Clinical trials: Histiocytosis
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
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
Leukemia, Lymphoma
BL22 Immunotoxin In Treating Young Patients With Relapsed or Refractory Acute Lymphoblastic Leukemia or Non-Hodgkin's Lymphoma
RATIONALE: BL22 immunotoxin can locate tumor cells and kill them without harming normal cells. BL22 immunotoxin may be effective in treating relapsed or refractory acute lymphoblastic leukemia and non-Hodgkin's lymphoma.
- Trial name
- NCT00077493
- Testing
- BL22 immunotoxin, antibody-drug conjugate therapy, immunotoxin therapy
- Where
- United States
- Volunteers
- 95 people
- Sponsor
- MedImmune LLC
NHL, Relapsed, Adult, B-cell Lymphoma
Allogeneic NK T-Cells Expressing CD19 Specific CAR in B-Cell Malignancies
This study is a multi-center study to evaluate the safety of KUR-502 in subjects with refractory/relapsed B-cell NHL or leukemia (ALL or CLL).
- Conditions
- NHL, Relapsed, Adult, B-cell Lymphoma, B-cell Leukemia, DLBCL - Diffuse Large B Cell Lymphoma
- Trial name
- ANCHOR2 (NCT05487651)
- Testing
- KUR-502
- Where
- 3 sites in United States
- Volunteers
- 36 people
- Sponsor
- Athenex, Inc.
Leukemia, Lymphoma
Study of @neWorld: A Virtual Community for Children With Cancer
RATIONALE: Using an Internet Web site that enables children with cancer to interact online with classmates, participate in classroom activities, get easy-to-read medical information, and chat with family members, medical staff…
- Conditions
- Leukemia, Lymphoma, Psychosocial Effects of Cancer and Its Treatment, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00070421
- Testing
- complementary or alternative medicine procedure, management of therapy complications, psychosocial assessment and care
- Where
- United States
- Sponsor
- Leap of Faith Technologies
Hemophagocytic Lymphohistiocytosis
A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under…
- Trial name
- NCT02069899
- Testing
- Emapalumab
- Where
- 16 sites in United States, France and 3 more
- Volunteers
- 58 people
- Sponsor
- Swedish Orphan Biovitrum
Lymphoma, Large B-Cell, Diffuse
A Study of Durvalumab in Combination With R-CHOP or Lenalidomide Plus R-CHOP in Previously Untreated High-Risk Diffuse Large B-Cell Lymphoma
This Phase 2, two-arm, open-label study is designed to evaluate the safety, clinical activity, and predictive biomarkers of durvalumab in combination with R-CHOP or R2-CHOP, followed by durvalumab consolidation therapy in…
- Trial name
- NCT03003520
- Testing
- Durvalumab, Rituximab, Doxorubicin
- Where
- 20 sites in United States, Austria and 3 more
- Volunteers
- 46 people
- Sponsor
- Celgene
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
Brain and Central Nervous System Tumors, Leukemia
Chemotherapy in Treating Young Patients With Recurrent or Refractory Meningeal Leukemia, Lymphoma, or Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of liposomal cytarabine in treating young patients who have…
- Conditions
- Brain and Central Nervous System Tumors, Leukemia, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00003073
- Testing
- liposomal cytarabine
- Where
- 6 sites in United States, Canada
- Volunteers
- 15 people
- Sponsor
- Pacira Pharmaceuticals, Inc
Acid Sphingomyelinase Deficiency, Niemann-Pick Disease
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.
- Trial name
- NCT00410566
- Testing
- rhASM
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Genzyme, a Sanofi Company
Niemann-Pick Disease, Type C
VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.
- Trial name
- NCT02534844
- Testing
- Parts A/B: Adrabetadex
- Where
- 29 sites in United States, Australia and 7 more
- Volunteers
- 56 people
- Sponsor
- Mandos LLC
GM2 Gangliosidosis, Niemann-Pick Disease, Type C
Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease
This phase 2 is a randomized, double-blind, placebo controlled, 12 weeks study with daily oral administration of AZ-3102 aiming to evaluate the safety and pharmacokinetic (PK) profile in GM2 Gangliosidosis and Niemann-Pick type C…
- Trial name
- RAINBOW (NCT05758922)
- Testing
- AZ-3102 (Dose 1), AZ-3102 (Dose 2)
- Where
- 3 sites in Brazil
- Volunteers
- 13 people
- Sponsor
- Azafaros B.V.
DLBCL - Diffuse Large B Cell Lymphoma
Study to Evaluate the Effectiveness of Tafasitamab in Combination With Lenalidomide Followed by Tafasitamab Monotherapy in Relapsed or Refractory Diffuse Large B-cell Lymphoma Non-transplant Eligible Patients in Italy (PRO-MIND)
The PRO-MIND study is an Italian, multicenter, prospective observational cohort study to evaluate the effectiveness and the safety of tafasitamab in combination with lenalidomide followed by tafasitamab monotherapy in patient…
- Trial name
- NCT06299553
- Where
- Italy
- Volunteers
- 200 people
- Sponsor
- Incyte Biosciences Italy S.r.l
Hemophagocytic Syndrome
Evaluate the Efficacy and Safety of PEG-rhG-CSF in Patients With Hemophagocytic Syndrome
Hemophagocytic syndrome (HPS), also known as hemophagocytic lymphohistiocytosis (HLH), is an immune mediated life-threatening disease. There is no uniform recommendation for salvage treatment of HLH.
- Trial name
- NCT04500886
- Testing
- PEG-rhG-CSF, rhG-CSF
- Where
- China
- Volunteers
- 50 people
- Sponsor
- CSPC Baike (Shandong) Biopharmaceutical Co., Ltd.
Diffuse Large B-Cell Lymphoma, Not Otherwise Specified, High-grade B-cell Lymphoma
P-CD19CD20-ALLO1 Allogeneic CAR-T Cells in the Treatment of Subjects With B Cell Malignancies
Phase 1 study comprised of open-label, dose escalation and expansion cohort study of P-CD19CD20-ALLO1 allogeneic T stem cell memory (Tscm) CAR-T cells in subjects with relapsed/refractory B cell malignancies
- Conditions
- Diffuse Large B-Cell Lymphoma, Not Otherwise Specified, High-grade B-cell Lymphoma, Primary Mediastinal Large B-cell Lymphoma (PMBCL), Transformed Follicular Lymphoma (tFL)
- Trial name
- NCT06014762
- Testing
- P-CD19CD20-ALLO1, Rimiducid
- Where
- 16 sites in United States
- Volunteers
- 120 people
- Sponsor
- Poseida Therapeutics, Inc.
Niemann-Pick Disease, Type C
Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease
This is a Phase-2, multicenter, multiple dose, open-label, 2-part evaluation study which will primarily assess the safety and tolerability of VTS-270 (2-hydroxypropyl beta-cyclodextrin [HP-β-CD]) in pediatric participants with…
- Trial name
- NCT03687476
- Testing
- VTS-270
- Sponsor
- Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
Non-Hodgkin Lymphoma Refractory/ Relapsed, DLBCL - Diffuse Large B Cell Lymphoma
This is a Phase 1 Study to Evaluate the Safety of LTZ-301 in Patients With Non-Hodgkin Lymphoma
This study is a first-in-human (FIH), Phase 1, multicenter, open-label study to determine the safety, tolerability, pharmacokinetics, pharmacodynamics, and evaluate the preliminary anti-tumor activity of LTZ-301 administered as a…
- Conditions
- Non-Hodgkin Lymphoma Refractory/ Relapsed, DLBCL - Diffuse Large B Cell Lymphoma, Mantle Cell Lymphoma (MCL), Follicular Lymphoma ( FL)
- Trial name
- NCT07121946
- Testing
- LTZ-301
- Where
- 6 sites in United States
- Volunteers
- 42 people
- Sponsor
- LTZ Therapeutics, Inc.
Sphingomyelin Lipidosis
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Primary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult…
- Trial name
- ASCEND (NCT02004691)
- Testing
- Olipudase alfa
- Where
- 24 sites in United States, Argentina and 15 more
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Brain and Central Nervous System Tumors, Lymphoma
DX-8951f in Treating Children With Advanced Solid Tumors or Lymphomas
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of DX-8951f in treating children who have advanced solid…
- Conditions
- Brain and Central Nervous System Tumors, Lymphoma, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- NCT00004212
- Testing
- filgrastim, exatecan mesylate
- Where
- 4 sites in United States
- Sponsor
- Daiichi Sankyo