What is the purpose of this study?
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 is involved?
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.
What are the benefits?
You or your child may or may not receive direct benefit from participating in this study. There may be an improved chance of successfully treating your child’s HLH. Information learned in this study may benefit other patients with HLH in the future. Two patients suffering from primary HLH have been treated so far with NI-0501 and then proceeded to transplantation.
Will I get all the facts about the study?
Parents and adult subjects will be given a consent form that thoroughly explains all of the details of the study. The consent form will cover all of the procedures, risks, benefits, compensation, who to contact with questions or concerns and more. A member of the study staff will review the consent form with you and answer your questions. Study procedures will not begin until the parent/guardian or you, the participating subject, have signed this consent form.