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Protocol Details

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

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts

Summary

Number

17-H-0091

Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 4
Max Age: 75

Referral Letter Required

Yes

Population Exclusion(s)

Neonates;
Pregnant Women and Fetuses

Special Instructions

Currently Not Provided

Keywords

Haploidentical;
Nonmyeloablative

Recruitment Keyword(s)

None

Condition(s)

Severe Aplastic Anemia;
Hypo-Plastic MDS;
Myelodysplastic Syndrome (MDS)

Investigational Drug(s)

CliniMACS CD34 Reagent
CordIn

Investigational Device(s)

None

Intervention(s)

Biological/Vaccine: CordIn

Supporting Site

National Heart, Lung, and Blood Institute

Background:

Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant. Researchers are testing ways to make stem cell transplant safer and more effective.

Objective:

To test if treating people with SAA or MDS with a co-infusion of blood stem cells from a family member and cord blood stem cells from an unrelated donor is safe and effective.

Eligibility:

Recipients ages 4-55 with SAA or MDS

Donors ages 4-75

Design:

Recipients will be screened with:

-Blood, lung, and heart tests

-Bone marrow biopsy

-CT scan

Recipients will have an IV line placed into a vein in the neck. Starting 11 days before the transplant they will have several chemotherapy infusions and 1 30-minute radiation dose.

Recipients will get the donor cells through the IV line. They will stay in the hospital 3-4 weeks. After discharge, they will have visits:

-First 3-4 months: 1-2 times weekly

-Then every 6 months for 5 years<TAB>

Donors will be screened with:

-Physical exam

-Medical history

-Blood tests

Donors veins will be checked for suitability for stem cell collection. They may need an IV line to be placed in a thigh vein.

Donors will get filgrastim injections daily for 5-7 days. On the last day, they will have apheresis: Blood drawn from one arm or leg runs through a machine and into the other arm or leg. This may be repeated 2 days or 2-4 weeks later.

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Eligibility

INCLUSION CRITERIA - RECIPIENT:

1. Diagnosed with severe aplastic anemia with bone marrow cellularity <30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or neutropenia (absolute neutrophil count <=1000 cells/ uL or for patients receiving granulocyte transfusions, absolute neutrophil count <=1000 cells/ uL before beginning granulocyte transfusions).

OR

History of severe aplastic anemia transformed to MDS that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) < 5% myeloblasts and < 30% of cellularity in the bone marrow on screening morphologic analysis.

2. Intolerance of or failure to respond to standard immunosuppressive therapy.

3. Identification of either a) at least one alternative donor (i.e. HLA- haploidentical related donor (i.e. greater than or equal to 5/10 HLA match: HLA-A, B, C, DR, and DQ loci) or greater than or equal to 9/10 HLA matched unrelated donor) who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the CordIn unit has been rejected or b) umbilical cord blood unit/s that can be used for a salvage cord blood transplant in the event that the CordIn unit has been rejected.

4. Availability of at least one greater than or equal to 4/8 HLA-matched (HLA-A, B, C, and DR loci) cord blood unit from the National Marrow Donor Program (NMDP).

5. The cord blood unit must contain a minimum TNC of at least 1.8 x 10^9 and at least 1.8x10^7/kg TNC and at least 8 x 10^6 CD34+ cells (all doses prior to thawing). 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.

6. Ages 4-55 years inclusive.

7. Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.

EXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):

1. Availability of an HLA identical or 9/10 HLA matched (HLA A, B, C, DR, and DQ loci) - relative to serve as a stem cell donor.

2. The patient is deemed to be a candidate for a 10/10 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).

3. ECOG performance status of 2 or more.

4. Major anticipated illness or organ failure incompatible with survival from transplant.

5. 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.

6. HIV positive.

7. Diagnosis of Fanconi s anemia (by chromosome breakage study)

8. Diffusion capacity of carbon monoxide (DLCO) <40% predicted 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 thus are unable to have their DLCO assessed).

9. Left ventricular ejection fraction < 40% (evaluated by ECHO).

10. Transaminases > 5x upper limit of normal.

11. Serum bilirubin >4 mg/dl.

12. Creatinine clearance < 50 cc/min/BSAm^2 by 24-hour urine collection adjusted by body surface area..

13. Serum creatinine > 2.5 mg/dl

14. Presence of an active infection not adequately responding to appropriate therapy.

15. History of a malignant disease liable to relapse or progress within 5 years.

16. Allergy to bovine, Gentamicin, or to any product which may interfere with the treatment.

17. Presence of donor-specific antibodies (DSA) to the umbilical cord blood unit and for cohort 1, to the haplo-identical donor.


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Citations:

Not Provided

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Contacts:

Principal Investigator

Referral Contact

For more information:

Richard W. Childs, M.D.
National Heart, Lung and Blood Institute (NHLBI)



Tatyana Worthy, R.N.
National Heart, Lung and Blood Institute (NHLBI)
National Institutes of Health
Building 10
Room 3-3485
10 Center Drive
Bethesda, Maryland 20892
(301) 827-1376
worthyt@mail.nih.gov

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: 1-866-411-1010
PRPL@cc.nih.gov

Clinical Trials Number:

NCT03173937

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