Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
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| ClinicalTrials.gov Identifier: NCT03226691 |
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Recruitment Status :
Completed
First Posted : July 24, 2017
Results First Posted : April 20, 2020
Last Update Posted : April 20, 2020
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The constitution of blood relies upon hematopoietic stem cells (HSCs), which stay in the bone marrow and differentiate to all lineages of peripheral blood cells. HSC transplantation is the only curative option currently available for sickle cell disease (SCD) patients either via allogeneic HSC transplantation or HSC-targeted gene therapy. Granulocyte-colony stimulating factor (G-CSF)- mobilized HSCs are frequently utilized in the adult setting of HSC transplantation because of the faster hematologic recovery as compared to bone marrow. As an autologous HSC source for gene therapy, bone marrow harvest has been generally employed since G-CSF has been prohibitive in SCD patients due to granulocyte stimulation and the associated reports of vaso-occlusive crises, multi-organ failure, and death. However, when bone marrow harvest is used, the amounts of collected cells are limited and anesthesia is required. In order to obtain HSCs in large numbers without anesthesia, patients will undergo mobilization followed by large volume apheresis. Plerixafor is an alternative treatment for mobilization without direct stimulation to granulocytes, and it is theoretically applicable for SCD patients. The primary endpoint of this study is to obtain sufficient amounts of HSCs collected from the peripheral blood in SCD patients after plerixafor
mobilization with an acceptable safety profile. The harvested products will be stored as backup for patients undergoing gene therapy as well as allogeneic HSC transplantation.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Sickle Cell Disease | Drug: Plerixafor | Phase 1 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 15 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Supportive Care |
| Official Title: | Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients Using Plerixafor |
| Actual Study Start Date : | July 25, 2017 |
| Actual Primary Completion Date : | February 27, 2019 |
| Actual Study Completion Date : | February 27, 2019 |
| Arm | Intervention/treatment |
|---|---|
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Experimental: Single Cohort - Plerixafor
Plerixafor at a single dose of 240 microgram/kg
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Drug: Plerixafor
Single-dose subcutaneous administration of plerixafor (Mozobil®) at 240 μg/kg
Other Name: Mozobil® |
- Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events [ Time Frame: 1 day ]Sufficient collection of HSCs (target 2.0x106 CD34+ cells/kg) from the PB after plerixafor mobilization without serious adverse events (SAEs)
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
- INCLUSION CRITERIA:
- SCD patients who are 18 or older, and (a) planned to enroll in an active allogeneic HSCT study where back-up autologous HSCs are needed; OR (b) are eligible for an allogeneic HSCT study (i.e. have the same disease severity as group (a), but no active allogeneic HSCT study is available), and are willing to donate autologous HSCs for a future gene therapy, gene editing, or allogeneic HSCT study.
- Adequate renal function: serum/plasma creatinine <1.5 mg/dL.
- Adequate liver function: direct bilirubin and ALT <5 times the upper limit of normal range.
- Blood counts: WBC >3,000/mm^3, granulocytes >1,000/mm^3, hemoglobin>7.0g/dL, platelets>150,000/mm^3.
- Female patients of childbearing age should have a negative serum pregnancy test within one week of beginning plerixafor administration, have had a hysterectomy, post-menopausal, or absence of a menses for over a year.
- Meets NIH Department of Transfusion Medicine (DTM) eligibility criteria for blood component donation for in vitro research use (negative serologic tests for syphilis, hepatitis B and C, HIV, and HTLV-1).
- Ability to give informed consent to participate in the protocol.
- Female and male individuals of reproductive potential must agree to one of the contraceptive regimens stated above if sexually active
EXCLUSION CRITERIA:
- Pregnancy. Female patients of childbearing age should have a negative serum pregnancy test within one week of beginning plerixafor administration, except those that have had a hysterectomy, post-menopausal, or an absence of a menses for over a year.
- Active viral, bacterial, fungal, or parasitic infection.
- History of cancer, excluding squamous carcinoma of the skin and cervical carcinoma in situ.
- Active and painful splenomegaly or splenomegaly (size greater than upper limit of normal) determined by ultrasound.
- Allergy to plerixafor.
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): NCT03226691
| United States, Maryland | |
| National Institutes of Health Clinical Center | |
| Bethesda, Maryland, United States, 20892 | |
| Principal Investigator: | John F Tisdale, M.D. | National Heart, Lung, and Blood Institute (NHLBI) |
Documents provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | National Heart, Lung, and Blood Institute (NHLBI) |
| ClinicalTrials.gov Identifier: | NCT03226691 |
| Other Study ID Numbers: |
170124 17-H-0124 |
| First Posted: | July 24, 2017 Key Record Dates |
| Results First Posted: | April 20, 2020 |
| Last Update Posted: | April 20, 2020 |
| Last Verified: | June 6, 2019 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
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Plerixafor Leukapheresis Mobilization Stem Cells Sickle Cell Disease |
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Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies |
Genetic Diseases, Inborn Plerixafor Anti-HIV Agents Anti-Retroviral Agents Antiviral Agents Anti-Infective Agents |