A Prospective International Multicenter Clinical Trial for Eyes With Relapsed Retinoblastoma

• Status: Not yet recruiting
• Trial end date: 2026-05-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Prof. Beck Popovic Maja
• Treatments: Melphalan; Topotecan

Criteria

Inclusion Criteria:

1. Eye with recurrent Rb clinically defined as one or the combination of the following:

• vitreous recurrence only

• retinal / diffuse subretinal relapse only not amenable to focal treatment such as thermotherapy, cryotherapy or plaque

• combined vitreous and retinal/diffuse subretinal relapse

2. 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

3. Photographic documentation of fundus at study entry

4. Registration into the study and start of treatment must occur no later than 14 days after diagnosis of recurrence

5. 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

6. Age ≥3 months and < 11 years (10.99)

7. Weight ≥5 kg (in case of IAC eligibility or sequential IVitC/IAC eligibility)

8. Possibility of follow-up until at least 2 years after end of current relapse treatment

9. Written informed consent by parents or legal representative before enrolment

Exclusion Criteria:

1. Relapse with any uveal involvement and/or anterior chamber involvement

2. Indication for another treatment option according to investigator's judgement

3. Clinical/MRI signs of extraocular disease, including metastatic disease

4. Inadequate organ function (in case of IAC or sequential IVitC / IAC eligibility):

• absolute neutrophils count <0.5 G/l

• thrombocytes count <100 G/l

• creatinine above normal value for age

• ALAT more than 2x above upper normal limit

• bilirubin above upper normal limit

5. Other (simultaneous) malignancies

6. Contraindication or known hypersensitivity to study drugs

7. Severe concomitant diseases (e.g. immune deficiency syndrome)

8. 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