NCT01151735

Brief Summary

The study hypothesis is that treatment of Hereditary Angioedema at the time of prodromal symptoms will decrease morbidity associated with the disease

Trial Health

15
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Jul 2010

Status
withdrawn

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

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 25, 2010

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 28, 2010

Completed
3 days until next milestone

Study Start

First participant enrolled

July 1, 2010

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2012

Completed
Last Updated

November 30, 2012

Status Verified

November 1, 2012

Enrollment Period

2 years

First QC Date

June 25, 2010

Last Update Submit

November 29, 2012

Conditions

Keywords

AngioedemaHereditary DiseasesOrphan DiseaseHAE

Outcome Measures

Primary Outcomes (1)

  • percentage of full blown HAE attacks occurring within 24 hours following treatment at the prodromal

    To compare the percentage of full blown HAE attacks occurring within 24 hours following treatment at the prodromal stage of an attack using placebo or one of two doses of C1-INH.

    24 hours

Study Arms (3)

C-1-esterase inhibitor 1000 units

ACTIVE COMPARATOR

1000 units of C-1-esterase inhibitor given at time of prodromal symptoms

Drug: C-1-esterase

1500 units of C-1-esterase inhibitor

ACTIVE COMPARATOR

treatment with 1500 units of C-1-esterase inhibitor IV at the time of prodromal symptoms to decrease risk of exacerbation of HAE

Drug: C-1-esterase

placebo injection

PLACEBO COMPARATOR

placebo injection given for prodromal symptoms as double blinded therapy

Drug: placebo

Interventions

1000 units of C-1-esterase inhibitor

Also known as: low dose intervention
C-1-esterase inhibitor 1000 units

placebo

Also known as: placebo arm
placebo injection

Eligibility Criteria

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

You may qualify if:

  • Documented HAE type 1 or 2 by C4 level and C1-INH level or function.
  • Able to read, understand, and sign informed consent.
  • Above the age of 12 years.
  • Willing to complete daily diary.
  • Have at least 1 HAE exacerbations per month averaged over the last 6 months.
  • Present for treatment within 6 hours of onset of prodromal symptoms.
  • Treatment for an acute attack can be given at any time without restriction.

You may not qualify if:

  • Inability to read English.
  • Prior adverse effects to C1-INH.
  • Participation in alternate investigational drug trial.
  • Diabetes, neurologic diseases, cardiac diseases, dermatologic diseases that may have associated symptoms that mimic prodromal symptoms.
  • Inability to withdraw from androgens or C1-INH prophylaxis.
  • Pregnant or breast feeding mothers.
  • Prisoners or other institutionalized individuals.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Angioedemas, HereditaryAngioedemaGenetic Diseases, InbornRare Diseases

Interventions

Complement C1Methods

Condition Hierarchy (Ancestors)

Vascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency SyndromesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Complement System ProteinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsInvestigative Techniques

Study Officials

  • Michael Lunn, DO

    Penn State University

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

June 25, 2010

First Posted

June 28, 2010

Study Start

July 1, 2010

Primary Completion

July 1, 2012

Study Completion

July 1, 2012

Last Updated

November 30, 2012

Record last verified: 2012-11