Ongoing Clinical Trials Related to Stem Cell Transplant
There are currently 3 ongoing clinical trials focused on preventing and managing complications associated with stem cell transplantation. These studies are investigating treatments for blood vessel damage, gut bacteria restoration, and immune system modulation to improve outcomes for both children and adults undergoing stem cell transplants.
Clinical trial locations
- France
- Germany
- Netherlands
- Spain
Study of Narsoplimab for Children with High-Risk Blood Vessel Damage After Stem Cell Transplant
This trial is testing a medication called Narsoplimab in children who develop a serious complication after receiving a stem cell transplant. The condition, called Haematopoietic Stem Cell Transplant Thrombotic Microangiopathy (HSCT-TMA), involves damage to small blood vessels that can affect multiple organs.
Who can participate:
- Children aged from 28 days to less than 18 years old
- Those who have received a donor stem cell transplant for either cancer or non-cancer blood conditions
- Children diagnosed with high-risk HSCT-TMA, which includes having low platelet counts (less than 50,000 per microliter) and signs of blood cell damage
- Patients must meet at least one high-risk criterion, such as persistent HSCT-TMA lasting 2 weeks or more, kidney problems, digestive system involvement, nervous system complications, or fluid around the heart or lungs
- Parent or legal guardian consent is required, and the child must give assent if old enough
Who cannot participate:
- Patients who have had a stem cell transplant but do not have thrombotic microangiopathy
- Those not considered high-risk for complications
- Patients outside the specified age range
What the study involves: The trial focuses on evaluating whether Narsoplimab, given as an intravenous injection directly into a vein, can improve survival rates and overall health in children with high-risk HSCT-TMA. The medication works by inhibiting a protein called MASP-2, which plays a role in the immune system’s inflammatory response, thereby reducing inflammation and blood vessel damage. Researchers will monitor participants for 100 days after diagnosis and continue to assess long-term health outcomes, including how the body processes the medication and any potential side effects.
Study on Fecal Microbiota Transplantation to Prevent Complications in Patients After Stem Cell Transplant for Blood Cancer
This trial investigates whether Fecal Microbiota Transplantation (FMT) can help prevent complications in adults who have undergone donor stem cell transplants for blood cancers. FMT involves transferring healthy bacteria from a donor’s stool to the patient’s gut to restore a healthy balance of intestinal bacteria.
Who can participate:
- Adults aged 18 years or older
- Those who have received a donor stem cell transplant from peripheral blood (not cord blood) for a controlled blood-related cancer
- Patients who received myeloablative conditioning, which is a strong preparatory treatment before transplant
- Those enrolled in a social security organization with health insurance coverage
- Patients who have signed informed consent
Who cannot participate:
- Patients who have not undergone donor stem cell transplantation
- Those who did not receive myeloablative conditioning
- Patients not suffering from blood-related cancers
- Vulnerable populations such as children, pregnant women, or those with mental disabilities
What the study involves: The trial aims to determine if FMT can improve the chances of patients staying free from Graft-versus-Host Disease (a condition where donor immune cells attack the recipient’s body) and cancer relapse for one year after transplant. The treatment is administered as a rectal suspension. Researchers will monitor various outcomes including overall survival, infection rates, quality of life, and changes in gut bacteria composition and diversity over time.
Study on How Rabbit Anti-Human Thymocyte Immunoglobulin Helps Prevent Graft-versus-Host Disease in Children and Adults Undergoing Stem Cell Transplants
This study examines how a medication called Thymoglobulin behaves in the body and helps prevent Graft-versus-Host Disease (GvHD) in both children and adults receiving donor stem cell transplants. GvHD occurs when the transplanted donor cells attack the recipient’s body.
Who can participate:
- Patients aged 2 years or older
- Those receiving Thymoglobulin therapy as part of their first donor stem cell transplant
- Patients with a Lansky or Karnofsky score of 50% or higher (measures ability to perform daily activities)
- Those enrolled in a social security plan
- Adult patients (18 years or older) must provide informed consent; for minors, parents or guardians must consent
- Patients of childbearing potential must use effective contraception methods throughout the study
Who cannot participate:
- Patients with severe allergic reactions to Thymoglobulin or its ingredients
- Those with uncontrolled active infections
- Patients with a history of cancer (except certain skin cancers)
- Pregnant or breastfeeding patients
- Those with severe heart, liver, or kidney problems
- Patients who received another investigational drug within the last 30 days
- Those with a history of drug or alcohol abuse in the past year
What the study involves: Participants will receive Thymoglobulin as a solution through an intravenous infusion directly into the bloodstream. This immunosuppressive medication works by reducing the activity of certain immune cells to prevent the body from attacking the transplanted cells. The study will monitor how the medication is processed by the body over time by measuring medication levels in the blood and assessing its effects on preventing GvHD. Regular follow-up assessments will evaluate overall health and treatment outcomes.
Summary
The three ongoing clinical trials address different but related complications that can occur after stem cell transplantation. One trial focuses specifically on pediatric patients with blood vessel damage using Narsoplimab, a monoclonal antibody treatment. Another investigates the novel approach of using fecal microbiota transplantation to restore gut bacteria balance and prevent complications in adult blood cancer patients. The third study examines Thymoglobulin for preventing Graft-versus-Host Disease in both children and adults.
Geographically, France hosts two of these trials, while the pediatric Narsoplimab study is conducted across three European countries: Netherlands, Spain, and Germany. This distribution suggests strong research interest in transplant complications across Europe, with France showing particular activity in this field. Each trial addresses a different aspect of post-transplant care, reflecting the complex challenges faced by patients who undergo this life-saving but demanding procedure.
