eye disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Weight decreased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Blood cholesterol increased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Sinusitis | Infections and infestations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hot flush | Vascular disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Sinus tachycardia | Cardiac disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| White blood cell count decreased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Injection site reaction | General disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Fatigue | General disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Mucosal infection | Infections and infestations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Localised oedema | General disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Cellulitis | Infections and infestations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Peripheral motor neuropathy | Nervous system disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Amnesia | Nervous system disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Bilirubin conjugated increased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Dry mouth | Gastrointestinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Lymphocyte count decreased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Neutrophil count decreased | Investigations | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Tongue ulceration | Gastrointestinal disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | MedDRA Version 20.1 | Non-systematic Assessment |
|
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.
| D010455 |
| Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D011239 | Prednisolone |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D012709 | Serum Albumin |
| Stage 3 |
|
| Stage 4 |
|
| Day 28: Degree of GI Involvement |
|
| Day 28: Degree of liver Involvement |
|
| Day 56: Degree of skin Involvement |
|
| Day 56: Degree of GI Involvement |
|
| Day 56: Degree of liver Involvement |
|
| Day 180: Degree of skin Involvement |
|
| Day 180: Degree of GI Involvement |
|
| Day 180: Degree of liver Involvement |
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|
|
| Day 180 |
|
| Title | Measurements |
|---|
|
| Day 180 |
|
| Day 365 |
|
| Title | Measurements |
|---|
|
| Day180 |
|
| Title | Measurements |
|---|
|
| Day 180 |
|
| Title | Measurements |
|---|
|
| Day180 |
|
| Title | Measurements |
|---|---|
|
| Treatment-related SAEs |
|
| Temporally-associated AEs |
|