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

ConditionHemophagocytic Lymphohistiocytoses
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorChildren's Hospital Medical Center, Cincinnati

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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)

Disqualifiers

None

Trial population

Patients with clinically suspected or confirmed HLH

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

200 Participants
are grouped into 1 trial group
Group A: Patients with clinically suspected or confirmed Hemophagocytic Lymphohistiocytosis

Trial outcomes

Primary outcomes

1

Time to HLH diagnosis from the initial presentation

Date of initial presentation and the date of HLH diagnosis as defined by HLH diagnostic criteria (HLH-2004/MAS classification criteria)

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.
2

Number of patients with an autoimmune disease at the time of HLH diagnosis

Presence of an autoimmune disease at the time of diagnosis (e.g., Systemic juvenile idiopathic arthritis, lupus)

Time frame
Up to 1 month from HLH diagnosis
3

Number of patients with malignancy at the time of HLH diagnosis

Presence of hematologic and solid malignancies at the time of HLH diagnosis.

Time frame
Up to 1 month from HLH diagnosis.
4

Number of patients treated with immune-activating agents before HLH diagnosis

The number of patients treated with immune-activating agents before initial diagnosis (checkpoint inhibitors, CAR-T constructs)

Time frame
Up to 1 month before HLH diagnosis.

Secondary outcomes

1

Treatment response rate to HLH-related treatments.

The response of all treatments for all patients using the criteria used to assess the efficacy of anti-IFN treatment in the NI-0501-04 04 and NI-0501-14 clinical trials.

Time frame
Week two from the start of treatment.
2

Time to response to HLH-related therapy for patients in the registry.

The response of all treatments for all patients using the criteria used to assess the efficacy of anti-IFN treatment in the NI-0501-04 04 and NI-0501-14 clinical trials.

Time frame
Assessed up to 12 weeks from start of treatment.
3

The survival probability of patients in the registry

Data on the occurrence and date of death and the date of last documentation for living patients will be gathered.

Time frame
From HLH diagnosis to last follow-up or death, whichever comes first, assessed up to 5 years post-HLH diagnosis.
4

Number of patients who received hematopoietic stem cell transplantation (HSCT)

Data on the frequency of HSCT will be gathered.

Time frame
From HLH diagnoses up to 5 years post-HLH diagnosis.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital Medical Center, Cincinnati

Lead sponsor

Sobi, Inc.

Collaborator

Baylor College of Medicine

Collaborator

Source ClinicalTrials.gov
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