A Prospective International Multicenter Clinical Trial for Eyes With Relapsed Retinoblastoma (NCT04455139) | Clinical Trial Compass
TerminatedPhase 2
A Prospective International Multicenter Clinical Trial for Eyes With Relapsed Retinoblastoma
Stopped: Difficulties in recruiting patients due to changing in treatment standards for the target population
Switzerland2 participantsStarted 2021-11-15
Plain-language summary
While 95% of patients with retinoblastoma can be cured nowadays, treatment of relapse remains challenging, ending often in enucleation and/or radiotherapy. In the last 10 years, new treatment modalities have been developed to give the chance of cure also in relapse, avoiding enucleation which results in esthetic sequelae and orbital growth problems, and radiotherapy which significantly increases the risk of secondary cancers in hereditary retinoblastoma. The current protocol aims at covering all types of relapses in retinoblastoma, with treatments adapted to the site of relapse, at harmonizing the new eye- and vision-preserving treatment procedures, and evaluating their efficacy and toxicity.
Who can participate
Age range
3 Months – 11 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Eye with recurrent Rb clinically defined as one or the combination of the following:
. Minimally required interval between study entry and time of the last treatment: 2 months (with a monthly follow-up), except for small retinal / subretinal tumors treated focally, not related to the current relapse
. Photographic documentation of fundus at study entry
. Registration into the study and start of treatment must occur no later than 14 days after diagnosis of recurrence
. Mandatory ultrasound biomicroscopy (UBM) at 35 or 50 MHz in case of opaque media or insufficient pupillary dilatation for evaluation of the posterior chamber / pars plana
. Age ≥3 months and \< 11 years (10.99)
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Rate of retinal toxicity of intravitreous administration of melphalan versus topotecan assessed by CTCAE v5.0
Timeframe: at 1 month after treatment completion
2
Relapse rate after IAC by melphalan only and IAC by melphalan + topotecan
. Weight ≥5 kg (in case of IAC eligibility or sequential IVitC/IAC eligibility)
. Possibility of follow-up until at least 2 years after end of current relapse treatment
Exclusion criteria
. Relapse with any uveal involvement and/or anterior chamber involvement
. Indication for another treatment option according to investigator's judgement
. Clinical/MRI signs of extraocular disease, including metastatic disease
. Inadequate organ function (in case of IAC or sequential IVitC / IAC eligibility):
. Other (simultaneous) malignancies
. Contraindication or known hypersensitivity to study drugs
. Severe concomitant diseases (e.g. immune deficiency syndrome)
. Current or recent (within 30 days prior to date of written informed consent) treatment with another investigational drug or participation in another interventional clinical trial