Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

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Brief Title

Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

Official Title

An Open, Single-centre Non-randomized Phase II Clinical Trial on Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage

Brief Summary

      For selected cases with advanced Retinoblastoma (RTB) intraocular involvement(stage V of the
      Reese-Ellsworth classification) in which enucleation would usually be the standard
      therapeutic approach, in this project the investigators propose an alternative conservative
      treatment using intra-arterial chemotherapy with melphalan, via direct administration by
      catheterization of the ophthalmic artery.
    

Detailed Description

      Retinoblastoma (RTB) is the most frequent tumour of the eye in early childhood and the
      commonest cancer in the first year of life. Approximately 60% of cases are sporadic and
      unilateral. Unilateral tumours are usually diagnosed in the advanced intraocular stage and
      the most frequent treatment prescribed is enucleation. This prevents disease progression but
      has an important visual risk, and also constitutes a mutilation, with potentially devastating
      psychological effects on patients and their relatives.

      At diagnosis, patients affected with RTB and their relatives are faced with the important
      effects of this disease, such as a threat to life, although rare in developed countries, and
      the risk of losing their sight, which depends on the uni- or bilateral nature of the tumour,
      the topography of the tumour or tumours, and the still prevalent need for enucleation as a
      treatment. In fact, almost all advanced stage unilateral RTBs are treated with enucleation.
      In addition to the risk to life and the patient's sight associated with this treatment, it is
      also important to take into account the risk to the eye itself. For selected cases with
      advanced intraocular involvement (stage V of the Reese-Ellsworth classification) in which
      enucleation would usually be the standard therapeutic approach, in this project we propose an
      alternative conservative treatment using intra-arterial chemotherapy with melphalan, via
      direct administration by catheterization of the ophthalmic artery.

      The treatment aims to preserve the eye ball and visual acuity as much as possible in these
      patients, and has been demonstrated to be extremely effective at achieving volumetric
      reduction of tumours, which permits, if necessary, the subsequent conservative treatment,
      mainly with brachytherapy for anterior tumours or thermotherapy with laser diode for
      posterior tumours. In cases of retinal detachment, significant volume reduction, such as that
      achieved after injection with melphalan would, in most cases, permit retinal reapplication
      that would favour visual prognosis.

      This technique was first described by David H. Abramson in the Sloan-Kettering Cancer Center
      Memorial Hospital of New York (A Phase I/II Study of direct Intra-arterial (Ophthalmic
      Artery) Chemotherapy with Melphalan for Intraocular Retinoblastoma). Here, in this study we
      propose using this technique for the first time outside New York city, in our own clinical
      setting (Retinoblastoma Unit, affiliated to the Oncology Development Department and the
      Ophthalmology Department of the Sant Joan de Déu Hospital in Barcelona).

      The attainment of positive results, in addition to those previously obtained by the New York
      project, could consolidate this treatment as an alternative to enucleation in most cases of
      advanced intraocular RTB, and open the way for the future indication of this technique in
      other stages of RTB.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

To assess the saving of eyes affected with RTB for patients who would have been candidates for enucleation.

Secondary Outcome

 To preserve visual acuity, by studying the affected eye after the third cycle of treatment.

Condition

Retinoblastoma

Intervention

Melphalan

Study Arms / Comparison Groups

 Melphalan
Description:  Intra-arterial chemotherapy with melphalan, via direct administration by catheterization of the ophthalmic artery. Dosage range from 3 to 5 mg, depending of patient's weight and estimated tumour volume: a)3 mg for patients under 10 kg and tumour volume size under 1,5 cm3; b)5 mg for patients over 10 kg and tumour volume over 1,5 cm3; c)4 mg in all other situations(tumour volume over 1,5 cm3 in patients under 10 kg or tumour volume under 1,5 cm3 in patients over 10 kg).

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

5

Start Date

November 2009

Completion Date

May 2013

Primary Completion Date

December 2012

Eligibility Criteria

        Inclusion Criteria:

          1. Patients with unilateral RTB.

          2. Patients with advanced intraocular involvement, corresponding to Stage D of the
             International Classification , selected by the Tumour Committee of the Retinoblastoma
             Unit.

             By contrast to most other cancers, histological confirmation is contraindicated in RTB
             prior to onset of treatment and, in our study, any biopsy of the tumour practiced was
             considered as an exclusion criterion.

          3. The only alternative to treatment is enucleation.

          4. Over six months old at diagnosis and younger than six years old.

          5. Informed consent of the parents or legal representative.

        Exclusion Criteria:

          1. Under 6 months old at diagnosis.

          2. Impaired kidney function, with creatinine clearance lower than 80 mL/min/1.73m2 or
             serum creatinine higher than 0.7 mg/dL.

          3. Impaired liver function, normal function being defined as presenting total bilirubin
             levels lower than 1.5 times the limit of normal for that age and ALT lower than 5
             times the limit of normal for that age.

          4. Patients with some type of coagulation disorder that could contraindicate the
             procedure or with a previous diagnosis of any thrombotic condition.

          5. Congenital cerebral anomalies diagnosed previously or detected by angioresonance prior
             to treatment for extraocular involvement by RTB shown by image techniques,
             cerebrospinal fluid (CSF) cytology or cytomorphology of bone marrow aspirates (BMA),
             or positive expression of GD2 synthase in CSF or BMA.

          6. Patients with heart disease, arterial hypertension, or diseases of the nervous system
             not referred to in point 5, or with active infections that the Anaesthesiology Service
             responsible for the procedure have studied and consider to contraindicate the
             procedure.

          7. Not having been selected for intra-arterial chemotherapy through the ophthalmic artery
             for any other reason than those given by the Tumour Committee of the RTB Unit of the
             HSJD.

          8. Concurrent administration of any other anti-cancer treatment.

          9. Any surgical or non-surgical procedure that could have changed the structure of the
             eye and, therefore, facilitate risk of dissemination, including histological
             confirmation prior to treatment.

         10. Participation in another clinical trial.
      

Gender

All

Ages

6 Months - 6 Years

Accepts Healthy Volunteers

No

Contacts

Andreu Parareda, MD, , 

Location Countries

Spain

Location Countries

Spain

Administrative Informations


NCT ID

NCT01393769

Organization ID

HSJD-RTB-QTIA

Secondary IDs

2009-010737-47

Responsible Party

Sponsor

Study Sponsor

Hospital Sant Joan de Deu

Collaborators

 Fundació Sant Joan de Déu

Study Sponsor

Andreu Parareda, MD, Principal Investigator, Hospital Sant Joan de Deu


Verification Date

August 2016