Study of Cenegermin Nasal Spray for Safety and Tolerability in Children with Spastic Cerebral Palsy

Treatment Period 1

During this period, you will receive either cenegermin or placebo. Cenegermin is a medication that contains a protein called recombinant human nerve growth factor. The placebo looks the same but does not contain the active medication.

The medication will be administered as a nasal spray. This means the medication will be sprayed into your child's nose.

The specific dosage and frequency of administration will be determined based on your child's body weight and the study protocol. Adjustments may be made if your child weighs more than 30 kilograms.

This is a double-blinded study, which means neither you nor the study doctor will know whether your child is receiving cenegermin or placebo during this period.

Washout Period

After the first treatment period ends, there will be a break called a washout period.

During this time, your child will not receive any study medication. This allows the body to clear any medication from the previous treatment period.

Your child will continue with regular physical therapy and occupational therapy sessions as before.

Treatment Period 2

After the washout period, your child will begin the second treatment period.

This is a crossover study, which means your child will receive whichever treatment they did not receive in the first period. If your child received cenegermin in the first period, they will receive placebo in this period, and vice versa.

The medication will again be administered as a nasal spray, with the same dosage and frequency as in the first treatment period.

Neither you nor the study doctor will know which treatment your child is receiving during this period.

Safety Monitoring Throughout the Study

Throughout all treatment periods, your child will be monitored for any side effects or changes in health. This includes checking vital signs such as blood pressure, heart rate, and temperature.

Your child's weight and height will be measured regularly.

Physical examinations will be performed by the study doctor to assess your child's overall health.

Blood samples will be taken to measure the levels of the medication in your child's blood and to perform laboratory tests.

An electrocardiogram (ECG) will be performed. This is a test that records the electrical activity of the heart using small sensors placed on the skin.

An electroencephalogram (EEG) will be performed. This is a test that records the electrical activity of the brain using small sensors placed on the scalp.

Assessment of Spasticity and Function

The study doctor will assess your child's spasticity (muscle stiffness) using a scale called the Modified Ashworth Scale (MAS). This measures how much resistance there is when the doctor moves your child's joints.

Your child's motor function will be assessed using the Gross Motor Function Measure (GMFM-66) and the Gross Motor Function Classification System (GMFCS-E&R). These tools measure how well your child can perform movements like sitting, standing, and walking.

You will be asked to complete questionnaires about your child's daily activities, social interactions, thinking skills, and communication abilities. These assessments are called PEDI-CAT (Pediatric Evaluation of Disability Inventory - Computer Adaptive Test) and CFCS (Communication Function Classification System).

You may also be asked to complete a Patient-Reported Outcome Measure (PROM), which is a questionnaire about your observations of your child's condition and progress.

Continuation of Regular Therapy

Your child must continue to receive physical therapy and/or occupational therapy throughout the entire study.

Your child should have been receiving these therapies for at least three months before joining the study and will need to continue them for the full duration of the study.

These regular therapy sessions are an important part of your child's care and must be maintained while participating in the study.