Ongoing Clinical Trials for Retinoblastoma

There are currently 3 ongoing clinical trials focused on retinoblastoma, a rare eye cancer that primarily affects young children. These studies are exploring different treatment approaches to preserve vision and control the disease, including chemotherapy delivered intravenously, directly into the eye’s blood supply, or in combination with local treatments such as laser therapy and cryotherapy.

Clinical trial locations

• France
  • Study on Adjuvant Treatment for Unilateral Retinoblastoma with Vincristine Sulfate and Drug Combination in Patients After Primary Eye Removal
  • Study on Retinoblastoma Treatment: Evaluating the Effectiveness of Topotecan, Vincristine Sulfate, and Etoposide in Patients Eligible for Conservative Therapy
  • Study on Retinoblastoma Treatment Using Etoposide, Carboplatin, and Melphalan for Patients with Specific Eye Conditions

Study on Adjuvant Treatment for Unilateral Retinoblastoma with Vincristine Sulfate and Drug Combination in Patients After Primary Eye Removal

This trial is examining additional treatments given after surgical removal of the affected eye in children with cancer in only one eye. The goal is to prevent the cancer from returning by addressing risk factors identified when examining the removed tissue.

Who can participate: Children between 2 months and 10 years old who have been diagnosed with non-familial extensive unilateral retinoblastoma treated by primary eye removal are eligible. Participants must not have received any prior chemotherapy or radiotherapy. They need adequate organ function, including proper functioning of the blood, liver, and kidneys, as well as normal hearing and heart test results. Written consent from parents or legal representatives is required, and the child must be covered by social security.

Who cannot participate: Children with medical conditions other than retinoblastoma, those who have not had their eye surgically removed, those who do not need additional treatment, or those with cancer in both eyes cannot join. Children with other serious health conditions that could interfere with the study or who are unable to attend follow-up visits are also excluded.

What the trial involves: This study focuses on adjuvant treatment, which means therapy given after surgery to reduce the risk of cancer returning. The investigational drugs being tested include vincristine sulfate, thiotepa, cyclophosphamide, etoposide, and carboplatin. These medications are given through injections or infusions directly into the bloodstream. Some participants may receive a placebo for comparison purposes. Researchers will monitor participants over time to check for cancer recurrence, evaluate side effects, and assess long-term health impacts. The study will also examine the genetic makeup of tumors and use MRI imaging to detect any spread of cancer beyond the eye.

Study on Retinoblastoma Treatment Using Etoposide, Carboplatin, and Melphalan for Patients with Specific Eye Conditions

This trial focuses on preserving the eye without using external beam radiation. The study evaluates several treatment approaches using different combinations of medications and delivery methods.

Who can participate: The study includes children from birth to 6 years old who have not previously received chemotherapy or radiotherapy. Parents must provide written consent, and participants must have social security coverage. Specific eligibility varies by study part: Study 1 includes children with unilateral or bilateral retinoblastoma in groups A, B, or C, or children under six months with more advanced disease in one eye. Study 2 includes children aged 6 months to 6 years with bilateral disease where one eye can be treated with intra-arterial chemotherapy and the other with local treatments. Study 3 includes children with bilateral group D disease or with only one eye that can be treated conservatively.

Who cannot participate: Children with bilateral group D disease, bilateral macular threat (cancer threatening the central vision in both eyes), those not suitable for local treatment in one eye, or those from vulnerable populations are excluded.

What the trial involves: The study is divided into three parts with different treatment approaches. The first part uses intravenous chemotherapy with etoposide and carboplatin, followed by chemothermotherapy (chemotherapy combined with heat therapy). The second part involves melphalan delivered directly to the eye’s blood supply through intra-arterial chemotherapy. The third part includes a combination of three drugs along with local treatments like laser therapy and cryotherapy, which uses extreme cold to destroy cancer cells. The study aims to determine how well these treatments preserve the eye, prevent the need for radiation therapy, and monitor side effects and recurrence risk over time.

Study on Retinoblastoma Treatment: Evaluating the Effectiveness of Topotecan, Vincristine Sulfate, and Etoposide in Patients Eligible for Conservative Therapy

This trial evaluates different treatment strategies to preserve vision and control the disease. The study compares the effectiveness of intra-arterial chemotherapy with other conservative treatments.

Who can participate: Children with newly diagnosed retinoblastoma who have at least one eye that can be treated without removal are eligible. They must not have received prior chemotherapy or radiotherapy and should have no medical reasons preventing the proposed treatments. Parents or legal representatives must sign informed consent, and participants must be covered by French Social Security. Study 1 includes children aged 6 months to 6 years with disease manageable by intra-arterial chemotherapy. Study 2 includes children with disease manageable without intra-arterial chemotherapy, such as those under 6 months with unilateral disease in groups A, B, C, or D, or those with bilateral disease in groups A through E that can still be treated without eye removal.

Who cannot participate: No specific exclusion criteria are listed for this trial.

What the trial involves: The study is divided into two parts. Study 1 evaluates intra-arterial chemotherapy using topotecan and melphalan, delivered directly to the eye through the artery supplying blood to it. The goal is to assess local disease control and eye preservation 24 months after starting treatment. Study 2 evaluates other conservative treatments, potentially including intravenous chemotherapy combined with local eye treatments or local treatments alone. Visual function is assessed using World Health Organization criteria. Medications used include topotecan, vincristine, etoposide, carboplatin, and melphalan, administered through various routes depending on the treatment approach. Regular follow-up visits monitor treatment effectiveness and side effects, with visual assessments continuing until the child reaches 6 years of age with at least 24 months of follow-up.

Summary

All three ongoing clinical trials for retinoblastoma are being conducted in France, indicating a concentrated research effort in this country. The trials focus on different stages and types of the disease, from post-surgical adjuvant treatment to various conservative approaches aimed at preserving the eye and vision.

The studies employ a range of chemotherapy drugs including vincristine, carboplatin, etoposide, melphalan, thiotepa, cyclophosphamide, and topotecan. A notable aspect of these trials is the exploration of different delivery methods, including traditional intravenous administration, innovative intra-arterial chemotherapy delivered directly to the eye’s blood supply, and combination approaches with local treatments such as laser therapy and cryotherapy.

All trials are designed for young children, typically from birth to 10 years old, reflecting the age group most commonly affected by this rare eye cancer. The studies share common goals of preserving vision, avoiding the need for external beam radiation, preventing cancer recurrence, and minimizing treatment side effects while maintaining quality of life for these young patients.