Workshop: May 29-31, 2006
Scope & Objectives
Workshop Agenda
Participants
Scientific Rationale
Expected Outcomes
Scope & Objectives
Workshop Agenda
Participants
Scientific Rationale
Expected Outcomes
CLINICAL TRIALS IN RETT SYNDROME
Potential for Early Intervention (An International Workshop) Introduction:
29 participants from 9 countries attended this international workshop titled Clinical Trials in Rett Syndrome in May 2006. Presentations and round table discussions explored the feasibility of conducting Clinical Trials for Rett syndrome. This international workshop promoted the development of treatment strategies for RTT, by bringing together researchers working in dissimilar but related areas. Through an active exchange of scientific information, the participants: 1) identified areas in which current knowledge is limited and 2) provided recommendations for future research on clinical trials and related issues in RTT.
Origin/History of RettSearch:
The interest in establishing an international collaboration between RTT researchers and centers, with a focus on clinical trials, originated from discussions during the 2nd International Scientific Research Workshop, at the Swedish Rett Center, in Froson, Sweden, in June 2003. Following the meeting and through electronic communication, in the Fall of 2003 the first consortium of clinically-oriented RTT researchers (RETTCON) was established. In addition to the group present at Froson, a large number of distinguished clinical researchers in the RTT field joined RETTCON. The first goal of RETTCON was to organize an open forum-like conference for discussing design and implementation of clinical trials in RTT. On behalf of RETTCON, Walter Kaufmann (P.I.), Sarojini Budden (Co-P.I.), and Daniel Glaze (Co-P.I.) applied and obtained funding from the National Institutes of Health (NIH) in 2005. Critical supplementary funding and organizational support from the International Rett Syndrome Association (IRSA) allowed the meeting to take place at the historic and elegant Palace Hotel, in San Francisco, California (U.S.) immediately following the 22nd Annual IRSA Conference. "Clinical Trials in Rett Syndrome, Potential for Early Intervention (An International Workshop)", May 29-31, 2006, covered a wide spectrum of topics with emphasis on design of clinical trials, subject selection, therapeutic targets, and outcome measures. Proceedings of the San Francisco conference were published in the Journal of Child Neurology.
Expected Outcomes of RettSearch:
The overall conclusion of the San Francisco conference was that, although significant advances have been made in design and implementation of clinical trials in RTT, a variety of important issues still needed to be addressed before multi-center trials are feasible. The consensus among conference participants was that, in order to reach this goal, the consortium of researchers who participated in the conference had to remain as an active group working towards narrowing the gap of knowledge needed for the implementation of multi-center clinical trials. For this purpose, many members of the renamed network - RettSearch - will participate in committees or "working groups", focused on specific issues of relevance to clinical trials in RTT, which constitute the core structure of RettSearch. Through its website, RettSearch serves as a repository of information, working place, and instrument of communication for members of the network and other individuals interested in RTT. In this way, RettSearch joins other successful international collaborative efforts in the RTT field, such as the genotype/mutation (RettBase) and phenotype/clinical (InterRett) databases. The history of effective collaboration among RTT researchers and clinicians anticipates a bright future for RettSearch.
Sponsors:
The National Institute of Child Health and Human Development
The National Institute of Mental Health
Office of Rare Diseases
International Rett Syndrome Association
Kennedy Krieger Institute
Location:
The Palace Hotel
2 New Montgomery Street
San Francisco, CA 94105-3402
http://www.sfpalace.com/main/home.htm
Dates:
Monday, May 29 - Wednesday, May 31, 2006
Objective:
The objective of this meeting was to provide a forum for an open discussion on the design and implementation of clinical trials in Rett syndrome. The workshop emphasized genotypic and phenotypic features, early intervention, pharmacological and non-pharmacological strategies, and outcome measures.
Organizers:
Walter E. Kaufmann, M.D. (Kennedy Krieger Research Institute, Principal Investigator)
Sarojini S. Budden, M.D. (Oregon Health Sciences University, Co-Principal Investigator)
Daniel G. Glaze, M.D. (Baylor College of Medicine, Co-Principal Investigator)
Ms. Kathy Hunter (President, IRSA)
*Note: Thank you to IRSA families for providing pictures for this site.
Potential for Early Intervention (An International Workshop) Introduction:
29 participants from 9 countries attended this international workshop titled Clinical Trials in Rett Syndrome in May 2006. Presentations and round table discussions explored the feasibility of conducting Clinical Trials for Rett syndrome. This international workshop promoted the development of treatment strategies for RTT, by bringing together researchers working in dissimilar but related areas. Through an active exchange of scientific information, the participants: 1) identified areas in which current knowledge is limited and 2) provided recommendations for future research on clinical trials and related issues in RTT.
Origin/History of RettSearch:
The interest in establishing an international collaboration between RTT researchers and centers, with a focus on clinical trials, originated from discussions during the 2nd International Scientific Research Workshop, at the Swedish Rett Center, in Froson, Sweden, in June 2003. Following the meeting and through electronic communication, in the Fall of 2003 the first consortium of clinically-oriented RTT researchers (RETTCON) was established. In addition to the group present at Froson, a large number of distinguished clinical researchers in the RTT field joined RETTCON. The first goal of RETTCON was to organize an open forum-like conference for discussing design and implementation of clinical trials in RTT. On behalf of RETTCON, Walter Kaufmann (P.I.), Sarojini Budden (Co-P.I.), and Daniel Glaze (Co-P.I.) applied and obtained funding from the National Institutes of Health (NIH) in 2005. Critical supplementary funding and organizational support from the International Rett Syndrome Association (IRSA) allowed the meeting to take place at the historic and elegant Palace Hotel, in San Francisco, California (U.S.) immediately following the 22nd Annual IRSA Conference. "Clinical Trials in Rett Syndrome, Potential for Early Intervention (An International Workshop)", May 29-31, 2006, covered a wide spectrum of topics with emphasis on design of clinical trials, subject selection, therapeutic targets, and outcome measures. Proceedings of the San Francisco conference were published in the Journal of Child Neurology.
Expected Outcomes of RettSearch:
The overall conclusion of the San Francisco conference was that, although significant advances have been made in design and implementation of clinical trials in RTT, a variety of important issues still needed to be addressed before multi-center trials are feasible. The consensus among conference participants was that, in order to reach this goal, the consortium of researchers who participated in the conference had to remain as an active group working towards narrowing the gap of knowledge needed for the implementation of multi-center clinical trials. For this purpose, many members of the renamed network - RettSearch - will participate in committees or "working groups", focused on specific issues of relevance to clinical trials in RTT, which constitute the core structure of RettSearch. Through its website, RettSearch serves as a repository of information, working place, and instrument of communication for members of the network and other individuals interested in RTT. In this way, RettSearch joins other successful international collaborative efforts in the RTT field, such as the genotype/mutation (RettBase) and phenotype/clinical (InterRett) databases. The history of effective collaboration among RTT researchers and clinicians anticipates a bright future for RettSearch.
Sponsors:
The National Institute of Child Health and Human Development
The National Institute of Mental Health
Office of Rare Diseases
International Rett Syndrome Association
Kennedy Krieger Institute
Location:
The Palace Hotel
2 New Montgomery Street
San Francisco, CA 94105-3402
http://www.sfpalace.com/main/home.htm
Dates:
Monday, May 29 - Wednesday, May 31, 2006
Objective:
The objective of this meeting was to provide a forum for an open discussion on the design and implementation of clinical trials in Rett syndrome. The workshop emphasized genotypic and phenotypic features, early intervention, pharmacological and non-pharmacological strategies, and outcome measures.
Organizers:
Walter E. Kaufmann, M.D. (Kennedy Krieger Research Institute, Principal Investigator)
Sarojini S. Budden, M.D. (Oregon Health Sciences University, Co-Principal Investigator)
Daniel G. Glaze, M.D. (Baylor College of Medicine, Co-Principal Investigator)
Ms. Kathy Hunter (President, IRSA)
*Note: Thank you to IRSA families for providing pictures for this site.
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