Congenital aplastic anaemia – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:37:12 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Congenital aplastic anaemia – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on the Safety and Effectiveness of Afatinib for Fanconi Anemia Patients with Advanced Squamous Cell Carcinoma in the Oral Cavity, Oropharynx, Hypopharynx, or Larynx https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-afatinib-for-fanconi-anemia-patients-with-advanced-squamous-cell-carcinoma-in-the-oral-cavity-oropharynx-hypopharynx-or-larynx/ Wed, 29 Apr 2026 15:01:07 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-afatinib-for-fanconi-anemia-patients-with-advanced-squamous-cell-carcinoma-in-the-oral-cavity-oropharynx-hypopharynx-or-larynx/ This clinical trial is focused on studying the effects of a medication called Afatinib in patients with a rare genetic disorder known as Fanconi anemia. This disorder can lead to a type of cancer called squamous cell carcinoma, which affects areas such as the mouth, throat, and voice box. The study aims to understand how safe and effective Afatinib is when used to treat these cancers, especially when they cannot be surgically removed or have spread to other parts of the body.

The treatment involves taking Afatinib in the form of film-coated tablets, which are taken orally. The study will compare the effects of different doses of Afatinib to see how well it works in controlling the cancer and improving the quality of life for patients. Participants will be monitored for any side effects and how their cancer responds to the treatment over a period of time.

The purpose of this study is to gather information on the effectiveness of Afatinib in treating cancers associated with Fanconi anemia. The study will also look at how long the treatment effects last, the overall survival of patients, and any changes in their health-related quality of life. This information will help in understanding the potential benefits and risks of using Afatinib for this specific group of patients.

]]> Study on the Safety and Effects of Infusing Modified CD34+ Cells for Patients with Fanconi Anemia Subtype A https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-infusing-modified-cd34-cells-for-patients-with-fanconi-anemia-subtype-a/ Wed, 29 Apr 2026 15:00:03 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-infusing-modified-cd34-cells-for-patients-with-fanconi-anemia-subtype-a/ This clinical trial is focused on studying a rare genetic disorder called Fanconi Anemia Subtype A. This condition affects the body’s ability to repair damaged DNA, leading to bone marrow failure and an increased risk of cancer. The study is testing a treatment called Fancalen, which involves using the patient’s own blood cells. These cells are collected and modified outside the body using a special tool called a lentiviral vector to carry a healthy version of the FANCA gene. The modified cells are then infused back into the patient through a process called infusion.

The purpose of the study is to evaluate the long-term safety and effectiveness of this treatment. Participants will receive the infusion of their modified cells and will be monitored over time to see how well the treatment works and to check for any side effects. The study will look at how the modified cells behave in the body, including their ability to stay in the bone marrow and blood, and whether they help stabilize blood counts. Researchers will also assess if the treatment reduces the risk of developing blood-related cancers or other tumors.

Participants in this study will have regular follow-up visits to monitor their health and the effects of the treatment. These visits will include blood tests and other assessments to ensure the treatment is working as intended and to identify any potential issues early. The study aims to provide valuable information on the potential benefits and risks of using gene therapy to treat Fanconi Anemia Subtype A.

]]> Long-Term Study on the Safety and Effects of RP-L102 Infusion for Patients with Fanconi Anemia Subtype A https://clinicaltrials.eu/trial/long-term-study-on-the-safety-and-effects-of-rp-l102-infusion-for-patients-with-fanconi-anemia-subtype-a/ Wed, 29 Apr 2026 14:25:10 +0000 https://clinicaltrials.eu/trial/long-term-study-on-the-safety-and-effects-of-rp-l102-infusion-for-patients-with-fanconi-anemia-subtype-a/ This clinical trial is focused on studying Fanconi anemia subtype A, a rare genetic disorder that affects the bone marrow, leading to decreased production of blood cells. The treatment being tested is called Fancalen, also known by its code name RP-L102. This is a type of gene therapy that uses the patient’s own blood stem cells, which are modified outside the body to carry a healthy version of the FANCA gene. These modified cells are then infused back into the patient through a process called intravenous infusion.

The purpose of this study is to evaluate the long-term safety and effectiveness of this gene therapy. Participants will receive the infusion of Fancalen and will be monitored over an extended period to observe how well the treatment works and to ensure it is safe. The study will look at how the modified cells persist in the body and whether they help maintain stable blood counts, which is crucial for preventing complications associated with Fanconi anemia.

Throughout the study, researchers will also assess the potential for any long-term side effects, including the risk of developing other blood-related conditions. This trial is part of ongoing research to find better treatments for Fanconi anemia subtype A and aims to provide valuable insights into the benefits and risks of using gene therapy for this condition.

]]> Fanconi Hope https://clinicaltrials.eu/organisation/fanconi-hope/ Wed, 29 Apr 2026 13:34:18 +0000 https://clinicaltrials.eu/organisation/fanconi-hope/ Fanconi Hope is a registered national charitable trust, collaboratively established by parents of children affected by Fanconi Anaemia (FA) and clinicians with a specialized interest in the condition. Fanconi Anaemia is characterized as a rare genetic disorder leading to bone marrow failure in children, along with a heightened susceptibility to gynaecological, head, and neck cancers, and other medical complexities throughout life.

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