Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment

This study has been terminated.

(This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo))

Sponsor:

Information provided by (Responsible Party):

Novartis ( Novartis Pharmaceuticals )

ClinicalTrials.gov Identifier:

NCT01688882

First received: September 17, 2012

Last updated: March 28, 2016

Last verified: March 2016

History of Changes

Results First Received: March 28, 2016

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Bullous Pemphigoid
Interventions:	Drug: QGE031 Drug: Placebo

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
QGE031	QGE031 240 mg Q2W s.c.
Placebo	Placebo to Match Q2W s.c.

Participant Flow: Overall Study

	QGE031	Placebo
STARTED	13	7 ^[1]
Safety Follow-up	11	5
COMPLETED	10	6 ^[2]
NOT COMPLETED	3	1
Adverse Event	1	0
Unsatisfactory therapeutic effect	1	1
Administrative problems	1	0

[1]	4 patients who completed 12 weeks of placebo changed to open-label QGE031 treatment.
[2]	The one patient that discontinued was already switched to open-label QGE031 treatment.

Baseline Characteristics

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Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
QGE031	QGE031 240 mg Q2W s.c.
Placebo	Placebo to Match Q2W s.c.
Total	Total of all reporting groups

Baseline Measures

	QGE031	Placebo	Total
Number of Participants [units: participants]	13	7	20
Age [units: years] Mean (Standard Deviation)	63.7 (14.85)	67.9 (14.60)	65.2 (14.52)
Gender [units: participants]
Female	3	4	7
Male	10	3	13

Outcome Measures

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1. Primary:

Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 [ Time Frame: 12 weeks ]

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2. Secondary:

Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks [ Time Frame: 6 weeks ]

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3. Secondary:

Number of Patients Investigator Global Assessment Score Over 12 Weeks [ Time Frame: Baseline (week 0), week 6 and week 12 ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.

Results Point of Contact:

Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300

Responsible Party:	Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:	NCT01688882 History of Changes
Other Study ID Numbers:	CQGE031X2202
Study First Received:	September 17, 2012
Results First Received:	March 28, 2016
Last Updated:	March 28, 2016
Health Authority:	United States: Food and Drug Administration Austria: Agency for Health and Food Safety Germany: Paul-Ehrlich-Institut Taiwan: Center for Drug Evaluation France: Ministry of Health

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