A Trial of a Single ProHema-CB Product Transplant in Pediatric Patients With Inherited Metabolic Disorders (PROVIDE)
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|ClinicalTrials.gov Identifier: NCT02354443|
Recruitment Status : Terminated (Business decision)
First Posted : February 3, 2015
Results First Posted : October 10, 2018
Last Update Posted : October 10, 2018
|Condition or disease||Intervention/treatment||Phase|
|Metabolic Disorders||Biological: ProHema-CB Transplant||Phase 1|
This study is an open-label trial of the safety of a single cord blood transplant using ProHema-CB following busulfan/cyclophosphamide/ATG conditioning for pediatric patients with inherited metabolic disorders.
A maximum of 12 eligible male and female subjects (1 to 18 years old, inclusive) will be enrolled and treated in the trial at approximately 1 to 3 centers within the U.S.
All subjects will be admitted to the hospital, per institutional practice and will receive a conditioning regimen, after which they will receive a HLA-matched or partially matched ProHema -CB unit on Day 0.
They will receive study follow up assessments weekly following Day 0 through Day 100 and study visit Days 180, 270, 365 and 730.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||1 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 1 Trial of a Single ProHema® CB Product as Part of Single Cord Blood Unit Transplant After Busulfan/Cyclophosphamide/ATG Conditioning for Pediatric Patients With Inherited Metabolic Disorders|
|Study Start Date :||June 2015|
|Actual Primary Completion Date :||December 2016|
|Actual Study Completion Date :||February 2017|
ProHema-CB represents Ex Vivo Modulated Human Cord Blood Cells. Each subject will receive one administration of ProHema-CB unit transplant.
Biological: ProHema-CB Transplant
ProHema-CB, the cellular product, represents the cell populations contained within a human UCB unit after modulation on the day of transplantation by an ex vivo incubation process with the prostaglandin derivative, 16,16-dimethyl prostaglandin E2 (also referred to as FT1050). The cell populations include hematopoietic stem and progenitor cells.
- Safety Profile, Assessed Primarily by Neutrophil Engraftment [ Time Frame: Engraftment by Day 42 following study transplant procedure ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02354443
|United States, Massachusetts|
|Boston Children's Hospital|
|Boston, Massachusetts, United States, 02115-5450|
|United States, North Carolina|
|Duke University Medical Center|
|Durham, North Carolina, United States, 27705|
|Study Director:||Chris Storgard, MD||Fate Therapeutics|