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Item Modified 3+3 Design for MTD Re-estimation(2024-06) Zhang, Tianshu; Zang, Yong; Han, Yan; Liu, ZiyueThe 3+3 clinical trial design is one of the most popular dose-finding designs used in phase I oncology trials to identify the maximum tolerated dose (MTD) for new treatment regimens. While this design is widely used due to its simplicity , it has some notable limitations, including a maximum of six patients per dose level and fixed target toxicity rates. To address these issues, we propose a modified 3+3 design that extends the traditional 3+3 design by treating the remaining patients at the MTD level for additional dose-limiting toxicity (DLT) assessment. This modification allows for a more flexible and accurate way to identify the MTD, enhanced by the use of isotonic regression to calculate DLT rates. To compare the modified 3+3 designs and the traditional 3+3 design, computer simulation studies have been carried out under various dose-toxicity scenarios. The results show that the modified 3+3 design yields higher accuracy in MTD identification.Item Participant experiences in phase I pediatric oncology clinical trials(2017-08-31) Crane, Stacey M.; Haase, Joan E.; Hickman, Susan; Carpenter, Janet S.; Schwartz, PeterPhase I clinical trials (P1Ts) are the first step in testing new medical therapies in humans, and are essential for developing new and innovative therapies for children with cancer. P1Ts are ethically controversial as they are not intended to directly benefit participants, but are particularly controversial for children with cancer who are only able to participate when there is no known curative therapy for their cancer. Benefits of pediatric oncology P1T participation may include improved quality of life (QOL) and hope. Risks may include fostering unrealistic hope, burdening children with additional medical procedures and toxicities, and limiting the opportunity for palliation. The goal of this dissertation was to investigate the P1T participation experience for children with cancer and their parents by: (1) assessing what is currently known about the participation experience, (2) exploring ways to understand and assess treatment burden and QOL during participation, and (3) interviewing parents about the experience of having a child participate in a P1T. Following a review of the literature, two studies were conducted: a longitudinal pilot study of 13 parent and child dyads who enrolled in a pediatric oncology early phase clinical trial at the recruiting institution, and a phenomenological study of 11 parents of children with cancer who participated in pediatric oncology P1Ts. Key findings included a dearth of research on the experiences of children and parents in pediatric oncology P1Ts. Instead, existing research has focused on consent processes. The longitudinal pilot study provided some insight into experiences of children and parents during trial participation, including that there may be time points when parents’ and children’s perceptions of the child’s quality of life substantively differ. Interviews with parents confirmed some of the anticipated benefits and risks of participation in P1Ts, and highlighted parents’ sense of running out of time to find an effective treatment and needing to use time they have with their child well. Specific challenges in conducting this research were participant attrition due to disease progression and the need for multi-site research to obtain an adequate sample.Item Understanding Treatment Burden and Quality of Life Impact of Participating in an Early-Phase Pediatric Oncology Clinical Trial: A Pilot Study(Sage, 2018-01) Crane, Stacey; Backus, Lori; Stockman, Beth; Carpenter, Janet S.; Lin, Li; Haase, Joan E.; School of NursingPURPOSE: Early-phase clinical trials (EPTs) have led to new, more effective treatment options for children with cancer. Despite the extensive use of EPTs in pediatric oncology, little is known about parent and child experiences during EPT participation. The purposes of this pilot study were to assess the feasibility and preliminary results of having children with cancer and their parents complete measures of treatment burden and quality of life (QOL) concurrent with EPT participation. METHODS: In this descriptive, longitudinal, pilot study, parents and children were followed for the first 60 days of an EPT. Feasibility was assessed by participant enrollment and retention and completion of measures. Measures completed included the following: demographic form (completed at baseline); Diary of Trial Experiences to capture treatment burden (completed ongoing); and PedsQL™ Quality of Life Inventories, Cancer Modules, and Family Impact Module (completed at baseline, post-first disease evaluation, and off-study). Data were analyzed using descriptive statistics. RESULTS: Feasibility goals of enrollment, retention, and measure completion were partially met. Preliminary treatment burden and QOL results are provided. CONCLUSIONS: While QOL assessments may provide insight into EPT experiences, future studies need to be conducted at multiple sites and enrollment goals must account for participant attrition.