NCT01684410

Brief Summary

This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and older with cystic fibrosis (CF). The treatment duration in this study was intended to provide multi-dose safety information prior to proceeding to longer durations of exposure.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Aug 2012

Shorter than P25 for phase_2

Geographic Reach
1 country

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2012

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

September 6, 2012

Completed
7 days until next milestone

First Posted

Study publicly available on registry

September 13, 2012

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
2.4 years until next milestone

Results Posted

Study results publicly available

February 8, 2016

Completed
Last Updated

February 8, 2016

Status Verified

January 1, 2016

Enrollment Period

1.2 years

First QC Date

September 6, 2012

Results QC Date

August 10, 2015

Last Update Submit

January 11, 2016

Conditions

Cystic Fibrosis

Keywords

Cystic FibrosisAlpha1-proteinase inhibitorAlpha1-antitrypsin

Outcome Measures

Primary Outcomes (1)

  • Adverse Events

    adverse event frequency

    3 weeks

Other Outcomes (2)

  • Percent Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Week 3

    3 weeks

  • Percent Change From Baseline in Forced Vital Capacity (FVC) at Week 3

    3 weeks

Study Arms (3)

Alpha-1 HC 100 mg

EXPERIMENTAL

100 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.

Biological: Alpha-1 HC 100 mg

Alpha-1 HC 200 mg

EXPERIMENTAL

200 mg of aerosolized Alpha-1 HC inhaled daily via nebulizer for 3 weeks.

Biological: Alpha-1 HC 200 mg

Placebo

PLACEBO COMPARATOR

Placebo inhaled daily via nebulizer for 3 weeks. Placebo (phosphate buffer saline with polysorbate).

Biological: Placebo

Interventions

Alpha-1 HC 100 mgBIOLOGICAL

Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 100 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.

Also known as: Alpha1-proteinase inhibitor, alpha1-antitrypsin
Alpha-1 HC 100 mg
PlaceboBIOLOGICAL

Phosphate Buffer Saline with Polysorbate (placebo) composed of the same elements listed for Alpha-1 HC, minus the 50 mg/mL of Alpha-1 HC. Placebo inhaled once daily for 21 days for a total of 21 inhaled treatments.

Placebo
Alpha-1 HC 200 mgBIOLOGICAL

Alpha-1 HC is a sterile, liquid preparation of purified alpha1-proteinase inhibitor prepared from pooled human plasma. Alpha-1 HC 200 mg inhaled once daily for 21 days for a total of 21 inhaled treatments.

Also known as: Alpha1-proteinase inhibitor, alpha1-antitrypsin
Alpha-1 HC 200 mg

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age 18 years or older.
  • Documentation of CF diagnosis.
  • Have a pre-bronchodilator FEV1 ≥ 40% of predicted at Visit 1 and have a Visit 2 pre-investigational product FEV1 that is ≥ 40% of predicted and within ± 15% of the Visit 1 result.
  • Deemed by the Investigator to be a suitable candidate for serial collection of expectorated sputum.

You may not qualify if:

  • Had a pulmonary exacerbation during the 4 weeks before screening (Visit 1) which required the initiation of new antibiotic treatment
  • Have a pulmonary exacerbation during the screening period (between Visit 1 and Visit 2) which requires the initiation of new antibiotic treatment
  • FEV1 \< 0.59 liters at the screening visit
  • Respiratory insufficiency with continuous supplemental oxygen therapy, or carbon dioxide retention
  • Elevated aspartate transaminase (AST) or alanine aminotransferase (ALT) that is ≥ 3 times the upper limit of normal for age and gender
  • Smoking during the past 6 months
  • Lung surgery during the past 2 years
  • Positive culture for Burkholderia cepacia or mycobacterium during the past two years.
  • Active allergic bronchopulmonary aspergillosis
  • Pre-treatment sputum collection at Visit 1 or Visit 2 (Randomization) characterized by problems such as inadequate sputum volume or quality.
  • Known selective Immunoglobulin A (IgA) deficiency with known antibody against IgA (anti-IgA antibody).
  • History of anaphylaxis or severe systemic response to any plasma-derived alpha1-proteinase inhibitor preparation or other blood product(s), or to polysorbates.
  • Use of chronic oral steroids during the study. Note: Inhaled corticosteroids that had been administered for at least 4 weeks prior to Visit 1 were permissible during the study.
  • Use of chronic, high dose ibuprofen therapy within 3 weeks of screening and at anytime during the study.
  • Chronic maintenance therapy with systemic antibiotics within 3 weeks of screening and through last dose of investigational product.
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

The University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

National Jewish Hospital

Denver, Colorado, 80206, United States

Location

Children's Hospital Boston

Boston, Massachusetts, 02115, United States

Location

UNC at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Rainbow Babies and Children's Hospital

Cleveland, Ohio, 44106, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

MeSH Terms

Conditions

Cystic Fibrosisalpha 1-Antitrypsin Deficiency

Interventions

alpha 1-Antitrypsin

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesLiver DiseasesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesSerpinsPeptidesAmino Acids, Peptides, and ProteinsAcute-Phase ProteinsBlood ProteinsProteinsAlpha-GlobulinsSerum GlobulinsGlobulins

Results Point of Contact

Title
Henry Li, PhD
Organization
Grifols Therapeutics Inc.
henry.li@grifols.com
919-316-6042

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 6, 2012

First Posted

September 13, 2012

Study Start

August 1, 2012

Primary Completion

October 1, 2013

Study Completion

October 1, 2013

Last Updated

February 8, 2016

Results First Posted

February 8, 2016

Record last verified: 2016-01

Locations

US(6)