RetinoblastomaPhase 2NCT01293539
Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma
A Phase 2 study testing Melphalan hydrochloride in people with Retinoblastoma, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
Trial summary
The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye. Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Retinoblastoma
- What is being tested?
- Melphalan hydrochloride
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2011to Aug 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients newborn to 18 years old.
- Patients with intraocular retinoblastoma, unilateral or bilateral, who would be treated either by systemic chemotherapy, EBR, or enucleation would be considered for this study.
Who cannot join
- Patients over the age of 18.
- Patients with small, localized intraocular Rb amenable to focal therapy (laser or cryotherapy).
- Patients with extraocular disease evident on MRI (extension into the optic nerve), massive choroidal/uveal invasion (grade IIC or IID per ARET0332) or disease outside the globe evident on MRI or physical examination.
- Documented hypercoagulable disorders or vasculopathies.
- Laboratory exclusion criteria: GFR < 60 mL/min/1.73 m2
Trial locations and contacts
1 site in 1 country.
United States1 site
The Johns Hopkins Hospital
Baltimore, Maryland, 21287
About this study
The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye.
Read the detailed description
Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy. Administration of the drug directly to the targeted site thus avoids the complications and adverse events associated with toxicity from systemic, rather than local, chemotherapy.
- Conditions
- Retinoblastoma
- Being tested
- Melphalan hydrochloride
- Keywords
- Retinoblastoma, intra-arterial chemotherapy, angiography
- Medical subject headings
- Retinoblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Melphalan hydrochlorideDrug
Also called Alkeran
Drug administered intra-arterially (injection in the artery).
Standard dose:
2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg (>3 years old)
Dose modification: decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response.
Frequency: 2 treatment cycles at 3-4 week intervals, with a third treatment cycle administered if the tumor requires it.
Dose not to exceed 0.5mg/kg, per treatment cycle.
| Study group | Role | What they receive |
|---|---|---|
| Intraocular Retinoblastoma Patients | Other | Single group assignment of patients with intraocular retinoblastoma, unilateral or bilateral. Receives: Drug: Melphalan hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
The primary objective of this study is to show that intra-arterial delivery of the chemotherapeutic agent is successful in treating intraocular retinoblastoma, defined as avoiding systemic chemotherapy, external beam radiation, and enucleation.
Measured: Within the first six months after the initial treatment.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2011 (estimated)
- Study startMar 2011
- Primary completionAug 2016
- Study completionAug 2016
Why it stopped: IAC is now recognized as part of standard of care treatment options in the US.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for RetinoblastomaStudies of the same condition, open and under way.
- More trials run by Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsThe sponsor's other registered studies.
- 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
- 10 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Monica Pearl, M.D., Principal investigator, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01293539
- Sponsor's protocol ID
- J1071
- Other IDs
- NA_00040637
- Status last verified
- Nov 2018
- First submitted
- 21 Jan 2011
- Last updated
- 27 Nov 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
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 18342944Abramson DH. Chemosurgery for retinoblastoma: what we know after 5 years. Arch Ophthalmol. 2011 Nov;129(11):1492-4. doi: 10.1001/archophthalmol.2011.354. No abstract available.
PubMed 22084221
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma
- ClinicalTrials.gov ID
- NCT01293539
- Acronym / sponsor ID
- J1071
- Conditions
- Retinoblastoma
- Keywords
- Retinoblastoma, intra-arterial chemotherapy, angiography
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: IAC is now recognized as part of standard of care treatment options in the US.
- Enrolment
- 10 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Aug 2016 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 10 Feb 2011 (estimated)
- Results
- Posted 21 Jul 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to show that chemotherapy delivered directly through the artery supplying the eye (ophthalmic artery) to patients with retinoblastoma is a safe and effective treatment alternative to conventional systemic chemotherapy, external beam radiation, and surgical removal of the eye.
Detailed description
Delivering the chemotherapeutic agent in the arterial system through the ophthalmic artery transforms the treatment of retinoblastoma from systemic chemotherapy to local chemotherapy. Administration of the drug directly to the targeted site thus avoids the complications and adverse events associated with toxicity from systemic, rather than local, chemotherapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Intraocular Retinoblastoma Patients | Other | Drug: Melphalan hydrochlorideSingle group assignment of patients with intraocular retinoblastoma, unilateral or bilateral. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Patients newborn to 18 years old.
- Patients with intraocular retinoblastoma, unilateral or bilateral, who would be treated either by systemic chemotherapy, EBR, or enucleation would be considered for this study.
Key exclusion criteria (5 listed)
- Patients over the age of 18.
- Patients with small, localized intraocular Rb amenable to focal therapy (laser or cryotherapy).
- Patients with extraocular disease evident on MRI (extension into the optic nerve), massive choroidal/uveal invasion (grade IIC or IID per ARET0332) or disease outside the globe evident on MRI or physical examination.
- Documented hypercoagulable disorders or vasculopathies.
- Laboratory exclusion criteria: GFR < 60 mL/min/1.73 m2
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 |
|---|---|---|
| Melphalan hydrochlorideAlso: AlkeranDrug administered intra-arterially (injection in the artery). Standard dose: 2.5mg (3-6 month old) 3.0 mg (6-12 month old) 4.0 mg (1-3 year old) 5.0 mg (>3 years old) Dose modification: decrease standard dose by 25% if there are signs of toxicity. Increase the dose by 25% if there is inadequate tumor response. Frequency: 2 treatment cycles at 3-4 week intervals, with a third treatment cycle administered if the tumor requires it. Dose not to exceed 0.5mg/kg, per treatment cycle. | Drug | Intraocular Retinoblastoma Patients |
Primary outcome
Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
Time frame: Within the first six months after the initial treatment.
The primary objective of this study is to show that intra-arterial delivery of the chemotherapeutic agent is successful in treating intraocular retinoblastoma, defined as avoiding systemic chemotherapy, external beam radiation, and enucleation.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Nov 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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 22084221Abramson DH. Chemosurgery for retinoblastoma: what we know after 5 years. Arch Ophthalmol. 2011 Nov;129(11):1492-4. doi: 10.1001/archophthalmol.2011.354. No abstract available.
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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.
Monica Pearl, M.D. principal investigator
Johns Hopkins University
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 hydrochloride in people with Retinoblastoma, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 1 site in United States.
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 2 inclusion and 5 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 27 Nov 2018. 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 United States. 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?
Yes. Results are posted in the registry (first posted 21 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation. (time frame: Within the first six months after the initial treatment.). 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 NCT01293539, which also lists 2 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 27 Nov 2018.