RetinoblastomaPhase 2NCT01393769
Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage
A Phase 2 study testing Melphalan in people with Retinoblastoma, run by Hospital Sant Joan de Deu at 1 site in Spain.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 6 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Melphalan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2009to May 2013
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 6 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients with unilateral RTB.
- 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.
- The only alternative to treatment is enucleation.
- Over six months old at diagnosis and younger than six years old.
- Informed consent of the parents or legal representative.
Who cannot join
- Under 6 months old at diagnosis.
- Impaired kidney function, with creatinine clearance lower than 80 mL/min/1.73m2 or serum creatinine higher than 0.7 mg/dL.
- 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.
- Patients with some type of coagulation disorder that could contraindicate the procedure or with a previous diagnosis of any thrombotic condition.
- 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.
- 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.
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- 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.
- Concurrent administration of any other anti-cancer treatment.
- 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.
- Participation in another clinical trial.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Retinoblastoma
- Being tested
- Melphalan
- Keywords
- Retinoblastoma, RTB, Ocular tumour, Melfalan, Enucleation, Tumor of the eye, Eye, Ocular cancer, Ocular chemotherapy, Ophthalmic artery, Intra-arterial chemotherapy
- Medical subject headings
- Retinoblastoma, Eye Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MelphalanDrug
Also called Melfalan, Melfalan GlaxoSmithKline Inyectable
The usual number of chemotherapy cycles will be 3. First treatment with Melphalan will be initiated as soon as possible, once the patients are selected for the study. Once the first treatment has been completed, day 0, successive treatments will be considered, second and third (days 21 and 42). In those cases in which some patients could be considered for an additional treatments after the third cycle, these will be administrated in intervals at least of 21 days.Dosage range from 3 to 5 mg, depending of patient's weight and estimated tumour volume.
| Study group | Role | What they receive |
|---|---|---|
| Melphalan | Experimental | 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). Receives: Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the saving of eyes affected with RTB for patients who would have been candidates for enucleation.
The primary endpoint will be the objective response to treatment determined by funduscopy and RetCam explorations, recorded as a percentage of partial response (PR) or complete response (CR) to the treatment administered.
Measured: From V1 (Baseline) to V14 (1+ year after last treatment)
The response will be evaluated as a function of tumoral volumetric size.
The response will be evaluated as a function of tumoral volumetric size by comparing images obtained in successive funduscopy and RetCam explorations.
Measured: From V1 (Baseline) to V14 (+1 year after last treatment)
Secondary outcomes (3)
To preserve visual acuity, by studying the affected eye after the third cycle of treatment.
The secondary endpoint studied will correspond to changes in visual acuity of the affected eye after the third cycle of treatment, assessed by a visual acuity study.
Measured: V1 (Basal), V13 (day +52 to +60), V14 (+1 year after last treatment)
To modify the result of electroretinographic studies and visual evoked potentials after the third cycle of treatment.
The secondary endpoint studied will correspond to changes in electroretinographic studies and visual evoked potentials after the third cycle of treatment
Measured: V1 (Basal), V13 (day +52 to +60), V14 (+1 year after last treatment dosage)
To evaluate the safety of the technique and medicinal product used, by studying the ophthalmologic and systemic adverse events.
The secondary safety endpoints studied will correspond to analytical changes in the CBC (essentially in the number of absolute neutrophils) and biochemistry analysis after 10 days of each treatment, and potential ophthalmologic side effects.
Measured: Adverse events:on ongoing basis-assessed in each protocol visit / Laboratory test: V1 (Basal), V3&4 (day +1 to+10), V7&8 (day +22 to +31), V11&12 (day +43 to +52)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2011 (estimated)
- Study startNov 2009
- Primary completionDec 2012
- Study completionMay 2013
Why it stopped: Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (21)Papers the sponsor has linked to this study.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital Sant Joan de Deu
- Sponsor type
- University, hospital or other
- Collaborators
- Fundació Sant Joan de Déu, Hospital Universitari General de Catalunya, Ministry of Health, Spain
- Responsible party
- Sponsor
- Investigators
- Andreu Parareda, MD, Principal investigator, Hospital Sant Joan de Deu
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01393769
- Sponsor's protocol ID
- HSJD-RTB-QTIA
- Other IDs
- 2009-010737-47, 2009
- Status last verified
- Aug 2016
- First submitted
- 7 Jul 2011
- Last updated
- 2 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Publications
Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.
PubMed 18342944Aerts I, Pacquement H, Doz F, Mosseri V, Desjardins L, Sastre X, Michon J, Rodriguez J, Schlienger P, Zucker JM, Quintana E. Outcome of second malignancies after retinoblastoma: a retrospective analysis of 25 patients treated at the Institut Curie. Eur J Cancer. 2004 Jul;40(10):1522-9. doi: 10.1016/j.ejca.2004.03.023.
PubMed 15196536Apushkin MA, Apushkin MA, Shapiro MJ, Mafee MF. Retinoblastoma and simulating lesions: role of imaging. Neuroimaging Clin N Am. 2005 Feb;15(1):49-67. doi: 10.1016/j.nic.2005.02.003.
PubMed 15927860Brisse HJ, Lumbroso L, Freneaux PC, Validire P, Doz FP, Quintana EJ, Berges O, Desjardins LC, Neuenschwander SG. Sonographic, CT, and MR imaging findings in diffuse infiltrative retinoblastoma: report of two cases with histologic comparison. AJNR Am J Neuroradiol. 2001 Mar;22(3):499-504.
PubMed 11237973Chantada G, Doz F, Antoneli CB, Grundy R, Clare Stannard FF, Dunkel IJ, Grabowski E, Leal-Leal C, Rodriguez-Galindo C, Schvartzman E, Popovic MB, Kremens B, Meadows AT, Zucker JM. A proposal for an international retinoblastoma staging system. Pediatr Blood Cancer. 2006 Nov;47(6):801-5. doi: 10.1002/pbc.20606.
PubMed 16358310Chantada G, Fandino A, Manzitti J, Urrutia L, Schvartzman E. Late diagnosis of retinoblastoma in a developing country. Arch Dis Child. 1999 Feb;80(2):171-4. doi: 10.1136/adc.80.2.171.
PubMed 10325735Chantada GL, Dunkel IJ, de Davila MT, Abramson DH. Retinoblastoma patients with high risk ocular pathological features: who needs adjuvant therapy? Br J Ophthalmol. 2004 Aug;88(8):1069-73. doi: 10.1136/bjo.2003.037044.
PubMed 15258027Chantada GL, Fandino A, Mato G, Casak S. Phase II window of idarubicin in children with extraocular retinoblastoma. J Clin Oncol. 1999 Jun;17(6):1847-50. doi: 10.1200/JCO.1999.17.6.1847.
PubMed 10561224Chantada GL, Rossi J, Casco F, Fandino A, Scopinaro M, de Davila MT, Abramson DH. An aggressive bone marrow evaluation including immunocytology with GD2 for advanced retinoblastoma. J Pediatr Hematol Oncol. 2006 Jun;28(6):369-73. doi: 10.1097/00043426-200606000-00009.
PubMed 16794505Dunkel IJ, Aledo A, Kernan NA, Kushner B, Bayer L, Gollamudi SV, Finlay JL, Abramson DH. Successful treatment of metastatic retinoblastoma. Cancer. 2000 Nov 15;89(10):2117-21. doi: 10.1002/1097-0142(20001115)89:103.0.co;2-9.
PubMed 11066053Dunkel IJ, Gerald WL, Rosenfield NS, Strong EW, Abramson DH, Ghavimi F. Outcome of patients with a history of bilateral retinoblastoma treated for a second malignancy: the Memorial Sloan-Kettering experience. Med Pediatr Oncol. 1998 Jan;30(1):59-62. doi: 10.1002/(sici)1096-911x(199801)30:13.0.co;2-3.
PubMed 9371391Kaneko A, Suzuki S. Eye-preservation treatment of retinoblastoma with vitreous seeding. Jpn J Clin Oncol. 2003 Dec;33(12):601-7.
PubMed 14769836Kiribuchi M. [Retrograde infusion of anti-cancer drugs to ophthalmic artery for intraocular malignant tumors]. Nippon Ganka Gakkai Zasshi. 1966 Nov;70(11):1829-33. No abstract available. Japanese.
PubMed 6009660Moll AC, Imhof SM, Bouter LM, Kuik DJ, Den Otter W, Bezemer PD, Koten JW, Tan KE. Second primary tumors in patients with hereditary retinoblastoma: a register-based follow-up study, 1945-1994. Int J Cancer. 1996 Aug 7;67(4):515-9. doi: 10.1002/(SICI)1097-0215(19960807)67:43.0.CO;2-V.
PubMed 8759610Linn Murphree A. Intraocular retinoblastoma: the case for a new group classification. Ophthalmol Clin North Am. 2005 Mar;18(1):41-53, viii. doi: 10.1016/j.ohc.2004.11.003.
PubMed 15763190
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01393769
- Acronym / sponsor ID
- HSJD-RTB-QTIA
- Conditions
- Retinoblastoma
- Keywords
- Retinoblastoma, RTB, Ocular tumour, Melfalan, Enucleation, Tumor of the eye, Eye, Ocular cancer
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Hospital Sant Joan de DeuUniversity, hospital or other
- Collaborators
- Fundació Sant Joan de Déu, Hospital Universitari General de Catalunya, Ministry of Health, Spain
- Study start
- Nov 2009
- Primary completion
- Dec 2012 (actual)
- Study completion
- May 2013 (actual)
- First posted
- 13 Jul 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Melphalan | Experimental | Drug: MelphalanIntra-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). |
Who may qualify
- Ages
- 6 months to 6 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Patients with unilateral RTB.
- 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.
- The only alternative to treatment is enucleation.
- Over six months old at diagnosis and younger than six years old.
Key exclusion criteria (10 listed)
- Under 6 months old at diagnosis.
- Impaired kidney function, with creatinine clearance lower than 80 mL/min/1.73m2 or serum creatinine higher than 0.7 mg/dL.
- 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.
- Patients with some type of coagulation disorder that could contraindicate the procedure or with a previous diagnosis of any thrombotic condition.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MelphalanAlso: Melfalan, Melfalan GlaxoSmithKline InyectableThe usual number of chemotherapy cycles will be 3. First treatment with Melphalan will be initiated as soon as possible, once the patients are selected for the study. Once the first treatment has been completed, day 0, successive treatments will be considered, second and third (days 21 and 42). In those cases in which some patients could be considered for an additional treatments after the third cycle, these will be administrated in intervals at least of 21 days.Dosage range from 3 to 5 mg, depending of patient's weight and estimated tumour volume. | Drug | Melphalan |
Primary outcome
To assess the saving of eyes affected with RTB for patients who would have been candidates for enucleation.
Time frame: From V1 (Baseline) to V14 (1+ year after last treatment)
The primary endpoint will be the objective response to treatment determined by funduscopy and RetCam explorations, recorded as a percentage of partial response (PR) or complete response (CR) to the treatment administered.
The response will be evaluated as a function of tumoral volumetric size.
Time frame: From V1 (Baseline) to V14 (+1 year after last treatment)
The response will be evaluated as a function of tumoral volumetric size by comparing images obtained in successive funduscopy and RetCam explorations.
3 secondary outcomes
To preserve visual acuity, by studying the affected eye after the third cycle of treatment.
Time frame: V1 (Basal), V13 (day +52 to +60), V14 (+1 year after last treatment)
To modify the result of electroretinographic studies and visual evoked potentials after the third cycle of treatment.
Time frame: V1 (Basal), V13 (day +52 to +60), V14 (+1 year after last treatment dosage)
To evaluate the safety of the technique and medicinal product used, by studying the ophthalmologic and systemic adverse events.
Time frame: Adverse events:on ongoing basis-assessed in each protocol visit / Laboratory test: V1 (Basal), V3&4 (day +1 to+10), V7&8 (day +22 to +31), V11&12 (day +43 to +52)
Results and safety
The study was stopped early (reason given: Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2016.
- Background: PubMed 18342944Abramson DH, Dunkel IJ, Brodie SE, Kim JW, Gobin YP. A phase I/II study of direct intraarterial (ophthalmic artery) chemotherapy with melphalan for intraocular retinoblastoma initial results. Ophthalmology. 2008 Aug;115(8):1398-404, 1404.e1. doi: 10.1016/j.ophtha.2007.12.014. Epub 2008 Mar 14.
- Background: PubMed 15196536Aerts I, Pacquement H, Doz F, Mosseri V, Desjardins L, Sastre X, Michon J, Rodriguez J, Schlienger P, Zucker JM, Quintana E. Outcome of second malignancies after retinoblastoma: a retrospective analysis of 25 patients treated at the Institut Curie. Eur J Cancer. 2004 Jul;40(10):1522-9. doi: 10.1016/j.ejca.2004.03.023.
- Background: PubMed 15927860Apushkin MA, Apushkin MA, Shapiro MJ, Mafee MF. Retinoblastoma and simulating lesions: role of imaging. Neuroimaging Clin N Am. 2005 Feb;15(1):49-67. doi: 10.1016/j.nic.2005.02.003.
- Background: PubMed 11237973Brisse HJ, Lumbroso L, Freneaux PC, Validire P, Doz FP, Quintana EJ, Berges O, Desjardins LC, Neuenschwander SG. Sonographic, CT, and MR imaging findings in diffuse infiltrative retinoblastoma: report of two cases with histologic comparison. AJNR Am J Neuroradiol. 2001 Mar;22(3):499-504.
- Background: PubMed 16358310Chantada G, Doz F, Antoneli CB, Grundy R, Clare Stannard FF, Dunkel IJ, Grabowski E, Leal-Leal C, Rodriguez-Galindo C, Schvartzman E, Popovic MB, Kremens B, Meadows AT, Zucker JM. A proposal for an international retinoblastoma staging system. Pediatr Blood Cancer. 2006 Nov;47(6):801-5. doi: 10.1002/pbc.20606.
- Background: PubMed 10325735Chantada G, Fandino A, Manzitti J, Urrutia L, Schvartzman E. Late diagnosis of retinoblastoma in a developing country. Arch Dis Child. 1999 Feb;80(2):171-4. doi: 10.1136/adc.80.2.171.
- Background: PubMed 15258027Chantada GL, Dunkel IJ, de Davila MT, Abramson DH. Retinoblastoma patients with high risk ocular pathological features: who needs adjuvant therapy? Br J Ophthalmol. 2004 Aug;88(8):1069-73. doi: 10.1136/bjo.2003.037044.
- Background: PubMed 10561224Chantada GL, Fandino A, Mato G, Casak S. Phase II window of idarubicin in children with extraocular retinoblastoma. J Clin Oncol. 1999 Jun;17(6):1847-50. doi: 10.1200/JCO.1999.17.6.1847.
- Background: PubMed 16794505Chantada GL, Rossi J, Casco F, Fandino A, Scopinaro M, de Davila MT, Abramson DH. An aggressive bone marrow evaluation including immunocytology with GD2 for advanced retinoblastoma. J Pediatr Hematol Oncol. 2006 Jun;28(6):369-73. doi: 10.1097/00043426-200606000-00009.
- Background: PubMed 11066053Dunkel IJ, Aledo A, Kernan NA, Kushner B, Bayer L, Gollamudi SV, Finlay JL, Abramson DH. Successful treatment of metastatic retinoblastoma. Cancer. 2000 Nov 15;89(10):2117-21. doi: 10.1002/1097-0142(20001115)89:103.0.co;2-9.
- Background: PubMed 9371391Dunkel IJ, Gerald WL, Rosenfield NS, Strong EW, Abramson DH, Ghavimi F. Outcome of patients with a history of bilateral retinoblastoma treated for a second malignancy: the Memorial Sloan-Kettering experience. Med Pediatr Oncol. 1998 Jan;30(1):59-62. doi: 10.1002/(sici)1096-911x(199801)30:13.0.co;2-3.
- Background: PubMed 14769836Kaneko A, Suzuki S. Eye-preservation treatment of retinoblastoma with vitreous seeding. Jpn J Clin Oncol. 2003 Dec;33(12):601-7.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Andreu Parareda, MD principal investigator
Hospital Sant Joan de Deu
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Melphalan in people with Retinoblastoma, run by Hospital Sant Joan de Deu at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 2 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To assess the saving of eyes affected with RTB for patients who would have been candidates for enucleation. (time frame: From V1 (Baseline) to V14 (1+ year after last treatment)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01393769, which also lists 21 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Aug 2016.