Clinical trial NCT01164956
Conditions: Cancer
Keywords: methylphenidate fatigue
Sponsor: Dana-Farber Cancer Institute
Start date: 2011-07-01; Completion date: 2013-03-01
Enrollment: 3 participants; Allocation: RANDOMIZED; Intervention model: PARALLEL

Brief summary:
The overall aim of this pilot study is to conduct a combined N-of-1 trial (N-1-T) of MPH (methylphenidate) for amelioration of fatigue in children with cancer, and to evaluate the N-1-T design both for individual clinical decision making and for clinical trials in symptom management in pediatric oncology patients. Because no one knows which of the study options are best, participants will receive liquid MPH on some days and a placebo on other days. We will compare how the participant feels on MPH days with how they feel on placebo days to determine whether MPH makes a difference.

Detailed description:
I. To assess the N-1-T as a study design to evaluate a symptom-directed intervention in children with cancer

Primary objective

-To evaluate the feasibility of conducting an N-1-T to evaluate MPH for cancer-related fatigue in children as a group

Secondary objectives

* To evaluate the ability of the N-1-T to assess efficacy of MPH for an individual subject statistically and clinically definite answer (regarding the ability of MPH to reduce fatigue)
* To explore subject/family and oncologist perspectives on N1T participation
* To examine in a preliminary fashion whether there are patient, family, disease or study-related factors that are associated with attrition to help guide future large-scale N1Ts

II. To evaluate MPH for treatment of cancer-related fatigue and related symptoms in children

Primary objective

-To evaluate the effect of MPH on cancer-related fatigue in children based on various assessments including pedsFACIT-F and a unidimensional single-item Likert scale for measuring fatigue

Secondary objective

-To assess the side effect profile of MPH for fatigue in children with cancer

III. To evaluate fatigue assessment tools Primary objective

-To evaluate correlation between fatigue scores

Arms: arm 1: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 2: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 3: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 4: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 5: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 6: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 7: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted. arm 8: Participants were randomized to receive 3 treatment pairs of placebo (P) then methylphenidate (M) or M then P. Each M or P was given daily for 3 days and therefore treatment duration was 18 days over 3 treatment pairs. M or P was administered orally at a starting dose of 0.3 mg/kg/dose. For participants \> 40 kg, the maximum of dose in the first treatment pair was 12.5 mg per dose. Dose escalation to maximum 0.5 mg/kg/dose was permitted.
Interventions: intervention 1: methylphenidate intervention 2: Placebo
Intervention descriptions: intervention 1: None intervention 2: None

Protocol documents: