Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency (SPARK)

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Grifols Therapeutics Inc.

ClinicalTrials.gov Identifier:

NCT01213043

First received: September 29, 2010

Last updated: April 8, 2013

Last verified: April 2013

History of Changes

Results First Received: February 26, 2013

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Pharmacokinetics Study; Intervention Model: Crossover Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Conditions:	Emphysema Alpha 1-antitrypsin Deficiency (AATD)
Interventions:	Biological: Prolastin-C, 60 mg/kg Biological: Prolastin-C, 120 mg/kg

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Subjects entered a Screening Phase (up to 21 days in duration) to determine subject eligibility and for wash-out of prior alpha1-PI augmentation therapy, if applicable, prior to randomization to one of two treatment sequences.

Reporting Groups

	Description
60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	Weekly infusions of 60 mg/kg Prolastin-C for 8 weeks followed by a 2-week off-treatment washout period followed by weekly infusions of 120 mg/kg Prolastin-C for 8 weeks (total of 16 treatment weeks)
120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence	Weekly infusions of 120 mg/kg Prolastin-C for 8 weeks followed by a 2-week off-treatment washout period followed by weekly infusions of 60 mg/kg Prolastin-C for 8 weeks (total of 16 treatment weeks)

Participant Flow for 4 periods

Period 1: Treatment Period 1

	60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence
STARTED	15	15
COMPLETED	15	15
NOT COMPLETED	0	0

Period 2: Washout Period

	60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence
STARTED	15	15
COMPLETED	15	15
NOT COMPLETED	0	0

Period 3: Treatment Period 2

	60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence
STARTED	15	15
COMPLETED	15	15
NOT COMPLETED	0	0

Period 4: Follow-up

	60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence
STARTED	15	15
COMPLETED	15	15
NOT COMPLETED	0	0

Baseline Characteristics

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Reporting Groups

	Description
60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	Weekly infusions of 60 mg/kg Prolastin-C for 8 weeks followed by a 2-week off-treatment washout period followed by weekly infusions of 120 mg/kg Prolastin-C for 8 weeks (total of 16 treatment weeks)
120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence	Weekly infusions of 120 mg/kg Prolastin-C for 8 weeks followed by a 2-week off-treatment washout period followed by weekly infusions of 60 mg/kg Prolastin-C for 8 weeks (total of 16 treatment weeks)
Total	Total of all reporting groups

Baseline Measures

	60 mg/kg - 120 mg/kg Prolastin-C Treatment Sequence	120 mg/kg - 60 mg/kg Prolastin-C Treatment Sequence	Total
Number of Participants [units: participants]	15	15	30
Age [units: years] Mean ± Standard Deviation	59.7 ± 6.89	57.4 ± 6.34	58.6 ± 6.62
Gender [units: participants]
Female	8	8	16
Male	7	7	14
Race (NIH/OMB) [units: participants]
American Indian or Alaska Native	0	0	0
Asian	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	0	0	0
White	15	15	30
More than one race	0	0	0
Unknown or Not Reported	0	0	0

Outcome Measures

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1. Primary:

Subjects With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: 22 weeks ]

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2. Primary:

Subjects With Drug-Related TEAE(s) [ Time Frame: 22 weeks ]

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3. Primary:

Subjects With Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: 22 weeks ]

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4. Primary:

Subjects Withdrawn Due to an AE(s) [ Time Frame: 22 weeks ]

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5. Primary:

Subjects With Treatment-Emergent Pulmonary Exacerbation(s) [ Time Frame: 22 weeks ]

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6. Primary:

Subjects With Severe TEAE(s) or Pulmonary Exacerbation(s) [ Time Frame: 22 weeks ]

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7. Primary:

Number of TEAEs [ Time Frame: 22 Weeks ]

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8. Primary:

Number of Drug-related TEAEs [ Time Frame: 22 Weeks ]

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9. Primary:

Number of Treatment-Emergent Pulmonary Exacerbations [ Time Frame: 22 Weeks ]

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10. Secondary:

AUC0-7days [ Time Frame: Week 8 and Week 18 at the following timepoints: 0 (pre-infusion), completion of first infusion bag, completion of 2nd infusion bag, and 15 min, 30 min, and 1, 2, 4, 8, 24, 48, 120, and 168 hours post-dose ]

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11. Secondary:

Mean Trough [ Time Frame: Single measurment immediately prior to infusion at Weeks 6, 7, 8, 9 and Weeks 16, 17, 18, 19 ]

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Serious Adverse Events

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Other Adverse Events

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: No text entered.

Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.

Results Point of Contact:

Name/Title: Benjamin King, PhD
Organization: Grifols Therapeutics Inc.
e-mail: ben.king@grifols.com

Publications:

Willis T, Wee K, Mohn G. A high-purity Alpha-1 proteinase inhibitor from human plasma, TAL6004. Proceeding of the 19th European Respiratory Society Annual Congress; 2009 Sep 12-16; Vienna, Austria. Abstracts;34:S53.

Responsible Party:	Grifols Therapeutics Inc.
ClinicalTrials.gov Identifier:	NCT01213043 History of Changes
Other Study ID Numbers:	T6004-201/Version 2
Study First Received:	September 29, 2010
Results First Received:	February 26, 2013
Last Updated:	April 8, 2013
Health Authority:	United States: Food and Drug Administration

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