NCT01688882

Brief Summary

To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.

Trial Health

63
Monitor

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2013

Geographic Reach
6 countries

9 active sites

Status
terminated

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

First Submitted

Initial submission to the registry

September 17, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 20, 2012

Completed
3 months until next milestone

Study Start

First participant enrolled

January 1, 2013

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2015

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

April 28, 2016

Completed
Last Updated

April 28, 2016

Status Verified

March 1, 2016

Enrollment Period

2.2 years

First QC Date

September 17, 2012

Results QC Date

March 28, 2016

Last Update Submit

March 28, 2016

Conditions

Bullous Pemphigoid

Keywords

bullous pemphigoidQGE031

Outcome Measures

Primary Outcomes (1)

  • Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12

    Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 12 weeks based on the CGA-C in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating 'at least marked improvement from baseline' at 12 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement)

    12 weeks

Secondary Outcomes (2)

  • Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks

    6 weeks

  • Number of Patients Investigator Global Assessment Score Over 12 Weeks

    Baseline (week 0), week 6 and week 12

Study Arms (3)

QGE031

EXPERIMENTAL

QGE031 240 mg Q2W s.c.

Drug: QGE031

Placebo

PLACEBO COMPARATOR

Placebo to Match Q2W s.c.

Drug: Placebo

Open Label QGE031

EXPERIMENTAL

Open Label QGE031 Q2W s.c.

Drug: QGE031

Interventions

QGE031DRUG

QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen.

Open Label QGE031QGE031
PlaceboDRUG

Placebo will be used to control for normal variability in disease severity.

Placebo

Eligibility Criteria

Age20 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients diagnosed with bullous pemphigoid
  • Stable dose of prednisone at or above 10mg per day but no greater than 1 mg/kg/day
  • Weigh between 40-120kg
  • total IgE level up to 5000 IU/mL

You may not qualify if:

  • Use of rituximab within 1 year

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Novartis Investigative Site

Iowa City, Iowa, 52242, United States

Location

Novartis Investigative Site

Durham, North Carolina, 27710, United States

Location

Novartis Investigative Site

Vienna, A 1040, Austria

Location

Novartis Investigative Site

Rouen, Cedex, 76031, France

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Freiburg im Breisgau, 79104, Germany

Location

Novartis Investigative Site

Marburg, 35039, Germany

Location

Novartis Investigative Site

Shinjuku-ku, Tokyo, 160-8582, Japan

Location

Novartis Investigative Site

Taipei, Taiwan, 10002, Taiwan

Location

MeSH Terms

Conditions

Pemphigoid, Bullous

Interventions

ligelizumab

Condition Hierarchy (Ancestors)

Skin Diseases, VesiculobullousSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
862-778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

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 17, 2012

First Posted

September 20, 2012

Study Start

January 1, 2013

Primary Completion

March 1, 2015

Study Completion

March 1, 2015

Last Updated

April 28, 2016

Results First Posted

April 28, 2016

Record last verified: 2016-03

Locations

TW(1)DE(3)US(2)AU(1)FR(1)JP(1)