Why are we doing this research?
Cincinnati Children’s is participating in a research study sponsored by
NovImmune S.A. to evaluate the safety, tolerability and preliminary efficacy of
a new drug (NI-0501) aimed at controlling disease activity in patients
diagnosed with primary hemophagocytic lymphohistiocytosis who show an
unsatisfactory response to current recommended treatment or show intolerance to
drug will be administered on top of a glucocorticosteroid, which is usually
part of the current recommended treatment.
Who can participate?
Children and adolescents, up to and including 18 years old at diagnosis of
primary HLH, who, after having received conventional therapy, continue to have
active disease, are potentially eligible.
Children or adolescents with intolerance to HLH conventional treatment
can also participate in the study.
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
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
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
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.