What will happen in the study?

There is an anticipated 8-week treatment period with NI-0501 followed by an additional 4 week follow up period after the last dose of NI-0501. However, the protocol also allows for a shorter treatment duration, in case hematopoietic stem cell transplantation can be performed before the end of the treatment period, or a longer treatment duration in case transplantation must be postponed. The following is a list of procedures that will take place during the study:

Screening Period

Before treatment begins, you/your child will have tests done to confirm the study eligibility and assess the disease status. This will includes drawing blood and other samples, looking for infections, testing the immune system, spinal tap, test/picture to measure heart rhythm (electrocardiogram, ECG), and radiology tests such as ultrasound and chest x-ray (as well as an MRI if needed).

Treatment Period

The treatment regimen is divided into two phases:

·         Treatment Period 1 is characterized by close monitoring during NI-0501 administrations, 6 infusions given every 3 days. In addition, patients will receive Dexamethasone for the duration of the treatment with the study therapy.

·         Treatment Period 2 is characterized by less intense monitoring and includes 14 infusions, or less depending on the patient’s needs and conditions (Weeks 3 to 8). The time between NI-0501 infusions can be increased; however this will not impact the safety assessments which will occur at least every 6 days.

All patients will be followed closely throughout the 8 weeks on study to test for response to treatment and to look for side effects of the medications with routine and other tests, such as physical examinations, blood tests, ECG and radiology tests.