Clinical trials: Mixed Connective Tissue Disease
- Condition Mixed Connective Tissue Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Glioblastoma, Squamous Cell Carcinoma
Expanded Access to Immunomodulatory AVM0703 for Solid Tumor and Blood Cancer Patients
AVM Biotechnology, Inc., provides immunomodulatory AVM0703 to solid tumor and blood cancer patients upon request by a US licensed MD or DO.
- Conditions
- Glioblastoma, Squamous Cell Carcinoma, Hodgkin Lymphoma, Non-hodgkin Lymphoma
- Trial name
- NCT05974410
- Testing
- AVM0703, Hydrocortisone, Proton pump inhibitor
- Sponsor
- AVM Biotechnology Inc
Solid Tumors, CNS Tumors
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain…
- Trial name
- STARTRK-NG (NCT02650401)
- Testing
- Entrectinib
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Neuroblastoma, Rhabdomyosarcoma
Enoblituzumab (MGA271) in Children With B7-H3-expressing Solid Tumors
This study is a Phase 1, open-label, dose escalation and cohort expansion trial designed to characterize the safety, tolerability, PK, PD, immunogenicity and preliminary antitumor activity of enoblituzumab administered IV on a…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Osteosarcoma, Ewing Sarcoma
- Trial name
- NCT02982941
- Testing
- Enoblituzumab
- Where
- 6 sites in United States
- Volunteers
- 25 people
- Sponsor
- MacroGenics
Neuroblastoma, Ewing's Sarcoma
Use of Racotumomab in Patients With Pediatric Tumors Expressing N-glycolylated Gangliosides
This study will be carried out in children with diagnosis of cancer with tumors known to express N-glycolylated gangliosides. The disease must be resistant to conventional therapy.
- Conditions
- Neuroblastoma, Ewing's Sarcoma, Wilm's Tumor, Retinoblastoma
- Trial name
- NCT01598454
- Testing
- racotumomab
- Where
- Argentina
- Volunteers
- 15 people
- Sponsor
- Laboratorio Elea Phoenix S.A.
Solid Tumors, Neuroblastoma
Flavored, Oral Irinotecan VAL-413 (Orotecan®) Given With Temozolomide for Treatment of Recurrent Pediatric Solid Tumors
A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally administered irinotecan VAL-413 (Orotecan®) given with temozolomide for treatment of recurrent pediatric solid tumors including but not…
- Conditions
- Solid Tumors, Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma
- Trial name
- NCT04337177
- Testing
- VAL-413, Temozolomide
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Valent Technologies, LLC
Ewing Sarcoma, Solid Tumors
Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors
A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan or topotecan with cyclophosphamide) in children, adolescents and young adults…
- Conditions
- Ewing Sarcoma, Solid Tumors, Rhabdoid Tumor, Rhabdomyosarcoma
- Trial name
- NCT03709680
- Testing
- Palbociclib, Temozolomide, Irinotecan
- Where
- 84 sites in United States, Brazil and 11 more
- Volunteers
- 128 people
- Sponsor
- Pfizer
Melanoma, Hodgkin Lymphoma
A Study of Cobolimab Plus Dostarlimab in Pediatric and Young Adult Participants With Cancer
The goal of this interventional study is to determine the strength of cobolimab and dostarlimab that is most tolerated in children and young adults who have advanced solid tumors.
- Conditions
- Melanoma, Hodgkin Lymphoma, High and Low Grade Glioma, Glioblastoma Multiforme (GBM)
- Trial name
- NCT06521567
- Testing
- Cobolimab, Dostarlimab
- Where
- 20 sites in United States, Czechia and 4 more
- Volunteers
- 83 people
- Sponsor
- GlaxoSmithKline
Acne Vulgaris, Acne Vulgaris Superficial Mixed Comedonal and Inflammatory
Full Spectrum Hemp Observational Study
In westernized societies, acne vulgaris is nearly a universal skin disease afflicting 79% to 95% of the adolescent population; older than 25 years - 40% to 54% have facial blemishes and persists into middle age in 12% of women…
- Trial name
- NCT04730414
- Testing
- Full-spectrum hemp (0.018% THC)
- Volunteers
- 50 people
- Sponsor
- Charlotte's Web, Inc
Raynaud's Disease
Safety and Efficacy of Pletal (Cilostazol) for the Treatment of Juvenile Primary and Secondary Raynaud's Phenomenon
Juvenile secondary Raynaud's (ray-knows) Phenomenon is a disorder of the blood vessels in the fingers and sometimes can affect the toes, nose, or ears.
- Trial name
- NCT00048776
- Testing
- Pletal
- Where
- 19 sites in United States
- Volunteers
- 75 people
- Sponsor
- Otsuka America Pharmaceutical
Medulloblastoma, Rhabdomyosarcoma
A Phase I Dose Finding and Safety Study of Oral LDE225 in Children and a Phase II Portion to Assess Preliminary Efficacy in Recurrent or Refractory MB
Phase I dose-escalation study to characterize the safety, tolerability, pharmacokinetics and pharmacodynamics of LDE225 given orally on a daily dosing schedule in children with recurrent or refractory medulloblastoma, or other…
- Conditions
- Medulloblastoma, Rhabdomyosarcoma, Neuroblastoma, Hepatoblastoma
- Trial name
- NCT01125800
- Testing
- LDE225
- Where
- 14 sites in United States, Australia and 4 more
- Volunteers
- 76 people
- Sponsor
- Novartis Pharmaceuticals
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.
Sarcoma, Neuroblastoma
To Evaluate the Safety, Activity and Pharmacokinetics of Marqibo in Children and Adolescents With Refractory Cancer
Background: Marqibo(Registered Trademark) is a new anticancer drug. It combines Vincristine sulfate, which is a widely used anticancer drug, and packages it into a tiny fat bubble known as a liposome.
- Conditions
- Sarcoma, Neuroblastoma, Wilms Tumor, Leukemia
- Trial name
- NCT01222780
- Testing
- Marqibo
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Acrotech Biopharma Inc.
Brain and Central Nervous System Tumors, Neuroblastoma
Radiolabeled MAB Therapy in Patients With Refractory, Recurrent, or Advanced CNS or Leptomeningeal Cancer
The purpose of this study is to test the feasibility and toxicity of administering intrathecal immunotherapy for patients with central nervous system/leptomeningeal (CNS/LM) malignancies.
- Conditions
- Brain and Central Nervous System Tumors, Neuroblastoma, Sarcoma
- Trial name
- NCT00089245
- Testing
- Iodine I 131 MOAB 8H9
- Where
- United States
- Volunteers
- 177 people
- Sponsor
- Y-mAbs Therapeutics
Relapsed Solid Tumor, Refractory Solid Tumor
A Study of Abemaciclib (LY2835219) in Combination With Other Anti-Cancer Treatments in Children and Young Adult Participants With Solid Tumors, Including Neuroblastoma
The study's purpose is to see if the drug, abemaciclib, is safe and effective when given with other drugs to kill cancer cells. The study is open to children and young adults with solid tumors, including neuroblastoma, that did…
- Trial name
- NCT04238819
- Testing
- Abemaciclib, Irinotecan, Temozolomide
- Where
- 25 sites in United States, Australia and 6 more
- Volunteers
- 47 people
- Sponsor
- Eli Lilly and Company
Pediatric Oncology
A Phase I Dose Finding Study in Children With Solid Tumors Recurrent or Refractory to Standard Therapy
Dose escalation phase of the study : To define the safety profile, maximum tolerated dose (MTD) and recommended phase II dose (RP2D) of regorafenib administered orally as a single agent in a 3-weeks-on/1- week-off schedule in…
- Trial name
- NCT02085148
- Testing
- Regorafenib (BAY73-4506), Vincristine (Cellcristin®), Irinotecan (Irinotecan Cell pharm®)
- Where
- 12 sites in France, Italy and 2 more
- Volunteers
- 62 people
- Sponsor
- Bayer
Venous Leg Ulcer
Efficacy and Safety of URGOBD001 Compression System
Evaluation of the efficacy (wound epithelialization and time to closure) and safety (emergence and nature of adverse event) of the new URGO BD001 compression system versus a reference compression in the local treatment of venous…
- Trial name
- FUTURE (NCT05158764)
- Testing
- Venous compression system of the lower limbs stage C6 / C6r of the CEAP classification
- Where
- France
- Volunteers
- 178 people
- Sponsor
- Laboratoires URGO
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
Neuroblastoma, Rhabdomyosarcoma
Safety Study of Recombinant Vaccinia Virus to Treat Refractory Solid Tumors in Pediatric Patients
This is a Phase I, open-label, dose-escalation trial of JX-594 (Pexa-Vec) in pediatric patients with advanced/metastatic, unresectable solid tumors refractory to standard therapy and/or the patient does not tolerate standard…
- Conditions
- Neuroblastoma, Rhabdomyosarcoma, Lymphoma, Wilm's Tumor
- Trial name
- NCT01169584
- Testing
- Recombinant Vaccinia GM-CSF; RAC VAC GM-CSF (JX-594)
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Jennerex Biotherapeutics