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New treatment option for Severe Aplastic Anemia
Official title Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
ClinicalTrials.gov ID: NCT03173937
What this study is testing
What is Omidubicel (former CordIn)?
Omidubicel (former CordIn) is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for severe aplastic anemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 75
You may be able to join if
- - RECIPIENT:
- Diagnosed with severe aplastic anemia with bone marrow cellularity \ =5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or \>=9/10 HLA matched unrelated...
- the pre-expansion CD34/kg recipient cell number is at least 1.5 x 10\^5 /kg AND
- approval for use of this cord unit for expansion is granted by Gamida Cell.
- the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.
You likely can't join if
- RECIPIENT (ANY OF THE FOLLOWING):
- Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft...
- ECOG performance status of 2 or more.
- Major anticipated illness or organ failure incompatible with survival from transplant.
- Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from...
- HIV positive.
See the full eligibility criteria
- - RECIPIENT:
- Diagnosed with severe aplastic anemia with bone marrow cellularity \ =5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or \>=9/10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage...
- the pre-expansion CD34/kg recipient cell number is at least 1.5 x 10\^5 /kg AND
- approval for use of this cord unit for expansion is granted by Gamida Cell.
- the final cell counts on the cultured and non-cultured fractions meet the IND specified minimal release criteria.
- The CBU will have undergone volume reduction (both plasma and red blood cell depletion) prior to cryopreservation. All CBUs should be procured from public banks that meet local applicable regulations.
- Ages 4-60 years inclusive.
- Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to people aged 4-17 years with formal consent being obtained from parents or legal guardian.
- RECIPIENT (ANY OF THE FOLLOWING):
- Availability of an HLA identical (12/12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral...
- ECOG performance status of 2 or more.
- Major anticipated illness or organ failure incompatible with survival from transplant.
- Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.
- HIV positive.
- Diagnosis of Fanconi s anemia (by chromosome breakage study).
- Diffusion capacity of carbon monoxide (DLCO) \<40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and...
- Left ventricular ejection fraction \< 40% (evaluated by ECHO).
- Transaminases \> 5x upper limit of normal.
- Serum bilirubin \>4 mg/dl.
- Creatinine clearance \< 50 cc/min/BSAm2 by 24-hour urine collection adjusted by body surface area.
- Serum creatinine \> 2.5 mg/dl
- Presence of an active infection not adequately responding to appropriate therapy.
- History of a malignant disease liable to relapse or progress within 5 years.
- Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.
- Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.