Study of Narsoplimab for Children with High-Risk Blood Vessel Damage After Stem Cell Transplant

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What is this study about?

This clinical trial is focused on studying a condition called Haematopoietic Stem Cell Transplant Thrombotic Microangiopathy (HSCT-TMA), which can occur in children who have undergone a stem cell transplant. This condition involves damage to small blood vessels, leading to problems such as low platelet counts and other complications. The study is testing a treatment called Narsoplimab, which is given as a solution for injection. Narsoplimab is being investigated to see if it can help improve the survival rate and overall health of children affected by this condition.

The purpose of the study is to evaluate the effectiveness and safety of Narsoplimab in children aged 28 days to 18 years who have been diagnosed with high-risk HSCT-TMA. Participants in the study will receive Narsoplimab through an intravenous injection, which means it is administered directly into a vein. The study will monitor the participants over a period to assess their survival rate 100 days after the diagnosis of HSCT-TMA, as well as other health outcomes over a longer period.

Throughout the study, researchers will also look at how the body processes Narsoplimab and any potential side effects. The study aims to gather important information on how well Narsoplimab works in treating HSCT-TMA and its safety in pediatric patients. This research could provide valuable insights into managing this serious condition in children who have undergone a stem cell transplant.

1 joining the study

Eligibility is confirmed based on age, consent, and medical history.

Informed consent is obtained from a parent or legal guardian, and assent from the patient if required.

2 initial assessment

A diagnosis of high-risk haematopoietic stem cell transplant thrombotic microangiopathy (HSCT-TMA) is confirmed.

Baseline health assessments are conducted, including blood tests and other necessary evaluations.

3 treatment administration

The medication narsoplimab is administered as a solution for injection through an intravenous route.

The dosage and frequency of administration are determined by the study protocol and the patient’s specific needs.

4 monitoring and follow-up

Regular monitoring of health status and response to treatment is conducted.

Blood tests and other assessments are performed to evaluate the effectiveness and safety of the treatment.

5 completion of treatment

The treatment phase concludes after a specified period, as outlined in the study protocol.

Final assessments are conducted to evaluate the overall health and response to treatment.

6 post-treatment follow-up

Follow-up visits are scheduled to monitor long-term health outcomes.

Additional assessments may be conducted to gather data on survival rates and other study endpoints.

Who Can Join the Study?

Be at least 28 days old and less than 18 years old before giving consent to participate.
Have consent from at least one parent or legal guardian, as required by local laws. If the patient is old enough according to local laws, they must also give their own consent.
Agree to participate, as required by local laws.
Have received a type of treatment called an allogeneic haematopoietic stem cell transplant for a disease, which can be either cancerous or non-cancerous. This means the stem cells came from a donor, and any type of donor match is acceptable.
Have a diagnosis of high-risk HSCT-TMA, which includes having a low platelet count (less than 50,000 per microliter) or a significant drop in platelet count, and signs of a condition called microangiopathic hemolysis (such as certain blood cell fragments, high levels of a substance called LDH, or low levels of a protein called haptoglobin).
Meet at least one of the following high-risk criteria for HSCT-TMA:
- HSCT-TMA lasting 2 weeks or more after changing certain medications.
- Signs of high-risk HSCT-TMA, such as:
  - A high protein/creatinine ratio in urine.
  - Abnormal kidney function related to TMA.
  - Evidence of TMA in the digestive system from a biopsy.
  - Nervous system problems related to TMA, like confusion or seizures.
  - Fluid around the heart or lungs without another cause.
  - High blood pressure in the lungs without another cause.
  - Severe or potentially severe GVHD (a condition where the donor cells attack the patient’s body) or a risk of developing it if medications are changed.
  - High levels of a substance called C5b-9 in the blood.
If sexually active and able to have children (for females, starting from the first menstrual period), must agree to use a very effective method of birth control during the study and for at least 12 weeks after the last dose of the study drug. Effective methods include implants, injections, certain birth control pills, some devices placed in the uterus, complete sexual abstinence, or having a partner who has had a vasectomy.
Male patients must agree not to father children during the study and for at least 12 weeks after the last dose of the study medication.

Who Cannot Join the Study?

Patients who have had a stem cell transplant but do not have thrombotic microangiopathy. Stem cell transplant is a procedure to replace damaged or destroyed bone marrow with healthy stem cells. Thrombotic microangiopathy is a condition where small blood clots form in small blood vessels.
Patients who are not considered high-risk for the condition. High-risk means having a greater chance of developing complications.
Patients who are not within the specified age range for the study. Age range refers to the specific ages that are allowed to participate.
Patients who do not belong to the specified clinical trial groups. Clinical trial groups are categories of patients that the study is focusing on.
Patients who are not part of the vulnerable population selected for the study. Vulnerable population refers to groups of people who may need special protection or consideration.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

Site Name	City	Country	Status
Medizinische Hochschule Hannover	Hanover	Germany
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country	Status
Justus-Liebig-Universitaet Giessen	Giessen	Germany
Pxarwlow Mghtce Ctvrck Uquwcny	Utrecht	The Netherlands

Trial status

Country	Status	Recruitment Start
Germany	Recruiting	03.05.2023
Spain	Recruiting	03.05.2023
The Netherlands	Recruiting	03.05.2023

Trial locations

Investigated drugs:

Narsoplimab

Narsoplimab is a medication being studied for its effectiveness and safety in treating children and adolescents who have developed a condition called thrombotic microangiopathy (TMA) after undergoing a high-risk hematopoietic stem cell transplant (HSCT). This medication is being tested to see if it can help improve survival rates and manage symptoms associated with this condition.

Investigated diseases:

Thrombotic microangiopathy

Haematopoietic Stem Cell Transplant Thrombotic Microangiopathy – This condition occurs after a stem cell transplant and involves damage to small blood vessels, leading to the formation of blood clots. These clots can cause a reduction in blood flow to vital organs, potentially leading to organ dysfunction. The disease is characterized by the destruction of red blood cells, low platelet counts, and kidney problems. Symptoms may include fatigue, bruising, and high blood pressure. It is considered a rare complication of stem cell transplantation. The progression of the disease can vary, with some cases resolving spontaneously while others may persist or worsen.

Trial ID:

2023-509710-11-00

Protocol code:

OMS721-HCT-002

NCT ID:

NCT05855083

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of Narsoplimab for Children with High-Risk Blood Vessel Damage After Stem Cell Transplant

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?