Clinical Trials Logo

Clinical Trial Summary

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes.


Clinical Trial Description

This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell transplant (HSCT) in patients with acquired and inherited bone marrow failure (BMF) syndromes. Previously established, disease-specific transplant preparative regimens will be administered based on the specific underlying BMF condition. Mobilized PBSC will be processed using the CliniMACS system for TCR alpha/beta+ T cell depletion plus cluster of differentiation 19+ (CD19+) B cell depletion. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one year overall and event-free survival. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03047746
Study type Interventional
Source Children's Hospital of Philadelphia
Contact Patricia Hankins, BSN, RN, CCRC
Phone 215-590-5168
Email hankinsp@chop.edu
Status Recruiting
Phase N/A
Start date February 1, 2017
Completion date March 2026

See also
  Status Clinical Trial Phase
Recruiting NCT04432584 - A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors Phase 3
Completed NCT05828485 - Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects Phase 1
Recruiting NCT02179359 - Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies N/A
Active, not recruiting NCT04434092 - A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. Phase 3
Terminated NCT05131204 - Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Phase 3
Recruiting NCT01374360 - Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
Active, not recruiting NCT05389449 - A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH Phase 3
Recruiting NCT06100900 - Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Phase 1
Completed NCT01272817 - Nonmyeloablative Allogeneic Transplant N/A
Completed NCT06326814 - A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults Phase 1
Completed NCT04463056 - Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH Phase 3
Recruiting NCT05476887 - To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 Phase 2
Completed NCT01192399 - Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients Phase 2
Active, not recruiting NCT06051357 - Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria Phase 2
Recruiting NCT06154512 - A Real-world, Multi-center, Prospective, Observational Study for PNH in China
Completed NCT04128943 - Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Active, not recruiting NCT03329365 - Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
Recruiting NCT05755867 - Global PNH Patient Registry
Completed NCT04679103 - A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) Phase 3
Completed NCT05642585 - A Study of Single-dose MY008211A in Healthy Adults Phase 1