Not recruitingRare disease

Study on the Safety and Effectiveness of BOOST Cells for Treating Severe Osteogenesis Imperfecta in Children Before and After Birth

Fast replyInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Osteogenesis Imperfecta, often referred to as Brittle Bone Disease. This condition is characterized by bones that break easily, often with little or no apparent cause. The study specifically targets severe forms of this disease, including Osteogenesis Imperfecta type III and severe type IV. The treatment being tested in this trial involves the use of BOOST cells, which are a type of cell therapy made from allogeneic fetal mesenchymal stem cells. These cells are derived from the liver of a human fetus and are used in a solution that is administered through an infusion into a vein.

The purpose of this study is to evaluate the safety and tolerability of administering these stem cells to children and fetuses with severe forms of Brittle Bone Disease. Participants in the study will receive four doses of the BOOST cells, either after birth or both before and after birth. The study will compare the outcomes of these treatments with historical data and untreated controls. The trial aims to understand how safe the treatment is and how well it is tolerated by the participants.

Throughout the study, researchers will monitor the participants for any side effects related to the treatment. They will also track the number of bone fractures, the time it takes for a fracture to occur after the last dose, and changes in bone density and growth. The study will also look at changes in the clinical status of the disease and biochemical markers related to bone turnover. The trial is expected to continue until 2030, providing valuable insights into the potential benefits and risks of using BOOST cells for treating severe Brittle Bone Disease.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
The study involves children diagnosed with a severe form of Brittle bone disease, known as Osteogenesis Imperfecta (OI) type III or severe type IV.
Participation requires a signed informed-consent form from a parent or legal guardian.
Eligible participants are under 18 months old, with a specific genetic diagnosis related to the COL1A1 or COL1A2 gene.
Step 2
Treatment administration
The treatment involves the use of BOOST cells, which are allogeneic fetal mesenchymal stem cells.
These cells are administered as a solution for infusion through an intravenous route.
Participants receive four doses of the treatment, either after birth or both before and after birth.
Step 3
Monitoring and assessment
The primary focus is on assessing the safety and tolerability of the treatment in children.
Monitoring includes tracking any treatment-related adverse events, such as their seriousness, severity, and frequency.
Secondary assessments involve measuring the number of fractures, changes in bone mineral density, growth, and overall clinical status of the disease.
Step 4
Follow-up
The study includes a long-term follow-up to evaluate the time to the first fracture after the last dose and changes in bone health over time.
The study is expected to continue until April 2030, allowing for comprehensive data collection and analysis.

Who can join the trial?

5 criteria

The child's parent or legal guardian must sign a form giving their permission for the child to participate.
The child must have a clinical diagnosis of **Osteogenesis Imperfecta (OI)** type III or type IV. This is a condition also known as **brittle bone disease**, where bones break easily.
The child must have a **molecular diagnosis** of OI, which means there is a specific change in the genes called **COL1A1** or **COL1A2** that affects bone strength.
The child must be less than 18 months old. This age is calculated from what would be the full-term birth date (40 weeks of pregnancy).
The parent or legal guardian must be over 18 years old.

Who cannot join the trial?

8 criteria

Patients who do not have **Osteogenesis Imperfecta (OI)** type III or severe type IV cannot participate. **Osteogenesis Imperfecta** is a condition that makes bones very fragile and easy to break.
Individuals who are not within the specified age range for the study are excluded. The study includes specific age groups, so those outside these groups cannot join.
Participants who are not able to receive the treatment through an **intravenous** method are excluded. **Intravenous** means the treatment is given directly into a vein.
Patients who have other medical conditions that might interfere with the study or make it unsafe for them to participate are excluded.
Individuals who are unable to follow the study procedures or attend the required visits are excluded.
Participants who are pregnant or planning to become pregnant during the study period are excluded, unless the study specifically includes prenatal treatment.
Patients who have participated in another clinical trial recently may be excluded to avoid interference with the study results.
Individuals who have allergies or reactions to the study treatment or its components are excluded.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Karolinska University Hospital
Verified
Solna, Sweden
Leids Universitair Medisch Centrum (LUMC)
Responsive
Leiden, The Netherlands
Universitair Medisch Centrum Utrecht
Responsive
Utrecht, The Netherlands

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Investigated drugs

BOOST cells are a type of stem cell therapy used in this clinical trial. These cells are derived from fetal mesenchymal stem cells and are expanded in the laboratory. The therapy involves administering these cells intravenously to patients with severe Osteogenesis Imperfecta (OI), a genetic disorder characterized by fragile bones. The goal of using BOOST cells is to evaluate their safety and effectiveness in improving bone strength and reducing fractures in affected individuals.

Allogeneic Fetal Mesenchymal Stem Cells

What is already known about the treatment

BOOST cells – This medication is administered intravenously, either postnatally or both prenatally and postnatally. It is currently being explored in clinical trials for its safety and effectiveness in treating severe forms of Osteogenesis Imperfecta, a condition also known as brittle bone disease. The main therapeutic indication is for Osteogenesis Imperfecta type III and severe type IV. BOOST cells work at the molecular level by utilizing allogeneic expanded fetal mesenchymal stem cells, which are believed to aid in bone strength and repair. They are classified under cell-based therapies, focusing on regenerative medicine.

Investigated diseases

Osteogenesis Imperfecta (OI) Type III and Severe Type IV – Osteogenesis Imperfecta is a genetic disorder characterized by fragile bones that break easily, often with little or no apparent cause. Type III is a severe form where fractures are common, and individuals may experience bone deformities, short stature, and respiratory issues. Severe Type IV is similar but may present with slightly milder symptoms compared to Type III. Both types can lead to frequent fractures, bone pain, and potential hearing loss. The condition is present from birth and affects the development and strength of bones throughout life. It is caused by defects in the genes responsible for producing collagen, a crucial protein for bone strength.