Methylphenidate in Pediatric Brain Tumor Survivors With Cancer-related Fatigue

NCT ID: NCT06905587

Last Updated: 2025-12-11

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-09-02
• Study Completion Date: 2029-12-31

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

Brain Tumor, Pediatric; Cancer-related Fatigue; Methylphenidate

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Methylphenidate, Then Placebo

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.

Group Type: EXPERIMENTAL

Methylphenidate (MPH)

Intervention Type: 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.

Placebo

Intervention Type: 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).

Placebo, Then Methylphenidate

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.

Group Type: EXPERIMENTAL

Methylphenidate (MPH)

Intervention Type: 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.

Placebo

Intervention Type: 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).

Interventions

Methylphenidate (MPH)

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.

Intervention Type: DRUG

Placebo

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).

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Diagnosed and treated for a brain tumor during childhood or adolescence (0-≤18 years).

2. Treated for a PBT during the previous 10 years, starting from date of diagnosis.

3. Aged ≥6 years 0 months at the start of the trial.

4. Off therapy/active treatment for pediatric brain tumor (PBT) for 12 months at the start of the trial.

5. No known signs of clinical or radiological tumor progression at last follow-up.

6. Danish is the sole or primary language (enabling provision of validated assessment tools).

7. Patient and family have provided consent for inclusion in the trial.

8. Clinically significant fatigue based on the PedsQL MFS questionnaire at baseline, defined by a score ≥ 1 standard deviation below the normative mean.

9. History of clinically relevant fatigue after treatment of PBT compared to estimated premorbid ability, as assessed from consultations in the childhood cancer outpatient clinics.

Exclusion Criteria

1. 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.

2. History of recent poorly controlled seizures.

3. Motor tics or Tourette syndrome (including family history of tic disorder).

4. Known diagnosis of Attention Deficit/Hyperactivity Disorder or Autism Spectrum Disorder.

5. Known diagnosis of Full Scale Intelligence Quotient (FSIQ) of <50.

6. 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.

7. Concerns about family ability to safely store or administer MPH, or to report side effects appropriately/concerns about familial substance abuse.

8. Concurrent use of opiods (ATC N02A) or benzodiazepines (ATC N05BA and N05CF).

9. Simultaneously enrolled in another clinical trial investigating cancer-related fatigue with a pharmaceutical intervention.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 27 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Odense University Hospital

OTHER

Sponsor Role: lead

Aarhus University Hospital Skejby

OTHER

Sponsor Role: collaborator

Aalborg University Hospital

OTHER

Sponsor Role: collaborator

Rigshospitalet, Denmark

OTHER

Sponsor Role: collaborator

University of Southern Denmark

OTHER

Sponsor Role: collaborator

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mathias Rathe, Consultant, MD, PhD

Role: STUDY_DIRECTOR

Odense University Hospital

Locations

Aalborg University Hospital

Aalborg, , Denmark

Site Status: NOT_YET_RECRUITING

Aarhus University Hospital

Aarhus, , Denmark

Site Status: NOT_YET_RECRUITING

Rigshospitalet

Copenhagen, , Denmark

Site Status: NOT_YET_RECRUITING

Odense University Hospital

Odense, , Denmark

Site Status: RECRUITING

Countries

Denmark

Central Contacts

Sebastian W Most-Mottelson, MD, PhD Student

Role: CONTACT

sebastian.wenzel.most-mottelson@rsyd.dk

+45 21567257

Mathias Rathe, Consultant, MD, PhD

Role: CONTACT

mathias.rathe@rsyd.dk

+45 20469682

Facility Contacts

Eckhard Schomerus, Consultant, MD, Dr.med.

Role: primary

e.schomerus@rn.dk

+45 97654983

Louise T Henriksen, MD, PhD

Role: primary

louhen@rm.dk

+45 20921611

Christine Dahl, Consultant, MD, PhD

Role: primary

christine.dahl@regionh.dk

+45 35454137

Mathias Rathe, Consultant, MD, PhD

Role: primary

mathias.rathe@rsyd.dk

+45 20469682

Other Identifiers

R352-A20781

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2023-001094

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2023-507926-18-00

Identifier Type: CTIS

Identifier Source: secondary_id

24/51848

Identifier Type: -

Identifier Source: org_study_id