Clinical trials: Histiocytosis,Langerhans-Cell
Advanced Tumors, Melanoma
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patient With Advanced Tumors
This is a First-in-Human, Open Label, Phase I/II Study to Evaluate the Safety, Tolerability and Pharmacokinetics of HH2710 in Patients with Advanced Tumors, composed of a Phase I dose escalation and dose expansion stage and a…
- Conditions
- Advanced Tumors, Melanoma, Non-Small-Cell Lung Cancer, Erdheim-Chester Disease
- Trial name
- NCT04198818
- Testing
- HH2710
- Where
- 3 sites in United States, China
- Volunteers
- 37 people
- Sponsor
- Haihe Biopharma Co., Ltd.
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Niemann-Pick Disease, Type C
Safety and Efficacy of Miglustat in Chinese NPC Patients
This is a prospective, multi-center, open-label, non-randomized, single-arm Phase IV confirmatory study. Approximately 19 subjects with Niemann Pick Type C disease (NPC) will be enrolled in this study.
- Trial name
- NCT03910621
- Testing
- Miglustat
- Where
- 2 sites in China
- Volunteers
- 17 people
- Sponsor
- Actelion
Niemann-Pick Disease Type C, GM2 Gangliosidosis
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
This is an open-label extension trial to evaluate the long-term safety and tolerability of nizubaglustat (AZ-3102) in participants with rare lysosomal disorders who have participated in a previous nizubaglustat trial and wish to…
- Conditions
- Niemann-Pick Disease Type C, GM2 Gangliosidosis, GM1 Gangliosidosis, Gaucher Disease Type 3
- Trial name
- HARBOR (NCT07885917)
- Testing
- AZ-3102
- Volunteers
- 218 people
- Sponsor
- Azafaros B.V.
Primary Haemophagocytic Lymphohistiocytosis
Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis.
- Trial name
- NCT01818492
- Testing
- NI-0501
- Where
- 20 sites in United States, Germany and 4 more
- Volunteers
- 45 people
- Sponsor
- Swedish Orphan Biovitrum
GM2 Gangliosidosis, Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat in participants previously enrolled in the Phase 2 RAINBOW study…
- Trial name
- PRISMA (NCT07399704)
- Testing
- AZ-3102
- Where
- 3 sites in Brazil
- Volunteers
- 21 people
- Sponsor
- Azafaros B.V.
Gaucher Disease, Splenomegaly, Acid SphingoMyelinase Deficiency
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g.
- Trial name
- OPPUS (NCT04845958)
- Where
- 40 sites in France
- Volunteers
- 60 people
- Sponsor
- Sanofi
Niemann-Pick Disease, Type C
Early Access Program With Arimoclomol in US Patients With NPC
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy.
- Trial name
- NCT04316637
- Testing
- Arimoclomol
- Where
- 13 sites in United States
- Sponsor
- ZevraDenmark
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Langerhans Cell Histiocytosis, LCH
To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH
This is a rare disease, single-arm, open-label,multi-center, non-randomized Phase 2 clinical study to evaluate the efficacy, safety, and pharmacokinetic characteristics of FCN-159 monotherapy in pediatric patients with…
- Trial name
- NCT05997602
- Testing
- FCN-159
- Where
- 11 sites in China
- Volunteers
- 56 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
Granulomatosis With Polyangiitis, Hemophagocytic Syndrome
Study on Pharmacokinetics of Meperizumab Injection and NUCALA® in Healthy Male Volunteers
The trial was designed as a single-center, randomized, double-blind, single-dose parallel controlled phase I study to evaluate the similarity of pharmacokinetics and safety of Meperizumab injection and NUCALA® in healthy male…
- Trial name
- NCT05353179
- Testing
- Meperizumab injection, NUCALA®
- Where
- China
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Niemann-Pick Disease, Type C1
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1).
- Trial name
- TransportNPC (NCT04860960)
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 35 sites in United States, Argentina and 11 more
- Volunteers
- 94 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C
Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C
A prospective, randomized, double-blind, placebo controlled therapeutic study in participants with confirmed diagnosis of Niemann-Pick disease type C (NPC).
- Trial name
- NCT02612129
- Testing
- arimoclomol
- Where
- 14 sites in United States, Denmark and 7 more
- Volunteers
- 50 people
- Sponsor
- ZevraDenmark
Niemann-Pick Type C Disease
Miglustat in Niemann-Pick Type C Disease
This is a phase II randomized controlled study of miglustat in adult and juvenile patients with Niemann-Pick Type C disease. Up to 42 patients will be randomised in a 2:1 ratio to either treatment with miglustat or to a…
- Trial name
- NCT00517153
- Testing
- miglustat
- Volunteers
- 29 people
- Sponsor
- Actelion
Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic
A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)
Macrophage Activation Syndrome (MAS) is a rare, life-threatening condition characterized by uncontrolled hyperinflammation which may develop on the background of systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset…
- Conditions
- Macrophage Activation Syndrome, Lymphohistiocytosis, Hemophagocytic, Arthritis, Juvenile, Adult Onset Still Disease
- Trial name
- NCT03311854
- Testing
- Emapalumab
- Where
- 5 sites in United States, France and 3 more
- Volunteers
- 14 people
- Sponsor
- Swedish Orphan Biovitrum
Farber's Lipogranulomatosis, Ceramidase Deficiency
Biomarker for Farber Disease (BioFarber)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Farber disease from the blood
- Conditions
- Farber's Lipogranulomatosis, Ceramidase Deficiency, Hepatomegaly, Splenomegaly
- Trial name
- BioFarber (NCT02298634)
- Where
- 3 sites in Egypt, Germany and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Diffuse Astrocytoma, Anaplastic Astrocytoma
Pediatric Long-Term Follow-up and Rollover Study
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT03975829
- Testing
- dabrafenib, trametinib
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 163 people
- Sponsor
- Novartis Pharmaceuticals
Hemophagocytic Lymphohistiocytoses
A Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetic Characteristics, and Immunogenicity of Plonmarlimab in Subjects With Rheumatic and Immunological Disease-associated Haemophagocytic Lymphohistiocytosis (HLH) (Also Known as Macrophage Activation Syndrome (MAS))
This study adopts an open-label, single-arm, multicenter design to evaluate the efficacy, safety, tolerability, immunogenicity, and PK characteristics of Plonmarlimab administration in patients with rheumatic and immunological…
- Trial name
- NCT07034209
- Testing
- Plonmarlimab
- Where
- 6 sites in China
- Volunteers
- 18 people
- Sponsor
- TJ Biopharma Co., Ltd.
Primary Hemophagocytic Lymphohistiocytosis
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term…
- Trial name
- NCT03312751
- Testing
- Emapalumab
- Where
- 27 sites in United States, Canada and 6 more
- Volunteers
- 35 people
- Sponsor
- Swedish Orphan Biovitrum