ClinicalTrials.gov record NCT05277272
INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)
INTO-HLH- Insight Into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (HLH): A Disease Registry for Patients With HLH
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a prospective observational registry that collects clinical and outcome data on people with clinically suspected or confirmed hemophagocytic lymphohistiocytosis (HLH). The study gathers information on diagnosis timing, treatments given, genetic testing, complications, and long-term outcomes up to five years. The goal is to describe the natural history and treatment outcomes of HLH.
What it is studying. The study is gathering real-world information to describe how HLH presents, how long diagnosis takes, what treatments patients receive and how they respond, how often genetic causes or infections are present, and what complications or long-term outcomes (including after stem cell transplant) occur.
What the researchers hope to learn. To define the natural history and clinical and etiologic diversity of HLH; to measure time to diagnosis; to describe treatment patterns and responses; to estimate the frequency of genetic diagnoses, infections, CNS involvement, organ failure, and long-term complications; and to summarize outcomes after hematopoietic stem cell transplantation.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The purpose of this observational study is to collect data on the natural history of disease of patients with Hemophagocytic Lymphohistiocytosis (HLH) including diagnosis, treatments, responses, and outcomes.
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Patients with clinically suspected or confirmed HLH, including those meeting the HLH-2004 diagnostic criteria (primary or secondary forms, including malignancy) and other forms of HLH (macrophage activation syndrome [MAS], cytokine release syndrome [CRS], etc.)
- Signed and dated informed consent and assent (adolescents)
Exclusion criteria
- None
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
Hemophagocytic Lymphohistiocytosis (HLH) is a complex, hyperinflammatory syndrome resulting from the interplay of genetic predisposition and various environmental factors. Despite available treatment options for HLH, approximately 30% of patients do not respond to therapy. Moreover, the standard therapy is constrained by its toxicities, and safer treatments are pursued. There is an unmet need for a deeper understanding of the natural history, clinical/etiologic diversity, complications, and treatment outcomes of patients with HLH, specifically from North America. The proposed study, a collaboration between Cincinnati Children's Hospital Medical Center (CCHMC), Texas Children's Hospital, and Sobi Inc. aims to establish a robust registry that will enable investigators to better define the natural history of HLH.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
If you join, the study team will collect information from your medical records about diagnosis, treatments, laboratory and imaging results, genetic testing results, and outcomes (this may include MRI or cerebrospinal fluid/spinal tap results if those were done as part of clinical care). They will record dates (for example first symptoms and HLH diagnosis), infections, organ failure, treatments and responses, any hematopoietic stem cell transplant (HSCT) and related complications, and survival. Participants' data will be followed and gathered for up to five years after HLH diagnosis. The registry record requires informed consent/assent and does not specify any study-driven tests or treatments.
How the study is structured. A multi-center, prospective observational registry (cohort). No treatments are assigned by the study. Medical records, test results, and follow-up data will be collected and followed for up to five years to track diagnosis, treatments, responses, complications, and survival.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on hemophagocytic lymphohistiocytosis
Important dates
- Study start
- Dec 31, 2021
- Primary completion
- January 2027 (estimated)
- Study completion
- January 2027 (estimated)
- Target enrollment
- 200 participants (estimated)
- Last updated on registry
- Feb 24, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Collaborators
- Sobi, Inc.
- Baylor College of Medicine
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Michael Jordan, MD 📞 (513) 803-9063 ✉ Michael.Jordan@cchmc.org
- Adi Zoref Lorenz, MD ✉ Adi.Zoref.Lorenz@cchmc.org
Participating sites (1)
- Cincinnati Children's Hospital Medical CenterRecruiting
Cincinnati, Ohio, 45229 · United States
- Michael Jordan, MD · 513-803-9063 · Michael.Jordan@cchmc.org
- Michael Jordan, MD
Terms used on this page · explanations by MedicalUpdates.ai
- natural history
- A study that follows how a condition changes over time without assigning an experimental treatment. It may involve study visits and tests, and is often used to plan future trials.
- cytokine release syndrome
- A reaction in which the immune system releases a large amount of signalling proteins at once, causing fever and sometimes low blood pressure or breathing problems. It can occur after some immune therapies, such as CAR-T cells or bispecific antibodies.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- standard therapy
- Treatment generally considered appropriate for a condition in a particular clinical setting. It can vary by patient, place and over time. In many studies the new treatment is added to, or compared with, standard of care.
- registry
- An organised collection of information about people with a particular condition, gathered over time for research. Joining a registry usually means sharing medical information, not receiving a treatment.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
← Back to clinical trials