Clinical trials: Phase 1
Acute Myeloid Leukemia
A Phase I-II Study Investigating the All-Oral Combination of the Menin Inhibitor SNDX-5613 With Decitabine/Cedazuridine (ASTX727) and Venetoclax in Acute Myeloid Leukemia (SAVE)
Part 1b of this clinical research study is to find the highest tolerable dose of SNDX-5613 that can be given in combination with ASTX727 (a combination of the drugs decitabine/cedazuridine) and venetoclax for patients with acute…
- Trial name
- NCT05360160
- Testing
- SNDX-5613, Venetoclax, ASTX727
- Where
- United States
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- M.D. Anderson Cancer Center
Pediatric Acute Leukemia
A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients
The goal of Phase 1a of this clinical research study is to find the highest tolerable dose of revumenib that can be given in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin to patients who have acute…
- Trial name
- NCT07052994
- Testing
- Revumenib, Cytrabine, Daunorubicin
- Sponsor
- M.D. Anderson Cancer Center
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
A Phase I open-label, multicenter study, to evaluate the safety, feasibility, and maximum tolerated dose (MTD) of treating children with newly diagnosed DIPG or recurrent neuroblastoma with molecular targeted therapy in…
- Trial name
- PEACH (NCT04837547)
- Testing
- Tumor-specific ex vivo expanded autologous lymphocyte transfer (TTRNA-xALT)
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- University of Florida
Recurrent High-grade Glioma, Grade III Astrocytoma
Peds CHAMP1ON - Hematopoietic Stem Cell And Monoclonal Antibody PD-1 Blockade for RecurreNt Pediatric High-Grade Glioma
This is a Phase I study of ex vivo expanded CD34+ hematopoietic stem cells (exHSCs) plus nivolumab in pediatric patients with histologically confirmed diagnosis of a non-brainstem high-grade glioma (NB-HGG, WHO Grade III or IV…
- Conditions
- Recurrent High-grade Glioma, Grade III Astrocytoma, Oligodendroglioma, Oligoastrocytoma
- Trial name
- Peds CHAMP1ON (NCT07031765)
- Testing
- exHSC, Nivolumab, Resection or biopsy
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
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
Stargardt Disease 1, STGD1
A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 Participants
This is a first-in-human (FIH), multicenter, randomized, double-masked, placebo-controlled adaptive clinical study evaluating the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) of CITY-RBP4, following…
- Conditions
- Stargardt Disease 1, STGD1, Healthy Volunteers
- Trial name
- NCT07836452
- Testing
- CITY-RBP4
- Where
- Australia
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- City Therapeutics
Recurrent Childhood Ewing Sarcoma, Recurrent Childhood Malignant Solid Neoplasm
A Phase 1/2 Study of CBX-12 in Children, Adolescents, or Young Adults With Solid Tumors That Have Come Back After Treatment or Are Difficult to Treat
This phase I/II trial tests the safety, best dose, and effectiveness of CBX-12 in the treatment of pediatric patients (ages 12 months to 30 years) with rhabdomyosarcoma, Ewing sarcoma, or other extracranial solid tumors that have…
- Conditions
- Recurrent Childhood Ewing Sarcoma, Recurrent Childhood Malignant Solid Neoplasm, Recurrent Childhood Rhabdomyosarcoma, Recurrent Ewing Sarcoma
- Trial name
- NCT07830667
- Testing
- Archive Sample Retrieval, Biospecimen Collection, pH Low Insertion Peptide-exatecan Conjugate CBX-12
- Volunteers
- 79 people
- Sponsor
- Children's Oncology Group
Autism Spectrum Disorder, Autism
Adia Med of Winter Park LLC Autism Spectrum Disorder Research Study
This 12-month study tests whether AdiaVita (umbilical cord blood-derived stem cells and exosomes) plus glutathione improves autism symptoms more than glutathione alone in children ages 3-12.
- Conditions
- Autism Spectrum Disorder, Autism, ASD, Autism Spectrum Disorder (ASD)
- Trial name
- NCT07304440
- Testing
- Umbilical Cord Blood-Derived Stem Cells and Exosomes, Glutathione
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Adia Med of Winter Park LLC
Advanced Cancer, Advanced Solid Tumor
SLV-154 Treatment of Advanced Cancers
This is a Phase 1 study comprising a Phase 1a dose-escalation portion and a Phase 1b expansion portion evaluating the safety, pharmacokinetics, pharmacodynamics, immunogenicity, and efficacy of SLV-154 across a range of dose…
- Conditions
- Advanced Cancer, Advanced Solid Tumor, Metastatic Cancer
- Trial name
- NCT06771219
- Testing
- SLV-154
- Where
- 12 sites in United States
- Volunteers
- 250 people
- Sponsor
- Solve Therapeutics
Diarrhoea
Racecadotril Versus Standard Treatment in Decreasing the Duration of Acute Diarrhoea in Children
Acute watery diarrhea is defined as the passage of 3 or more loose or liquid stools per day for 3 or more days but less than 14 days, including patients with mild to moderate dehydration per WHO classification.
- Trial name
- ERADICATE (NCT06529497)
- Testing
- Racecadotril
- Where
- Pakistan
- Volunteers
- 260 people
- Sponsor
- Benazir Bhutto Hospital, Rawalpindi
Astrocytoma, IDH-Mutant, Grade 2, Astrocytoma, IDH-Mutant, Grade 3
Telaglenastat With Radiation Therapy and Temozolomide in Treating Patients With IDH-Mutated Diffuse Astrocytoma or Anaplastic Astrocytoma
This phase 1b trial studies the side effects and best dose of telaglenastat in combination with radiation therapy and temozolomide in treating patients with IDH-mutated diffuse or anaplastic astrocytoma.
- Trial name
- NCT03528642
- Testing
- Questionnaire Administration, Radiation Therapy, Telaglenastat Hydrochloride
- Where
- 25 sites in United States
- Volunteers
- 23 people
- Sponsor
- National Cancer Institute (NCI)
Congenital Myasthenia Syndrome
Evaluating the Safety and Effects of AMP-101 in People With DOK7 Congenital Myasthenia Syndrome
This study is evaluating the safety and potential effects of AMP-101 in people with DOK7 Congenital Myasthenia Syndrome (CMS). Participants will receive a single dose of the study treatment and will be monitored to assess their…
- Trial name
- NCT07830563
- Testing
- AMP-101
- Volunteers
- 4 people
- Sponsor
- Amplo Biotechnology
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
This phase I/II trial studies the side effects and the best dose of selumetinib and how well it works in treating or re-treating young patients with low grade glioma that has come back (recurrent) or does not respond to treatment…
- Conditions
- Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma, Recurrent Neurofibromatosis Type 1, Recurrent Visual Pathway Glioma
- Trial name
- NCT01089101
- Testing
- Biospecimen Collection, Selumetinib
- Where
- 17 sites in United States
- Volunteers
- 217 people
- Sponsor
- National Cancer Institute (NCI)
Lysosomal Diseases, Gangliosidosis
A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme.
- Conditions
- Lysosomal Diseases, Gangliosidosis, GM1
- Trial name
- NCT03952637
- Testing
- AAV9-GLB1, Abdominal ultrasound, Rituximab
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Myotonic Dystrophy
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1
This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 55 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1).
- Trial name
- BrAAVe (NCT06844214)
- Testing
- SAR446268
- Where
- 10 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Sanofi
Mucopolysaccharidosis IIIB
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its…
- Trial name
- NCT07640984
- Testing
- JR-446
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 12 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Acute Myeloid Leukemia
A Multi-Site Break Through Cancer Trial: Phase II Study Investigating Dual Inhibition of BCL2 and Menin in AML MRD Using the Combination of Venetoclax and Revumenib
To learn if the combination of venetoclax and revumenib can help to control MRD-positive AML.
- Trial name
- NCT06284486
- Testing
- Venetoclax, Revumenib
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- M.D. Anderson Cancer Center
Human Metapneumovirus, Human Parainfluenza Virus Type 3
Study of the Infectivity, Safety and Immunogenicity of Two Recombinant, Live-Attenuated, B/HPIV3 Vectored Vaccines Expressing the Fusion Glycoprotein of HMPV Delivered by Nasal Spray to HPIV3-Seropositive Children 24 to <60 Months of Age
HPIV3 and HMPV are viruses that can cause breathing problems in children. The goal of this clinical trial is to look at the safety of 2 experimental HPIV3/HMPV vaccines in HPIV3-seropositive children ≥ 24 months to < 60 months of…
- Trial name
- NCT06546423
- Testing
- B/HPIV3/HMPV-PreF-A vaccine, B/HPIV3/HMPV-F-B365 vaccine
- Where
- 4 sites in United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Wheezing
Genes, Soy Isoflavones, and Virus
In this study, we will assess whether children without established asthma who have a variation in the switch for the PAI-1 gene will have higher PAI-1 levels and remodeling / allergic inflammatory pathways in their airways than…
- Trial name
- GSV (NCT07828405)
- Testing
- Soy Isoflavone Nutritional Supplements
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Edetate Calcium Disodium or Succimer in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome Undergoing Chemotherapy
This phase I trial studies the side effects and best dose of edetate calcium disodium or succimer in treating patients with acute myeloid leukemia or myelodysplastic syndrome undergoing chemotherapy.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome, Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Trial name
- NCT03630991
- Testing
- Edetate Calcium Disodium, Multivitamin, Succimer
- Where
- United States
- Volunteers
- 58 people
- Sponsor
- M.D. Anderson Cancer Center
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