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Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma

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ClinicalTrials.gov Identifier: NCT01293539
Recruitment Status : Terminated (IAC is now recognized as part of standard of care treatment options in the US.)
First Posted : February 10, 2011
Results First Posted : July 21, 2017
Last Update Posted : November 27, 2018
Sponsor:
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Retinoblastoma
Intervention Drug: Melphalan hydrochloride
Enrollment 10
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Intra-arterial Chemotherapy
Hide Arm/Group Description

IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site.

Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy.

Period Title: Overall Study
Started 10
Completed 9
Not Completed 1
Reason Not Completed
Physician Decision             1
Arm/Group Title Intra-arterial Chemotherapy
Hide Arm/Group Description

IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site.

Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy.

Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
<=18 years
10
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
7
  70.0%
Male
3
  30.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Hispanic or Latino
3
  30.0%
Not Hispanic or Latino
7
  70.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
2
  20.0%
White
7
  70.0%
More than one race
0
   0.0%
Unknown or Not Reported
1
  10.0%
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 10 participants
10
 100.0%
1.Primary Outcome
Title Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
Hide Description 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.
Time Frame Within the first six months after the initial treatment.
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Intra-arterial Chemotherapy
Hide Arm/Group Description:

IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site.

Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy.

Overall Number of Participants Analyzed 10
Measure Type: Count of Participants
Unit of Measure: Participants
10
 100.0%
Time Frame 4.5 years
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Intra-arterial Chemotherapy
Hide Arm/Group Description

IAC is offered to all patients with intraocular Rb who are deemed eligible after evaluation under anesthesia (EUA),with the exception of those few patients for whom focal modalities (i.e., laser or cryotherapy) would be effective.In cases of bilateral disease, both eyes would be treated sequentially during the same procedure utilizing the same femoral access site.

Two cycles of IAC will be performed at 3 to 4 week intervals. Melphalan dosing: 3-6 mth-old, 2.5mg; 3-12 mth-old, 3mg; 1-3 yr-old, 4mg; >3 yr-old, 5mg. For those tumors that have shown appropriate response and are amenable to local therapies, then no additional IAC will be performed. If the tumors do not regress after 2 cycles, then a third cycle of IAC will be offered. Local therapies (laser, cryotherapy) will not be given during the EUAs conducted prior to each treatment session, but will be allowed after toxicity and response has been assessed 3-4 weeks after the final cycle of intra-arterial therapy.

All-Cause Mortality
Intra-arterial Chemotherapy
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Intra-arterial Chemotherapy
Affected / at Risk (%) # Events
Total   3/10 (30.00%)    
Eye disorders   
Glaucoma * 1 [1]  2/10 (20.00%)  2
Retinal vascular disorder * 1 [2]  1/10 (10.00%)  1
Retinal vascular disorder * 1 [3]  1/10 (10.00%)  1
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
Eye-disorder, Grade 1
[2]
Eye-disorder, Grade 2
[3]
Eye-disorder, Grade 3
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Intra-arterial Chemotherapy
Affected / at Risk (%) # Events
Total   6/10 (60.00%)    
Eye disorders   
Eyelid edema & erythema * 1 [1]  1/10 (10.00%)  1
Eyelid function disorder * 1 [2]  1/10 (10.00%)  1
Eyelid function disorder * 1 [3]  1/10 (10.00%)  1
Eye pain * 1 [2]  1/10 (10.00%)  1
Mild swelling * 1 [4]  1/10 (10.00%)  1
Conjunctivitis * 1 [5]  1/10 (10.00%)  1
Gastrointestinal disorders   
Vomiting * 1 [6]  1/10 (10.00%)  1
General disorders   
Flu-like symptoms * 1 [7]  1/10 (10.00%)  1
Fever * 1 [8]  1/10 (10.00%)  1
Fever * 1 [9]  1/10 (10.00%)  1
Infections and infestations   
Ear infection * 1 [10]  1/10 (10.00%)  1
Injury, poisoning and procedural complications   
Injury - cut to lip during play * 1 [11]  1/10 (10.00%)  1
Respiratory, thoracic and mediastinal disorders   
Cough * 1 [12]  2/10 (20.00%)  2
Cough * 1 [13]  2/10 (20.00%)  2
*
Indicates events were collected by non-systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
Eyelid disorder, Grade 1
[2]
Eye disorder, Grade 1
[3]
Eye disorder, Grade 2
[4]
Eye disorders, other, Grade 1
[5]
Conjunctivitis, Grade 2
[6]
Vomiting, Grade 1
[7]
Flu-like symptoms, Grade 2
[8]
Fever, Grade 2
[9]
Fever, Grade 1
[10]
Otitis media, Grade 2
[11]
Injury, Grade 2
[12]
Cough, Grade 1
[13]
Cough, Grade 2
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Monica Pearl, M.D.
Organization: Johns Hopkins
Phone: 410-955-8525
Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT01293539     History of Changes
Other Study ID Numbers: J1071
NA_00040637 ( Other Identifier: JHMIRB )
First Submitted: January 21, 2011
First Posted: February 10, 2011
Results First Submitted: June 22, 2017
Results First Posted: July 21, 2017
Last Update Posted: November 27, 2018