Trial Outcomes & Findings for The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency (NCT NCT00301366)

Last Updated: 2014-08-19

Results Overview

An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

38 participants

Primary outcome timeframe

24 weeks

Results posted on

2014-08-19

Participant Flow

First-subject-first-dose was 12 June 2006, last-subject-last-visit was 20 March 2007. The study was performed in 10 clinical sites, 8 sites in the United States and 2 sites in the United Kingdom.

Participant milestones

Participant milestones
Measure	Alpha-1MP Treatment Group All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks
Overall Study STARTED	38
Overall Study COMPLETED	37
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Alpha-1MP Treatment Group All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks
Overall Study Adverse Event	1

Baseline Characteristics

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Entered Study n=38 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	32 Participants n=5 Participants
Age, Categorical >=65 years	6 Participants n=5 Participants
Age, Continuous	53.4 years STANDARD_DEVIATION 8.63 • n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants
Region of Enrollment United States	35 participants n=5 Participants
Region of Enrollment United Kingdom	3 participants n=5 Participants

PRIMARY outcome

Timeframe: 24 weeks

Population: 38 subjects received study medication and one subject discontinued from the study due to an AE. Therefore, 37 subjects completed the study. 18 subjects were naive ((i.e., never having received previous Alpha-1 protease inhibitor augmentation therapy) and 19 subjects were non-naive.

Outcome measures

Outcome measures
Measure	Alpha-1 MP Treatment Group n=38 Participants
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. Subjects with TEAEs <24 hrs. of A-1 MP infusion	17 Participants
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. Alpha-1 MP subjects with ≥1 drug-related TEAE	5 Participants
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. Number of Participants Analyzed	38 Participants
Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. Subjects experiencing TEAEs	26 Participants

Adverse Events

Alpha-1MP Treatment Group

Serious events: 2 serious events

Other events: 26 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Alpha-1MP Treatment Group n=38 participants at risk All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks
Infections and infestations Pneumonia	2.6% 1/38 • Number of events 1
Skin and subcutaneous tissue disorders Rash	2.6% 1/38 • Number of events 1

Other adverse events

Other adverse events
Measure	Alpha-1MP Treatment Group n=38 participants at risk All subjects received open-label weekly IV infusions of 60 mg/kg body weight of functional Alpha-1 MP for 20 weeks
Gastrointestinal disorders Nausea	15.8% 6/38 • Number of events 6
General disorders Chest pain	10.5% 4/38 • Number of events 4
General disorders Influenza like illness	5.3% 2/38 • Number of events 2
General disorders Chills	5.3% 2/38 • Number of events 2
Infections and infestations Nasopharyngitis	5.3% 2/38 • Number of events 2
Infections and infestations Oral candidiasis	5.3% 2/38 • Number of events 2
Infections and infestations Upper respiratory tract infection	15.8% 6/38 • Number of events 6
Infections and infestations Urinary tract infection	13.2% 5/38 • Number of events 5
Musculoskeletal and connective tissue disorders Arthralgia	5.3% 2/38 • Number of events 2
Musculoskeletal and connective tissue disorders Back pain	5.3% 2/38 • Number of events 2
Musculoskeletal and connective tissue disorders Muscle spasms	5.3% 2/38 • Number of events 2
Nervous system disorders Dizziness	5.3% 2/38 • Number of events 2
Nervous system disorders Headache	5.3% 2/38 • Number of events 3
Psychiatric disorders Anxiety	5.3% 2/38 • Number of events 2
Respiratory, thoracic and mediastinal disorders Cough	5.3% 2/38 • Number of events 2
Respiratory, thoracic and mediastinal disorders Dyspnoea	5.3% 2/38 • Number of events 2
Respiratory, thoracic and mediastinal disorders Pharyngolaryngeal pain	5.3% 2/38 • Number of events 2
Vascular disorders Hot flush	5.3% 2/38 • Number of events 2

Additional Information

Henry Li

Grifols Therapeutics

Phone: 1-800-520-2807

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The investigator must send a draft manuscript of the publication or abstract to the sponsor, thirty days in advance of submission, in order to obtain approval prior to submission of the final version for publication. This will be reviewed promptly and approval will not be withheld unreasonably. In case of a difference of opinion between the sponsor and the investigator(s), the contents of the publication will be discussed in order to find a solution that satisfies both parties.
Publication restrictions are in place

Restriction type: OTHER