Clinical trials: Phase 1
Solid Tumor Cancer, Medulloblastoma
Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).
Study in children and adolescents of 177Lu DOTATATE (Lutathera®) combined with the PARP inhibitor olaparib for treatment of recurrent or relapsed solid tumours expressing somatostatin receptors (SSTR) (LuPARPed)
- Conditions
- Solid Tumor Cancer, Medulloblastoma, High Risk Neuroblastoma, High Grade Gliomas
- Trial name
- LUPARPED (NCT06607692)
- Testing
- Olaparib; 177Lu-DOTATATE
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Fundación de investigación HM
Lymphoblastic Leukemia
TINI 2: Total Therapy for Infants With Acute Lymphoblastic Leukemia II
The purpose of this study is to improve upon the TINI study treatment. The study will test the ability of a type of immunotherapy called blinatumomab to clear persistent leukemia.
- Trial name
- NCT05848687
- Testing
- Dexamethasone, Mitoxantrone, PEG asparaginase
- Where
- 24 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- Tanja Andrea Gruber
Acute Pancreatitis
Study of CM4620 to Reduce the Severity of Pancreatitis Due to Asparaginase
This is a phase I/II clinical trial assessing the tolerability and efficacy of CM4620 in children and young adults with acute pancreatitis caused by asparaginase.
- Trial name
- NCT04195347
- Testing
- CM4620
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- St. Jude Children's Research Hospital
Non-Tuberculous Mycobacterial (NTM) Pneumonia, Mycobacterium Abscessus Infection
Bacteriophage Therapy for Mycobacterium Abscessus Pulmonary Infection
This study aims to use mycobacteriophage therapy, using identified in-vitro effective Mycobacteriophage Muddy_HRMN0052, along with combination conventional antimycobacterial therapy for their NTM pulmonary disease with…
- Trial name
- NCT07228702
- Testing
- Mycobacteriophage Muddy_HRMN0052, Amikacin Injection, Clofazimine
- Where
- Canada
- Volunteers
- 1 people
- Sponsor
- Vancouver Coastal Health
Glucocorticoid-Refractory aGVHD
A Study of TQ05105 Tablets in Subjects With Glucocorticoid-Refractory Acute Graft-Versus-Host Disease (aGVHD)
This is an open-label,single arm,Phase Ib study,in order to evaluate the safety,tolerability, preliminary efficacy and pharmacokinetics of TQ05105 tablets in subjects with Glucocorticoid-Refractory aGVHD
- Trial name
- NCT04941404
- Testing
- TQ05105 tablets
- Where
- 4 sites in China
- Volunteers
- 13 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Autism Spectrum Disorder, Autism
GAIP ASD Research Study
This clinical research study evaluates the safety and preliminary effects of AdiaVita (umbilical cord blood-derived stem cells and exosomes) combined with glutathione versus glutathione alone in people aged 3 and older with…
- Conditions
- Autism Spectrum Disorder, Autism, ASD, Autism Spectrum Disorder (ASD)
- Trial name
- NCT07622316
- Testing
- Umbilical Cord Blood-Derived Stem Cells and Exosomes, Glutathione
- Where
- 2 sites in United States
- Volunteers
- 100 people
- Sponsor
- Greater Atlanta Integrative Pediatrics
Healthy Volunteers
Study To Evaluate Safety, Tolerability, and Pharmacokinetics of MAM01 in African Population
This study will test a new drug (MAM01) to find which doses are safe and could help prevent people from getting malaria for at least 4 months. The study will take place in parts of Africa where malaria is common.
- Trial name
- NCT06408857
- Testing
- MAM01 300 mg SC, MAM01 300 mg IM, MAM01 2000 mg IV
- Where
- 2 sites in Uganda
- Volunteers
- 125 people, healthy volunteers welcome
- Sponsor
- Gates Medical Research Institute
CD7+ T-ALL/LBL
Single-arm, Open-label, Dose-escalating Phase I Clinical Study of PA3-17 Injection in Children and Adolescents With Relapsed/Refractory T-lymphoblastic Leukemia/Lymphoma
This is a phase I, open-label, dose-escalation clinical trial. The primary objectives are to assess the safety of PA3-17 injection in pediatric and adolescent participants with relapsed/refractory T-lymphoblastic…
- Trial name
- R/R T-ALL/LBL (NCT07623681)
- Testing
- T cell injection targeting CD7 chimeric antigen receptor
- Where
- China
- Volunteers
- 12 people
- Sponsor
- PersonGen BioTherapeutics (Suzhou) Co., Ltd.
Congenital Heart Disease (CHD)
Mesenchymal Stromal Cells for Infants With Congenital Heart Disease (MedCaP)
The proposed study will be a prospective, open-label, single-center, safety and feasibility phase 1 trial of allogeneic bone marrow-derived mesenchymal stromal cell (BM-MSC) delivery though cardiopulmonary bypass (CPB) using a…
- Trial name
- MedCaP (NCT04236479)
- Testing
- BM-MSC
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Catherine Bollard
Medulloblastoma, Glioblastoma Multiforme
A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma
Patients will receive a vaccine called SurVaxM on this study. While vaccines are usually thought of as ways to prevent diseases, vaccines can also be used to treat cancer.
- Conditions
- Medulloblastoma, Glioblastoma Multiforme, Anaplastic Astrocytoma, High-grade Astrocytoma NOS
- Trial name
- NCT04978727
- Testing
- SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥10 and ≤21 years, SurVaxM for patients with relapsed or progressive MB, HGG or ependymoma ages ≥1 and <10 years, SurVaxM for patients with non-relapsed DIPG post radiation-therapy ages ≥1 and ≤21 years
- Where
- 14 sites in United States, Canada
- Volunteers
- 35 people
- Sponsor
- Children's Oncology Group
Hematologic Malignancy
TCRαβ-depleted Progenitor Cell Graft With Early Memory T-cell DLI, Plus Selected Use of Blinatumomab, in naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies
This is a phase I, prospective clinical trial studying the safety and feasibility of providing early memory T-cell DLI. The primary objective is: To assess the safety and feasibility of early CD45RA-depleted DLI administration.
- Trial name
- NCT07052370
- Testing
- Thymoglobulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- St. Jude Children's Research Hospital
Ependymoma
HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children With Ependymoma
This is a Phase I study to evaluate the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells).
- Trial name
- NCT04903080
- Testing
- HER2 Specific CAR T Cell
- Where
- 13 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Children's Oncology Group
Acute Myeloid Leukemia Post Cytotoxic Therapy, Myelodysplastic Syndrome Post Cytotoxic Therapy
Highest Dose of Uproleselan in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, or Mixed Phenotype Acute Leukemia Relapsed or Refractory That Expresses E-selectin Ligand on the Cell Membrane
This phase I trial tests the safety, side effects, and determination of the best dose of uproleselan in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia, myelodysplastic syndrome or…
- Conditions
- Acute Myeloid Leukemia Post Cytotoxic Therapy, Myelodysplastic Syndrome Post Cytotoxic Therapy, Myeloid Leukemia Associated With Down Syndrome, Recurrent Acute Myeloid Leukemia
- Trial name
- NCT05146739
- Testing
- Cytarabine, Fludarabine, Leucovorin
- Where
- 19 sites in United States
- Volunteers
- 8 people
- Sponsor
- National Cancer Institute (NCI)
Antibiotic Resistance Prevention
Assessing the Impact of Unnecessary Antibiotic Treatment on the Development of Appropriate Adaptive Immune Responses in Malnourished Bangladeshi Infants
The investigators are examining the role of circulating T follicular helper (Tfh) cells in the induction of the antibody responses.
- Trial name
- NCT07615842
- Testing
- Azithromycin
- Where
- Bangladesh
- Volunteers
- 150 people
- Sponsor
- University of Virginia
Relapsed Acute Lymphoblastic Leukemia (ALL), Refractory Acute Lymphoblastic Leukemia (ALL)
Phase I Trial of the Selective Inhibitor of Nuclear Export, KPT-330, in Relapsed Childhood ALL and AML
This research study involves participants who have acute lymphoblastic or acute myelogenous leukemia that has relapsed or has become resistant (or refractory) to standard therapies.
- Conditions
- Relapsed Acute Lymphoblastic Leukemia (ALL), Refractory Acute Lymphoblastic Leukemia (ALL), Relapsed Acute Myelogenous Leukemia (AML), Refractory Acute Myelogenous Leukemia (AML)
- Trial name
- NCT02091245
- Testing
- KPT-330
- Where
- 10 sites in United States
- Volunteers
- 16 people
- Sponsor
- Dana-Farber Cancer Institute
Recurrent Acute Lymphoblastic Leukemia, Recurrent B Acute Lymphoblastic Leukemia
Genetically Engineered Cells (Anti-CD19/CD20/CD22 CAR T-cells) for the Treatment of Relapsed or Refractory Lymphoid Malignancies
This phase I trial tests the safety, side effects and best infusion dose of genetically engineered cells called anti-CD19/CD20/CD22 chimeric antigen receptor (CAR) T-cells following a short course of chemotherapy with…
- Conditions
- Recurrent Acute Lymphoblastic Leukemia, Recurrent B Acute Lymphoblastic Leukemia, Recurrent B-Cell Prolymphocytic Leukemia, Recurrent Chronic Lymphocytic Leukemia
- Trial name
- NCT05418088
- Testing
- Anti-CD19/CD20/CD22 CAR T-Cells, Cyclophosphamide, Fludarabine Phosphate
- Where
- 2 sites in United States
- Volunteers
- 54 people
- Sponsor
- Sumithira Vasu
Retinitis Pigmentosa 11, Retinal Degeneration
MAD of IVT VP-001 in PRPF31 Mutation-Associated Retinal Dystrophy Subjects (Wallaby)
A Phase 1 Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of Intravitreally Administered VP-001 in Participants with Confirmed PRPF31 Mutation-Associated Retinal Dystrophy
- Conditions
- Retinitis Pigmentosa 11, Retinal Degeneration, Eye Diseases, Retinal Disease
- Trial name
- NCT06455826
- Testing
- VP-001
- Where
- 5 sites in United States
- Volunteers
- 6 people
- Sponsor
- PYC Therapeutics
CACNA1C
A Phase I/II, First-In-Human Trial to Evaluate the Safety, Tolerability, and Pharmacokinetic Activity to Prevent or Treat Neuropsychiatric Symptoms in Pediatric Subjects With Timothy Syndrome
The goal of this clinical trial is to learn if an antisense oligonucleotide (TS1-ASO) can safely treat and potentially prevent neuropsychiatric and neurodevelopmental symptoms in pediatric participants (age >2 months) with…
- Trial name
- TS1-ASO (NCT07600658)
- Testing
- TS1-ASO
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Stanford University
Severe Sickle Cell Disease, Bone Marrow Failure Syndromes
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
- Conditions
- Severe Sickle Cell Disease, Bone Marrow Failure Syndromes, Metabolic Disorders, Immunologic Disorders
- Trial name
- FAM BMT (NCT03128996)
- Testing
- RIC regimen, GVHD prophylaxis regimen
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Graft-versus-host Disease
Post-Transplant Cyclophosphamide, Bortezomib and Abatacept for the Prevention of Graft-versus-Host-Disease (GvHD)
This is a phase I-II clinical trial. Adult subjects with hematological malignancies undergoing allogeneic HSCT from an HLA matched sibling or ≥7 out of 8 allele level HLA matched unrelated donor are eligible for the study if they…
- Trial name
- NCT05289167
- Testing
- Cyclophosphamide, Abatacept, Bortezomib
- Where
- United States
- Volunteers
- 74 people
- Sponsor
- Northwell Health
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