Clinical trials: Rare Cancer
Pediatric Solid Tumor, Pediatric Lymphoma
Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas
The study evaluates CLR 131 in children, adolescents, and young adults with relapsed or refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain tumors for which there are no standard treatment…
- Conditions
- Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG
- Trial name
- CLOVER-2 (NCT03478462)
- Testing
- CLR 131
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cellectar Biosciences, Inc.
Ewing's Sarcoma, Ewing Family of Tumors
Pbi-shRNA™ EWS/FLI1 Type 1 LPX in Subjects With Advanced Ewing's Sarcoma
The purpose of this study is to determine the safety and the maximum tolerated dose of of pbi-shRNA™ EWS/FLI1 Type 1 lipoplex in patients with advanced Ewing's sarcoma.
- Conditions
- Ewing's Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic
- Trial name
- NCT02736565
- Testing
- pbi-shRNA™ EWS/FLI1 Type 1 Lipoplex
- Where
- 2 sites in United States
- Volunteers
- 7 people
- Sponsor
- Gradalis, Inc.
Pheochromocytoma, Paraganglioma
Expanded Access Program of Ultratrace Iobenguane I131 for Malignant Relapsed/Refractory Pheochromocytoma/Paraganglioma
The purpose of this sub-study is to provide expanded access of AZEDRA (Ultratrace Iobenguane I 131) and to evaluate the safety and tolerability of AZEDRA in subjects with iobenguane-avid malignant and/or recurrent…
- Trial name
- NCT02961491
- Testing
- Ultratrace Iobenguane I131
- Where
- 3 sites in United States
- Sponsor
- Molecular Insight Pharmaceuticals, Inc.
Advanced or Recurrent Solid Tumor Harboring an NTRK Gene Fusion
NTRK Gene Fusion - Positive Advanced or Recurrent Solid Tumors, a Rare Cancer Caused by Specific Changes in the Genes
Researchers want to learn more about the use of larotrectinib as a real-world treatment for tropomyosin receptor kinase fusion cancer, also called TRK fusion cancer.
- Trial name
- NCT04945330
- Testing
- Larotrectinib (Vitrakvi, BAY2757556)
- Where
- Japan
- Volunteers
- 100 people
- Sponsor
- Bayer
Ewing Sarcoma, Ewing Family of Tumors
A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide
The goal of this clinical trial was to compare participants with first relapse or refractory Ewing's sarcoma when treated with investigational product (Vigil) in addition to the standard treatment of irinotecan and temozolomide…
- Conditions
- Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic
- Trial name
- VITA (NCT03495921)
- Testing
- Vigil, Irinotecan, Temozolomide
- Where
- 15 sites in United States
- Volunteers
- 32 people
- Sponsor
- Gradalis, Inc.
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Clinical Study To Further Evaluate The Efficacy Of Dabrafenib Plus Trametinib In Patients With Rare BRAF V600E Mutation-Positive Unresectable or Metastatic Solid Tumors
This study is a phase IV, pragmatic single-arm prospective, open label study in pediatric (1 years or older) and adult study participants with rare BRAF V600E mutation-positive unresectable or metastatic solid tumors for whom a…
- Trial name
- NCT05868629
- Testing
- Non-investigational
- Where
- 8 sites in United States
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures