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Familial Hemophagocytic Lymphohistiocytosis Type 1 — Clinical Trials

MONDO:00099743 activeLast updated: Mar 16, 2026, 3:53 AM

Trial Landscape

Familial Hemophagocytic Lymphohistiocytosis Type 1 has an emerging clinical trial program, driven by drug treatment and biological therapy.

Recruiting

Active Trials

By Approach

Drug Treatment2

Biological Therapy2

By Phase

Phase 1

Phase 2

Sponsor Types

Academic/Other3

Trial Locations

United States2 trials

France1 trial

Trials often recruit across multiple countries, so totals exceed the trial count.

3 Trials

Phase 1Not Yet RecruitingGene Therapy

NCT06736080

Safety and Efficacy of Gene Therapy of FHL Type 3 Caused by Mutations in the Human UNC13D Gene by Transplantation of a Single Dose of Autologous CD34+ Cells Transduced Ex Vivo with the UNC13D LV Vector Expressing the UNC13D CDNA

Sponsor:Assistance Publique - Hôpitaux de Paris

FranceStarted: Jan 2025Est. completion: Jan 2029Updated: Dec 2024

Phase 1Active, Not Recruiting

NCT01966367

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

Sponsor:Diane George

United StatesStarted: Mar 2013Est. completion: Feb 2030Updated: Aug 2025

Phase 1Active, Not Recruiting

NCT01494103

Administration of Donor T Cells With the Caspase-9 Suicide Gene

Sponsor:Baylor College of Medicine

United StatesStarted: Nov 2011Est. completion: May 2029Updated: Jul 2025

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Data sourced from ClinicalTrials.gov via Kisho's automated pipeline. Trials refreshed every 4 hours for monitored diseases.

Last sync: March 16, 2026 at 3:53 AM