Safety, efficacy and PK/PD of QGE031 vs. placebo in patients with active bullous phemphigoid despite oral steroid treatment.

• Register: EUCTR
• Last refreshed on: 10 July 2015
• Main ID: EUCTR2012-003370-10-AT
• Date of registration: 03/10/2012
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma AG
• Public title: Safety, efficacy and PK/PD of QGE031 vs. placebo in patients with active bullous phemphigoid despite oral steroid treatment.
• Scientific title: A randomized, double-blind, placebo controlled, parallel group study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of QGE031 in the treatment of patients with bullous pemphigoid with disease refractory to oral steroid treatment
• Date of first enrolment: 13/11/2012
• Target sample size: 42
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-003370-10
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 4

Countries of recruitment

Austria, Germany, Taiwan, United States

Contacts

• Name: Project Manager at CRO
• Affiliation: Assign Clinical Research GmbH

• Name: Project Manager at CRO
• Affiliation: Assign Clinical Research GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
- Patients diagnosed with bullous phemphigoid
- Stable dose of prednisone at or above 10mg per day but no greather than 1mg/kg/day
- Weight between 40-120kg
- Total IgE level up to 5000 IU/mL

listing incomplete, additional inclusion criteria applicable
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 32

Exclusion criteria:
- Use of rifuximab within 1 year

listing incomplete, additional exclusion criteria applicable


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Refractory Bullous Pemphigoid
MedDRA version: 15.0 Level: LLT Classification code 10006567 Term: Bullous pemphigoid System Organ Class: 10040785 - Skin and subcutaneous tissue disorders

Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Intervention(s)

Product Code: QGE031
Pharmaceutical Form: Concentrate for solution for injection/infusion
Current Sponsor code: QGE031
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 120-
Pharmaceutical form of the placebo: Concentrate for solution for injection/infusion
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: 12 weeks

Secondary Objective: 1.To evaluate the CGA-C responder rate in QGE031 240mg q2w vs. Placebo treated patients at 6 weeks
2.To evaluate the effect of QGE031 240mg q2w in BP patients as assessed by Investigator Global Assessment (IGA)
3.To evaluate the safety of QGE031 in BP patients

Primary end point(s): Change in the Clinical Global Assessment of change from baseline to week 12.

Main Objective: To demonstrate the efficacy of QGE031 240mg q2w relative to placebo at 12 weeks in patients with BP by reducing disease activity as determined by Clinical Global Assessment of Change (CGA-C) responder rate.

Secondary Outcome(s)

Secondary end point(s): - Change in the Clinical Global Assessment of change from baseline to week 6.
- Change from baseline in the Investigator Global Assessment over 48 weeks.
- Safety of QGE031 over 48 weeks.

Timepoint(s) of evaluation of this end point: - 6 weeks
- baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks
- baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks

Secondary ID(s)

CQGE031X2202

Source(s) of Monetary Support

Novartis Pharma Services AG