Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
The purpose of this study was to generate sufficient safety and tolerability information in support of an increase in the infusion rate of intravenous GLASSIA in the prescribing information from 0.04 to 0.2 mL/kg/min.
To achieve proper masking, 30 participants were randomly assigned to receive either GLASSIA at 0.04 mL/kg/min with a simultaneous administration of placebo (2.5% human albumin in normal saline) at 0.2 mL/kg/min (Cohort 1) or GLASSIA at 0.2 mL/kg/min with a simultaneous administration of placebo at 0.04 mL/kg/min (Cohort 2) on Day 1.
Two weeks later (Day 15), the same participants received the second infusion with the opposite rate of GLASSIA infusion and the corresponding masking placebo infusion.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Day 1:
Day 15:
|
|
| Cohort 2 | Experimental | Day 1:
Day 15:
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alpha1-proteinase inhibitor | Biological | GLASSIA will be supplied as a sterile, non-pyrogenic, ready-to-use solution, in single dose 50 mL vials; for intravenous administration. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Infusions Associated With a Reduction in Infusion Rate or Discontinuation of Infusion Due to an Adverse Event (Regardless of Adverse Event Causality Assessment) | Day 1 and Day 15 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 1 Hour of Infusion Completion | Number of infusions with temporally associated AEs with an onset time during or within 1 hour of infusion completion, regardless of causality assessment | Within 1 hour of infusion completion |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Overland Park | Kansas | 66211 | United States |
Thirty five healthy potential participants were enrolled at the clinical study site. Four were screen failures, and one was a back up participant who did not participate. Therefore, 30 participants were randomized.
Recruitment was conducted in the U.S at 1 study site. The first participant was enrolled in July 2012.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Day 1: - GLASSIA at 0.04 mL/kg/min - Placebo at 0.2 mL/kg/min Day 15: - GLASSIA at 0.2 mL/kg/min - Placebo at 0.04 mL/kg/min Alpha1-proteinase inhibitor: GLASSIA will be supplied as a sterile, non-pyrogenic, ready-to-use solution, in single dose 50 mL vials; for intravenous administration. Placebo: Human albumin 2.5%: Intravenous administration |
| FG001 | Cohort 2 | Day 1: - GLASSIA at 0.2 mL/kg/min - Placebo at 0.04 mL/kg/min Day 15: - GLASSIA at 0.04 mL/kg/min - Placebo at 0.2 mL/kg/min Alpha1-proteinase inhibitor: GLASSIA will be supplied as a sterile, non-pyrogenic, ready-to-use solution, in single dose 50 mL vials; for intravenous administration. Placebo: Human albumin 2.5%: Intravenous administration |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Safety Analysis Set
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | Day 1: - GLASSIA at 0.04 mL/kg/min - Placebo at 0.2 mL/kg/min Day 15: - GLASSIA at 0.2 mL/kg/min - Placebo at 0.04 mL/kg/min Placebo: Human albumin 2.5%: Intravenous administration |
| BG001 | Cohort 2 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 | Number of Infusions Associated With a Reduction in Infusion Rate or Discontinuation of Infusion Due to an Adverse Event (Regardless of Adverse Event Causality Assessment) | Safety Analysis Set | Posted | Number | Infusions | Day 1 and Day 15 |
|
|
Throughout the study period of 105 days per participant
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | GLASSIA Infusion Rate- 0.04 mL/kg/Min | Participants who received simultaneous infusions of: - GLASSIA at 0.04 mL/kg/min - Placebo at 0.2 mL/kg/min |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
Simultaneous infusion of GLASSIA and placebo did not allow adverse events (AEs) to be unquestionably ascribed to either one. Any observed AE which was assessed by the investigator as related to treatment was conservatively attributed to GLASSIA.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
Not provided
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D004194 | Disease |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
| Placebo: Human albumin 2.5% | Biological | Intravenous administration |
|
| Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 24 Hours of Completion of an Infusion |
Number of infusions with temporally associated AEs with an onset time during or within 24 hours of infusion completion, regardless of causality assessment |
| Within 24 hours of the end of infusion |
| Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 72 Hours of Completion of an Infusion | Number of infusions with temporally associated AEs with an onset time during or within 72 hours of infusion completion, regardless of causality assessment | Within 72 hours of the end of infusion |
| Number of Possibly or Probably Related Adverse Events (AEs) That Began During an Infusion | Number of AEs that occurred during an infusion and were deemed related to study product administration | Day 1 and Day 15 |
| Number of Possibly or Probably Related Adverse Events That Occurred Between 72 Hours and 14 Days After Infusion | Number of AEs that occurred between 72 hours and 14 day following an infusion and were deemed related to study product administration | 72 hours post infusion to 14 days post infusion |
| Number of Participants Testing Positive for Hepatitis A Virus (HAV), Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Parvovirus B19 (PVB19) or Human Immunodeficiency Virus (HIV) Following Treatment With GLASSIA | Number of participants with seroconversion | 105 days |
Day 1: - GLASSIA at 0.2 mL/kg/min - Placebo at 0.04 mL/kg/min Day 15: - GLASSIA at 0.04 mL/kg/min - Placebo at 0.2 mL/kg/min Placebo: Human albumin 2.5%: Intravenous administration
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
| Secondary | Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 1 Hour of Infusion Completion | Number of infusions with temporally associated AEs with an onset time during or within 1 hour of infusion completion, regardless of causality assessment | Safety Analysis Set | Posted | Number | Infusions | Within 1 hour of infusion completion |
|
|
|
| Secondary | Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 24 Hours of Completion of an Infusion | Number of infusions with temporally associated AEs with an onset time during or within 24 hours of infusion completion, regardless of causality assessment | Safety Analysis Set | Posted | Number | Infusions | Within 24 hours of the end of infusion |
|
|
|
| Secondary | Number of Infusions With Temporally Associated Adverse Events (AEs) That Began During or Within 72 Hours of Completion of an Infusion | Number of infusions with temporally associated AEs with an onset time during or within 72 hours of infusion completion, regardless of causality assessment | Safety Analysis Set | Posted | Number | Infusions | Within 72 hours of the end of infusion |
|
|
|
| Secondary | Number of Possibly or Probably Related Adverse Events (AEs) That Began During an Infusion | Number of AEs that occurred during an infusion and were deemed related to study product administration | Safety Analysis Set | Posted | Number | adverse events | Day 1 and Day 15 |
|
|
|
| Secondary | Number of Possibly or Probably Related Adverse Events That Occurred Between 72 Hours and 14 Days After Infusion | Number of AEs that occurred between 72 hours and 14 day following an infusion and were deemed related to study product administration | Safety Analysis Set | Posted | Number | adverse events | 72 hours post infusion to 14 days post infusion |
|
|
|
| Secondary | Number of Participants Testing Positive for Hepatitis A Virus (HAV), Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Parvovirus B19 (PVB19) or Human Immunodeficiency Virus (HIV) Following Treatment With GLASSIA | Number of participants with seroconversion | Safety Analysis Set | Posted | Number | participants | 105 days |
|
|
|
| 0 |
| 30 |
| 8 |
| 30 |
| EG001 | GLASSIA Infusion Rate- 0.2 mL/kg/Min | Participants who received simultaneous infusions of: - GLASSIA at 0.2 mL/kg/min - Placebo at 0.04 mL/kg/min | 0 | 30 | 4 | 30 |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) |
|
| Dizziness | Nervous system disorders | MedDRA (Unspecified) |
|
PI may not publish without the prior written consent of Sponsor
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| 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 |
| PVB19 |
|
| HIV |
|