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Recruiting NCT05277272

INTO-HLH: A Disease Registry for Patients With Hemophagocytic Lymphohistiocytosis (HLH)

— Observational Hemophagocytic Lymphohistiocytoses

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to
Registry conditions: Hemophagocytic Lymphohistiocytoses. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

INTO-HLH- Insight Into the Natural History and Treatment Outcomes of Hemophagocytic Lymphohistiocytosis (HLH): A Disease Registry for Patients With HLH

Overview

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.

Detailed description

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.

Primary outcome measures

  • Time to HLH diagnosis from the initial presentation [Time frame: Interval between date of presentation, as defined as the day of appearance of initial HLH symptom, and the date of full HLH diagnosis, as defined by fulfilling the HLH diagnostic criteria, will be measured. Timeframes up to 6 months will be assessed.]
  • Number of patients with an autoimmune disease at the time of HLH diagnosis [Time frame: Up to 1 month from HLH diagnosis]
  • Number of patients with malignancy at the time of HLH diagnosis [Time frame: Up to 1 month from HLH diagnosis.]
  • Number of patients treated with immune-activating agents before HLH diagnosis [Time frame: Up to 1 month before HLH diagnosis.]
  • Number of patients with central nervous system (CNS) involvement during the HLH disease course. [Time frame: Up to 1 month from HLH diagnosis.]
  • Frequency of a genetic diagnosis underlying the HLH. [Time frame: Up to 1 month from HLH diagnosis.]
  • Number of patients with infections (e.g., EBV, CMV, HHV6, HIV, fungal, bacterial) at the time of diagnosis. [Time frame: Up to 1 month from HLH diagnosis.]
  • Number of patients with organ failure. [Time frame: Up to 1 year from HLH diagnosis.]
  • Number of patients with long-term disease-related complications. [Time frame: Up to 5 years from HLH diagnosis.]
Secondary outcome measures (6)
  • Treatment response rate to HLH-related treatments. [Time frame: Week two from the start of treatment.]
  • Time to response to HLH-related therapy for patients in the registry. [Time frame: Assessed up to 12 weeks from start of treatment.]
  • The survival probability of patients in the registry [Time frame: From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.]
  • Number of patients who received hematopoietic stem cell transplantation (HSCT) [Time frame: From HLH diagnoses up to 5 years post-HLH diagnosis.]
  • Frequency of hematopoietic stem cell transplantation (HSCT) related complications [Time frame: From HSCT up to 5 years post HSCT.]
  • Number of participants with treatment-related adverse events >/= 3 as assessed by CTCAE 5.0 [Time frame: From initiation of HLH related treatment up to 30 days following discontinuation of treatment.]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

United States · 1 center
  • Cincinnati Children's Hospital Medical Center — Cincinnati

Identifiers

NCT: NCT05277272 · 2021-0821 · Sobi.HLH-RWE102

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗