Clinical Trials Logo

Clinical Trial Summary

The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment, particularly in pediatric populations. Clinical studies in adults with SCD have focused on interventions that prolong survival and improve the quality of life. Unlike children, adults with SCD are much more likely to have a debilitating complication. As a result, the risk/benefit ratio of HCT is very favorable in adults, particularly if an approach to HCT that defines an acceptable level of toxicity can be established. Whereas hematopoietic stem cell transplantation (HSCT) remains the only curative treatment currently available for patients with SCD, the morbidity, the frequent irreversible damage in target organs and the mortality reported in the natural course of patients with severe SCD are strong incentives to perform HSCTs in younger age groups. For those who lack a matched related donor, CB transplant is an appealing option, but despite been less problematic, CB accessibility related to cell dose of appropriately matched cord blood unit (CBU) remains a significant issue. Through a 7-day culture process of a CBU's hematopoietic stem cell HSCs with the UM171 compound, the total cell dose is increased mitigating this limitation. UM171-CB expansion (ECT-001-CB) allows a greater CB accessibility, the selection of better matched cords that might translate into favourable clinical outcomes as reported in previous trials, including a lower risk of graft-versus-host disease. After CB selection and ex-vivo expansion, ECT-001-CB transplant will follow a myeloablative reduced-toxicity conditioning regimen consisting of rATG, busulfan and fludarabine with doses of all agents optimized to the individual using model-based dosing and will be followed by standard supportive care and GVHD prophylaxis consisting of tacrolimus and MMF.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT04594031
Study type Interventional
Source ExCellThera inc.
Contact
Status Withdrawn
Phase Phase 1
Start date July 13, 2021
Completion date February 2, 2024

See also
  Status Clinical Trial Phase
Completed NCT02227472 - Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Recruiting NCT06301893 - Uganda Sickle Surveillance Study (US-3)
Recruiting NCT04398628 - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed NCT02522104 - Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) Phase 4
Recruiting NCT04688411 - An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease N/A
Terminated NCT03615924 - Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease Phase 3
Not yet recruiting NCT06300723 - Clinical Study of BRL-101 in Severe SCD N/A
Recruiting NCT03937817 - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Completed NCT04134299 - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease N/A
Completed NCT04917783 - Health Literacy - Neurocognitive Screening in Pediatric SCD N/A
Completed NCT02580565 - Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
Recruiting NCT04754711 - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition N/A
Completed NCT04388241 - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD N/A
Recruiting NCT05431088 - A Phase 2/3 Study in Adult and Pediatric Participants With SCD Phase 2/Phase 3
Completed NCT01158794 - Genes Influencing Iron Overload State
Recruiting NCT03027258 - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome N/A
Withdrawn NCT02960503 - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease Phase 1/Phase 2
Not yet recruiting NCT02525107 - Prevention of Vaso-occlusive Painful Crisis by Using Omega-3 Fatty Acid Supplements Phase 3
Completed NCT02567695 - A Single-Dose Relative Bioavailability Study Of GBT440 300 mg Capsules in Healthy Subjects Phase 1
Withdrawn NCT02630394 - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease Phase 1