## International Workshops on Myositis Outcome Measures and Clinical Trial Design Issues

*Second Workshop -*

*Developing Consensus on Outcomes and*

*Clinical Trial Design Issues in Adult and Juvenile Myositis*

# *The International Myositis Assessment and Clinical Studies Group (IMACS)*

The Pan American Life Conference Center, New Orleans, Louisiana, USA

October 22-23, 2002

### AGENDA

> 08:00 AM Bus leaves the W Hotel for trip to the Pan American Life
> Conference Center

08:15 -- 8:30 AM Registration -- Hallway outside Bayou Rooms 1 and 2

######### 

######### **\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-- Introduction, Background and Goals of the Workshop \-\-\-\-\-\-\-\-\-\-\-\-\--**

#### 

#### **Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Rooms 1 and 2**

08:30 - 08:40 AM Welcome and Introduction -- *Ann Reed*

08:40 -- 09:00 AM Background and Goals of the Workshop - *Fred Miller*

> ***\-\-\-\-\-- Development of Preliminary Definitions of Improvement
> for Myositis \-\-\-\-\--***

09:00 - 09:10 AM Review of What was Learned at the Previous Workshop -
*Lisa Rider*

09:10 -- 10:10 AM Developing a Preliminary Definition of Improvement:
General Concepts and Prior and New Approaches - *Edward Giannini and
Peter Lachenbruch*

10:10 -- 10:25 AM Break

> 10:25 -- 10:45 AM Development of a Common Definition of Improvement
> for both Adult and Juvenile Myositis: Advantages and Disadvantages -
> *Fred Miller and Lisa Rider*
>
> 10:45 -- 11:00 AM Presentation of Top Candidate Definitions of
> Improvement for Adult and Juvenile Myositis -- *Lisa Rider and Edward
> Giannini*
>
> 11:00 -- 11:15 AM Summary and Review of Nominal Group Technique
> Methodologies - *Edward Giannini*

#### **Separate Meetings of the Adult (Bayou 3 Room) and Juvenile (Bayou 4 Room)**

##### [Myositis Working Groups]{.underline}

> 11:15 -- 12:15 PM Development of Consensus on the Preliminary
> Definitions of Improvement by Ranking top Definitions using Nominal
> Group Technique within each Working Group -- *Edward Giannini, Nicola
> Ruperto and Participants*
>
> 12:15 -- 1:00 PM Lunch -- pre-ordered in the Pontchartrain Room

#### **Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Rooms 1 and 2**

> 1:00 -- 1:30 PM Presentation of the Results of both Working Groups,
> Joint Discussion of the Preliminary Definitions of Improvement --
> *Hermine Brunner, Edward Giannini, Nicola Ruperto and Participants*
>
> 1:30 -- 2:30 PM Development of Consensus on the Preliminary
> Definitions of Improvement by Ranking top Definitions using Nominal
> Group Technique -- *Edward Giannini, Nicola Ruperto and*
> *Participants*
>
> 2:30 -- 2:45 PM: Final discussion of the Preliminary Definitions of
> Improvement for Adult and Juvenile Myositis. *Hermine Brunner, Edward
> Giannini, Nicola Ruperto, Fred Miller, Lisa Rider and Participants*
>
> 2:45 -- 3:00 PM: Logistic Regression Approaches for Preliminary
> Definitions of Improvement to be Explored in Clinical Trials *-- Peter
> Lachenbruch, Lisa Rider*

3:00 -- 3:30 PM Break

***\-\-\-\-\-\-\-\-\-\-\-\-\-- Developing Consensus on Clinical Trial
Design Issues - Part 1 \-\-\-\-\-\-\-\-\-\-\-\-\-\--***

######## Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Rooms 1 and 2

3:30 -- 4:00 PM Background and Review of Delphi Consensus Achieved in
the Myositis Clinical Trial Design Surveys for Adult and Juvenile
Myositis - *Chet Oddis and Lisa Rider*

4:00 - 4:15 PM Overview of the Initial Clinical Trial Design Issues to
Be Discussed - *Chet Oddis and Lisa Rider*

#### 

#### **Separate Meetings of the Adult (Bayou 3 Room) and Juvenile (Bayou 4 Room)**

#### **Myositis Working Groups**

4:15 -- 6:15 PM Development of Consensus on the Initial Clinical Trial
Design Issues by Nominal Group Technique within each Working Group --
*Edward Giannini, Nicola Ruperto and Participants*

a.  *Adult Group: Classification Criteria for PM and DM, Concomitant
    therapy*

b.  *Pediatric Group: Classification Criteria for JPM, Concomitant
    therapy and Concomitant Illnesses*

**END OF WORKSHOP DAY 1 - Tuesday, October 22^nd^**

###### 

> 6:30 PM Bus departs from the Pan American Life Conference Center to
> return participants for the prearranged dinner at the W Hotel
> Restaurant

7:30 PM Dinner at the BACCO Restaurant at the W Hotel

**[END OF DAY 1 - Tuesday, October 22^nd^]{.underline}**

##  International Workshops on Myositis Outcome Measures and Clinical Trial Design Issues

*Second Workshop -*

*Developing Consensus on Outcomes and*

*Clinical Trial Design Issues in Adult and Juvenile Myositis*

# *The International Myositis Assessment and Clinical Studies Group (IMACS)*

The Pan American Life Conference Center, New Orleans, Louisiana, USA

October 22-23, 2002

### AGENDA

####### 

> 08:00 AM Bus leaves the W Hotel for trip to the Pan American Life
> Conference Center

***\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-- Developing Consensus on Clinical
Trial Design Issues - Part 2 \-\-\-\-\-\-\-\-\-\--***

####### [Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Room 1 and 2]{.underline}

> 08:30 - 09:00 AM Presentation of the Results of both Working Groups,
> Joint Discussion of the Consensus on the Initial Clinical Trial Design
> Issues -- *Edward Giannini, Nicola Ruperto, Hermine Brunner and
> Participants*
>
> 09:00 -- 09:30 AM Overview of the Final Clinical Trial Design Issues
> to Be Discussed -- *Chet Oddis and Lisa Rider*

a.  *Criteria for Worsening/Flare Requiring Alteration of Therapy or
    Withdrawal from a Trial*

b.  *Preliminary Definition of Complete Clinical Response/Remission*

#### **Separate Meetings of the Adult (Bayou 3 Room) and Juvenile (Bayou 4 Room)**

##### [Myositis Working Groups]{.underline}

09:30 -- 11:00 AM Development of Consensus on Final Clinical Trial
Design Issues by Nominal Group Technique within each Working Group --
*Edward Giannini, Nicola Ruperto and Participants*

11:00 -- 11:30 AM Break

**[Joint Meeting of the Adult and Juvenile Myositis Working Groups --
Bayou Room 1 and 2]{.underline}**

11:30 -- 12:30 PM Presentation of the Results of both Working Groups,
Joint Discussion and Final Resolution of the Clinical Trial Design
Issues -- *Edward Giannini, Nicola Ruperto, Hermine Brunner, Chet Oddis,
Lisa Rider and Participants*

12:30 -- 1:15 PM Lunch -- pre-ordered in the Pontchartrain Room

[]{.mark}

[]{.mark}

***\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-- Developing Consensus
on Myositis Damage Issues \-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-\--***

####### [Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Room 1 and 2]{.underline}

1:15 -- 1:45 PM Introduction to Myositis Damage: Definition, Tools for
Assessment, and Review of Adult Myositis Data from the London Workshops
-- *David Isenberg*

1:45 -- 2:00 PM Review of Juvenile Myositis Damage Data from the London
Workshops -- *Clarissa Pilkington*

2:00 - 2:20 PM Review of Retrospective Adult and Juvenile Myositis
Damage Data: Natural History and Damage Accumulation over 6 -- 9 months
Using the Myositis Damage Index - *Lisa Rider*

2:20 -- 2:45 PM Overview of the Damage Issues to Be Discussed - *Ann
Reed and Lisa Rider*

a.  *Preexisting/Coexisting Conditions as Part of a Definition of
    Damage*

b.  *How Much Damage Accumulation to Allow in the course of a 6 -- 9
    month clinical trial*

2:45 - 3:15 PM Break

#### **Separate Meetings of the Adult (Bayou 3 Room) and Juvenile (Bayou 4 Room)**

##### [Myositis Working Groups]{.underline}

> 3:15 -- 4:15 AM Development of Consensus on Damage Issues by Nominal
> Group Technique within each Working Group -- *Edward Giannini, Nicola
> Ruperto and Participants*

####### [Joint Meeting of the Adult and Juvenile Myositis Working Groups -- Bayou Rooms 1 and 2]{.underline}

#### 

> 4:15 -- 4:45 PM Joint General Discussion of Myositis Damage Issues *-
> Edward Giannini, Nicola Ruperto, Hermine Brunner, Lisa Rider, Ann Reed
> and Participants*

***\-\-\-\-\-\-\-\-\-\-\-\-\-\-\-- Introduction to a Planned Multicenter
Clinical Trial of Rituximab \-\-\-\-\-\-\-\-\-\-\--***

> 4:45 - 5:15 PM Introduction to a Planned Multicenter Clinical Trial of
> Rituximab in PM-DM --
>
> *Background on Rituximab - Chet Oddis*
>
> *Preliminary Data - Todd Levine*
>
> *Trial Design - Brian Feldman*
>
> *Future Plans - Chet Oddis and Ann Reed*

***\-\-\-\-\-\-\-\-\-\-\-- Summary of Accomplishments, Problem Areas and
Future Plans \-\-\-\-\-\-\-\-\--***

> 5:15 -- 6:00 PM Combined Group Discussion of Results/Problem Areas
> from the Workshop and Future Plans -- *Ann Reed, Chet Oddis, Fred
> Miller, Lisa Rider and Participants*
>
> 6:00 PM Adjourn -- End of Second Workshop

**END OF WORKSHOP DAY 2 - Wednesday, October 23^rd^**

> 6:15 PM Bus departs from the Pan American Life Conference Center
> Adjourn to return to the W Hotel
>
> 7:00 PM Bus leaves the W Hotel for Dinner Jazz Cruise

8:00 -- 10:00 PM Dinner Jazz Cruise

**END OF WORKSHOP**