Methylphenidate in Pediatric Brain Tumor Survivors With Cancer-related Fatigue

NCT06905587 | Recruiting Phase 3

• 4 other identifiers: 24/51848R352-A207812023-0010942023-507926-18-00
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 4

Brief Summary

Cancer-related fatigue is a common and debilitating late effect in pediatric brain tumor survivors. Currently, evidence-based recommendations to ameliorate this condition are lacking. The researchers will investigate the ability of methylphenidate to improve fatigue and cognition in pediatric brain tumor survivors suffering from cancer-related fatigue. Methylphenidate is a drug (central nervous stimulant) most commonly used in the treatment of hyperkinetic disorders such as attention-deficit/hyperactivity disorder (ADHD). If methylphenidate shows an effect, the prospects are important for this patient group, since methylphenidate may then be included as part of the treatment of brain tumor-related fatigue.

Conditions

Cancer-related Fatigue; Brain Tumor, Pediatric; Methylphenidate

Keywords

methylphenidate; cancer-related fatigue; brain tumor; childhood cancer; pediatric cancer; childhood cancer survivor; late effect; long-term effects secondary to cancer therapy in children; long-term effects of cancer-treatment; pediatric brain tumor

Outcome Measures

Primary Outcomes (1)

• PedsQL Multidimensional Fatigue Scale (MFS)

  Changes in patient self-reported fatigue (participants above 18 years of age) or parent proxy-reported fatigue (participants between 6-17 years of age) from baseline to week 6 of MPH or placebo treatment as measured by the PedsQL Multidimensional Fatigue Scale (MFS).

  At baseline, at Week 6, Week 16, and Week 20 (follow-up).

Secondary Outcomes (8)

• PedsQL Multidimensional Fatigue Scale (MFS)

  At baseline, at Week 6, Week 16, and Week 20 (follow-up).

• Behaviour Rating Inventory of Executive Function (BRIEF)

  At baseline, at Week 6, and Week 16.

• Connor's Continuous Performance Test (CPT) Online Suite

  At baseline, at Week 6, and Week 16.

• Wechsler Coding

  At baseline, at Week 6, and Week 16.

• Wechsler Digit Span

  At baseline, at Week 6, and Week 16.

Study Arms (2)

Methylphenidate, Then Placebo

EXPERIMENTAL

Patients will receive treatment with methylphenidate tablets for 6 weeks, and then cross over to treatment with methylphenidate-matched placebo tablets for an additional 6 weeks. A four week wash out period is incorporated between treatments.

Drug: Methylphenidate (MPH); Drug: Placebo

Placebo, Then Methylphenidate

EXPERIMENTAL

Patients will receive treatment with methylphenidate-matched placebo tablets for 6 weeks, and then cross over to treatment with methylphenidate tablets for an additional 6 weeks. A four week wash out period is incorporated between treatments.

Drug: Methylphenidate (MPH); Drug: Placebo

Interventions

Methylphenidate (MPH) DRUG

10 mg methylphenidate tablets with a scoreline. Tablets will be administered orally. For children aged 6-12, a daily dose of 5 mg x 2 will be administered during the first week with an increase in dose to 10 mg x 2 in the second week. For adolescents and adults above 12 years of age, the same starting dose will be used as for children, with weekly incremental increases up to a maximum of 15 mg x 2 daily in the third week. In case of potential toxicity events during study, dosage of methylphenidate can be modified according to protocol.

Methylphenidate, Then Placebo; Placebo, Then Methylphenidate

Placebo DRUG

10 mg methylphenidate-matched placebo tablets with a scoreline. Tablets will be administered orally. Dosage will follow the exact same principles as for the study drug (methylphenidate).

Methylphenidate, Then Placebo; Placebo, Then Methylphenidate

Eligibility Criteria

• Age: 6 Years - 27 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosed and treated for a brain tumor during childhood or adolescence (0-≤18 years).
• Treated for a PBT during the previous 10 years, starting from date of diagnosis.
• Aged ≥6 years 0 months at the start of the trial.
• Off therapy/active treatment for pediatric brain tumor (PBT) for 12 months at the start of the trial.
• No known signs of clinical or radiological tumor progression at last follow-up.
• Danish is the sole or primary language (enabling provision of validated assessment tools).
• Clinically significant fatigue based on the PedsQL MFS questionnaire at baseline, defined by a score ≥ 1 standard deviation below the normative mean.
• History of clinically relevant fatigue after treatment of PBT compared to estimated premorbid ability, as assessed from consultations in the childhood cancer outpatient clinics.

You may not qualify if:

• Any known contraindications to methylphenidate as outlined below:
• A) Hypersensitivity to the active substance or any excipients listed in the summary of product characteristics. B) Glaucoma. C) Pheochromocytoma. D) Hyperthyroidism. E) Mania. F) Psychosis. G) Anorexia nervosa. H) Current or previous severe depression. I) Suicidal behavior. J) Poorly controlled type 1 bipolar affective disorder. K) Antisocial or borderline personality disorder. L) Pre-existing cardiovascular disorders, including severe hypertension, heart failure, arterial occlusive disease, angina pectoris, hemodynamically significant congenital heart disease, cardiomyopathies, myocardial infarction, potentially life-threatening cardiac arrhythmias and channelopathies. M) Pre-existing cerebrovascular disease, cerebral aneurysm, vascular abnormalities including vasculitis or stroke. N) Treatment with irreversible MAO inhibitors within the last 14 days and reversible MAO inhibitors within the last 24 hours.
• History of recent poorly controlled seizures.
• Motor tics or Tourette syndrome (including family history of tic disorder).
• Known diagnosis of Attention Deficit/Hyperactivity Disorder or Autism Spectrum Disorder.
• Known diagnosis of Full Scale Intelligence Quotient (FSIQ) of \<50.
• Pregnancy. Participants known to be pregnant or breastfeeding at screening/registration will not be enrolled in the trial. All sexually active women of childbearing potential (WOCBP) must have a negative pregnancy test prior to the start of treatment. Acceptableeffective contraceptive must be used for the duration of the trial. No further testing is needed during trial, unless the participant suspects to have become pregnant.
• Concerns about family ability to safely store or administer MPH, or to report side effects appropriately/concerns about familial substance abuse.
• Concurrent use of opiods (ATC N02A) or benzodiazepines (ATC N05BA and N05CF).
• Simultaneously enrolled in another clinical trial investigating cancer-related fatigue with a pharmaceutical intervention.

Sponsors & Collaborators

• Odense University Hospital: lead
• Aarhus University Hospital Skejby: collaborator
• Aalborg University Hospital: collaborator
• Rigshospitalet, Denmark: collaborator
• University of Southern Denmark: collaborator

Study Sites (4)

Aalborg University Hospital

Aalborg, 9000, Denmark

NOT YET RECRUITING

Aarhus University Hospital

Aarhus, 8200, Denmark

RECRUITING

Rigshospitalet

Copenhagen, 2100, Denmark

NOT YET RECRUITING

Odense University Hospital

Odense, 5000, Denmark

RECRUITING

MeSH Terms

Conditions

Brain Neoplasms; Neoplasms

Interventions

Methylphenidate

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Phenylacetates; Acids, Carbocyclic; Carboxylic Acids; Organic Chemicals; Piperidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Mathias Rathe, Consultant, MD, PhD

  Odense University Hospital

  STUDY DIRECTOR

Central Study Contacts

Sebastian W Most-Mottelson, MD, PhD Student

CONTACT

+45 21567257; sebastian.wenzel.most-mottelson@rsyd.dk

Mathias Rathe, Consultant, MD, PhD

CONTACT

+45 20469682; mathias.rathe@rsyd.dk

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 27, 2024
• First Posted: April 1, 2025
• Study Start: September 2, 2025
• Primary Completion (Estimated): December 1, 2029
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

DK (4)