Clinical Trials
96
3 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1998
Active, not recruiting
1
1.0%
Completed
31
32.3%
Enrolling By Invitation
1
1.0%
Not yet recruiting
1
1.0%
Recruiting
3
3.1%
Terminated
49
51.0%
Unknown
2
2.1%
Withdrawn
8
8.3%
No approval data available
- Diffuse intrinsic pontine glioma (DIPG) remains an incurable pediatric brainstem tumor, with radiation providing only temporary symptom relief before the tumor regrows, typically within six to eight months. - CAR T-cell therapy has emerged as the most promising experimental approach in over 50 years, with early studies showing tumor shrinkage without radiation and a small number of patients experiencing longer survival. - Rally Foundation for Childhood Cancer Research is funding 13 active DMG projects spanning immunotherapy, tumor biology, blood-brain barrier delivery, and liquid biopsy monitoring. - Researchers emphasize that no cure yet exists, and families must navigate the difficult space between therapeutic promise and scientific proof.
- Iterion Therapeutics has dosed the first patient in a clinical study evaluating tegavivint, a first-in-class Wnt/β-catenin pathway inhibitor, combined with gemcitabine for relapsed or refractory osteosarcoma. - Tegavivint is a small-molecule inhibitor of TBL1 that has already demonstrated favorable tolerability and encouraging monotherapy clinical responses in hepatocellular carcinoma and desmoid tumors. - The trial targets osteosarcoma, the most common malignant bone tumor in children and adolescents, where Wnt/β-catenin signaling is highly active in relapsed and metastatic disease. - The drug has received both Orphan Drug Designation and Pediatric Rare Disease Designation from the FDA for osteosarcoma treatment.
- Thiogenesis Therapeutics announced a new investigator-initiated study collaboration with Dr. Larry Greenbaum at Emory University to evaluate TTI-0102, a next-generation cysteamine-based prodrug for nephropathic cystinosis. - TTI-0102 demonstrates sustained 24-hour cysteamine exposure with potential once-daily dosing and achieves target exposure at approximately half the daily dose of existing therapies. - The company is preparing for Phase 3 development using a non-inferiority design against standard-of-care cysteamine therapy under FDA's 505(b)(2) regulatory pathway. - Nephropathic cystinosis affects an estimated 2,000-2,500 patients worldwide, representing a global market opportunity of over $300 million.
- Moleculin's WP1066 demonstrated safety and anti-tumor immune responses in a Phase 1 trial treating 10 children with recurrent malignant brain tumors at Children's Healthcare of Atlanta. - The study showed partial tumor response in a diffuse intrinsic pontine glioma patient and suppressed STAT3 expression without significant toxicity. - Results published in Journal of Clinical Investigation Insight support advancing to Phase 2 trials for these devastating pediatric cancers with 9-11 month survival rates. - WP1066 targets multiple oncogenic transcription factors including p-STAT3, c-Myc, and HIF-1α while stimulating immune responses against tumors.
- Secretome Therapeutics has initiated a Phase 1 dose-escalating trial of STM-01, a neonatal cardiac progenitor cell therapy, for treating heart failure in young adults with dilated cardiomyopathy. - The trial is funded by The Marcus Foundation and will take place at Children's Healthcare of Atlanta, with a subsequent pediatric DCM study planned if safety is demonstrated. - Pediatric DCM affects 500-1,000 children in the US annually, with diagnosis typically at 2 years of age and a 5-year life expectancy without heart transplantation. - Preclinical studies show STM-01 produces marked improvements in cardiac function and is uniquely potent at improving ejection fraction compared to mesenchymal stem cells from older donors.
- Peach Bowl, Inc. donated $2.5 million to the Aflac Cancer and Blood Disorders Center, expanding support for 18 promising pediatric cancer clinical trials across seven facilities nationwide. - The donation builds on the original $20 million Peach Bowl LegACy Fund established in 2019, with 79 children currently enrolled in various cancer drug trials. - Four new trials launched in the latest funding cycle include studies of tumor-suppressing drugs, immunotherapy for osteosarcoma, brain tumor treatments, and prevention of transplant complications. - The fund was created in honor of Anna Charles Hollis, who died from acute myeloid leukemia in 2018, and aims to accelerate novel cancer treatments from bench to bedside.
