Study of Pegcetacoplan (APL-2) in Patients with Transplant-associated Thrombotic Microangiopathy After Stem Cell Transplantation

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What is this study about?

This study focuses on patients with Transplant-associated Thrombotic Microangiopathy (TA-TMA), a condition that can occur after receiving a hematopoietic stem cell transplant. TA-TMA is a serious complication that affects small blood vessels and can cause damage to various organs. The study will test a medication called pegcetacoplan (also known as APL-2) in patients who have developed this condition after their transplant.

The purpose of this research is to understand how the body processes pegcetacoplan and to evaluate if it is safe and well-tolerated by patients with TA-TMA. The medication works by targeting part of the body’s immune system known as the complement system, which plays a role in this condition.

This is an open-label study where all participants will receive pegcetacoplan. The study will monitor various aspects of patients’ health, including blood tests and organ function, for 24 weeks. The research will look at how the medication affects certain proteins in the blood and whether it helps improve the symptoms of TA-TMA, including kidney function, blood cell counts, and other health measures.

1 Initial medical assessment

A medical evaluation will confirm if you meet the study requirements after stem cell transplantation

Your blood tests must show specific changes including low platelet count (below 50 x 109/L) and elevated LDH (a blood enzyme)

2 Additional testing requirements

You must have at least 2 additional markers from the following:

Presence of specific blood cells called schistocytes in blood samples

Low blood count (anemia) requiring blood transfusions

Protein in urine (30 mg/dL or higher)

Elevated levels of a protein called sC5b-9 in blood

High blood pressure (140/90 mmHg or higher)

3 Treatment period

The study medication pegcetacoplan will be administered

Treatment continues for 24 weeks

Regular blood and urine tests will monitor your response to treatment

4 Monitoring visits

Your progress will be checked at week 12 and week 24

Tests will measure changes in blood markers and organ function

Your kidney, lung, digestive system, and nervous system function will be evaluated

Blood pressure and other cardiovascular measurements will be taken

5 Final assessment

At week 24, final evaluations will determine if the treatment was successful

Success is measured by improvement in blood test results and organ function

Your need for blood transfusions will be assessed

Long-term survival will be monitored up to 100 days from diagnosis

Who Can Join the Study?

Must be at least 18 years old at the time of signing consent
Must have received a stem cell transplant from a related or unrelated donor, using one of these sources:
– Stem cells from blood
– Bone marrow
– Umbilical cord blood
Must have a diagnosis of transplant-associated TMA with the following signs:
– Low platelet count (below 50 x 109/L or more than 50% decrease)
– High LDH levels (a blood enzyme showing tissue damage)
– At least 2 additional signs from:
• Presence of damaged red blood cells in blood tests
• Low blood hemoglobin or need for blood transfusions
• Protein in urine
• High levels of specific blood proteins (sC5b-9)
• High blood pressure
Must have TMA that continues despite initial treatment
Must have elevated protein levels in urine (rUPCR ≥ 1 mg/mg)
For women who can become pregnant:
– Must have negative pregnancy test
– Must use approved birth control during study and 8 weeks after
For men:
– Must avoid fathering children
– Must use approved birth control
– Must not donate sperm during study and 8 weeks after
Must be able to understand and sign the informed consent form

Who Cannot Join the Study?

Patients under 12 years old cannot participate
Patients who have had organ transplants other than stem cell transplants
Patients with active, uncontrolled bacterial, viral, or fungal infections
Patients who have received another experimental treatment within 30 days before starting this study
Pregnant or breastfeeding women
Patients with severe liver problems (defined as liver enzymes more than 3 times the normal level)
Patients with severe kidney problems (requiring regular dialysis – a process to filter blood when kidneys don’t work properly)
Patients with active cancer that requires treatment
Patients with severe heart conditions
Patients who have had major surgery in the past 30 days
Patients who are allergic to any components of the study medication (pegcetacoplan)
Patients who cannot follow study procedures or attend scheduled visits
Patients with severe mental health conditions that could interfere with the study
Patients who are currently participating in another clinical trial

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Hospital Universitario Puerta De Hierro De Majadahonda	Majadahonda	Spain
Geniko Nosokomeio Thessalonikis George Papanikolaou	Thessaloniki	Greece
Fondazione IRCCS San Gerardo Dei Tintori	Monza	Italy
AORN San Giuseppe Moscati Avellino	Avellino	Italy
ASST Grande Ospedale Metropolitano Niguarda	Milan	Italy
Centre Hospitalier Universitaire De Nice	Nice	France
Hopital Beaujon	Clichy	France
Aqhonls Oxpmlglffgd Oalqalbk Rwxfzfu Vwtlc Scuka Cflisvzn	Palermo	Italy
Ipljesye do Cmrgfnbotkrf Hizaribqcgp Uuimuohfewepy dl Swsls Eppcpmv (vrcxacw	Saint Priest En Jarez	France
Uisvqjjdme Gbguyuq Hptmzzvm Afblicw	Athens	Greece

Trial status

Country	Status	Recruitment Start
France	Not recruiting	01.02.2022
Greece	Not recruiting	01.02.2022
Italy	Not recruiting	01.02.2022
Spain	Not recruiting	01.02.2022

Trial locations

Pegcetacoplan is a medication designed to treat patients who have developed blood vessel complications (thrombotic microangiopathy) after receiving a stem cell transplant. It works by targeting and regulating a part of the immune system called the complement system. This medication is administered to help manage and potentially improve the condition of patients who experience these transplant-related complications.

Investigated diseases:

Thrombotic microangiopathy

Transplant-associated Thrombotic Microangiopathy (TA-TMA) – A rare complication that can occur after stem cell transplantation or organ transplantation. This condition affects small blood vessels throughout the body, causing damage to their walls and formation of blood clots. The disease process leads to the destruction of red blood cells, low platelet count, and can affect various organs including the kidneys. TA-TMA typically develops within the first 100 days after transplantation, though it can occur later. The condition causes changes in blood composition and can affect multiple organ systems.

Trial ID:

2023-510443-37-00

Protocol code:

Sobi.PEGCET-201

NCT ID:

NCT05148299

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of Pegcetacoplan (APL-2) in Patients with Transplant-associated Thrombotic Microangiopathy After Stem Cell Transplantation

What is this study about?

Who Can Join the Study?

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