Severe Combined ImmunodeficiencyNCT06731036
Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
A study testing CD34+ cell enriched stem cell graft in people with Severe Combined Immunodeficiency and Fanconi Anemia, run by University of Florida.
Trial summary
Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative disorders. Read more about this study
- Who is it for?
- Ages 1 month and older
- Conditions the study is for
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita
- What is being tested?
- CD34+ cell enriched stem cell graft
- How many people
- Not given
- Where
- No sites listed yet
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 month and older
- All sexes
- Child, Adult, Older adult
Who may be able to join
- Patients over 1 month of age
- The following category of patients is eligible for this protocol:
- Patients undergoing allogeneic transplantation for the following indications:
- Non-malignant disorders where graft versus host disease is detrimental:
- Severe combined immune deficiency
- Fanconi Anemia
Show all 18Show fewer
- Dyskeratosis Congenita
- Sickle Cell Disease
- or
- Patients undergoing autologous transplantation intended for hematopoietic/immunologic reconstitution
- or
- Patients with poor graft function defined as persistent cytopenia following stem cell transplant without evidence of recurrent disease
- Subjects must not have more than one active malignancy at the time of enrollment (Subjects with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen [as determined by the treating physician and approved by the PI] may be included).
- Written informed consent obtained from the subject and the subject agrees to comply with all the study-related procedures.
- Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy throughout participation at least 1 year after the stem cell infusion to minimize the risk of pregnancy. Prior to protocol enrollment, women of childbearing potential must be advised of the importance of avoiding pregnancy during trial participation and the potential risk factors for an unintentional pregnancy. WOCBP includes any woman who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or who is not post-menopausal. Post-menopause is defined as:
- Amenorrhea that has lasted for ≥ 12 consecutive months without another cause, or
- For women with irregular menstrual periods who are taking hormone replacement therapy (HRT), a documented serum follicle-stimulating hormone (FSH) level of greater than 35 mIU/mL.
- Males with female partners of child-bearing potential must agree to use physician-approved contraceptive methods (e.g., abstinence, condoms, vasectomy) throughout participation and should avoid conceiving children for 1 year following infusion of the stem cells.
Who cannot join
- Patients with a fully HLA matched sibling donor
- Patients with active graft versus host disease
- Patient with uncontrollable transplant associated thrombotic microangiopathy
- Patient not cleared by transplant provider to process
- Donor unable to donate peripheral blood stem cells
- Females or males of childbearing potential who are unwilling or unable to use an acceptable method to avoid pregnancy for the entire participation period and for at least 1 year after stem cell infusion.
- Females who are known to be pregnant or breastfeeding.
- Prisoners or subjects who are involuntarily incarcerated, or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness.
Trial locations and contacts
No sites are listed yet.
About this study
Official title: A Compassionate Release Protocol: Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative disorders. To mitigate graft versus host disease, graft manipulation has been taking place with CD34+ selection to decrease T-cells entering into the patient, thus lowering the risk of GVHD.
Historically CD34+ manipulation has been performed under a humanitarian use device by utilizing the Miltenyi CliniMACs CD34 Reagent System. This was used for patients with AML in first remission. This approach has additionally been used for patients with sickle cell disease, immune deficiencies, and poor graft function with excellent efficiency. The purpose of this protocol is to create expanded access of CD34+ manipulation for various underlying diseases utilizing the Miltenyi CliniMACS Prodigy® device.
- Conditions
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita, Sickle Cell Disease
- Being tested
- CD34+ cell enriched stem cell graft
- Keywords
- stem cell transplantation, graft manipulation
- Medical subject headings
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD34+ cell enriched stem cell graftBiological
Patients will receive a peripheral blood stem cell graft that has been CD34+ cell enriched using the Miltenyi CliniMACS Prodigy® system.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Combined ImmunodeficiencyStudies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jordan Milner, MD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06731036
- Sponsor's protocol ID
- UF-PEDS036
- Other IDs
- IRB202401729
- Status last verified
- Feb 2026
- First submitted
- 4 Dec 2024
- Last updated
- 18 Feb 2026
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Compassionate Release Protocol: Expanded Access to CD34+ Selection Utilizing Miltenyi CliniMACS Prodigy® for Patients Receiving Peripheral Blood Stem Cell Transplantations and Stem Cell Boosts
- ClinicalTrials.gov ID
- NCT06731036
- Acronym / sponsor ID
- UF-PEDS036
- Conditions
- Severe Combined Immunodeficiency, Fanconi Anemia, Dyskeratosis Congenita, Sickle Cell Disease
- Keywords
- stem cell transplantation, graft manipulation
- Study type
- Expanded access
- Recruitment status
- Available
- Lead sponsor
- University of FloridaUniversity, hospital or other
- First posted
- 12 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Allogeneic stem cell transplantation (alloSCT) is utilized for various underlying diseases. AlloSCT is limited by graft versus host disease (GVHD), graft rejection, viral infections, and post-transplant lymphoproliferative disorders. To mitigate graft versus host disease, graft manipulation has been taking place with CD34+ selection to decrease T-cells entering into the patient, thus lowering the risk of GVHD.
Historically CD34+ manipulation has been performed under a humanitarian use device by utilizing the Miltenyi CliniMACs CD34 Reagent System. This was used for patients with AML in first remission. This approach has additionally been used for patients with sickle cell disease, immune deficiencies, and poor graft function with excellent efficiency. The purpose of this protocol is to create expanded access of CD34+ manipulation for various underlying diseases utilizing the Miltenyi CliniMACS Prodigy® device.
Study design
Who may qualify
- Ages
- 1 month and older
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (18 listed)
- Patients over 1 month of age
- The following category of patients is eligible for this protocol:
- Severe combined immune deficiency
- Fanconi Anemia
- Dyskeratosis Congenita
Key exclusion criteria (8 listed)
- Patients with a fully HLA matched sibling donor
- Patients with active graft versus host disease
- Patient with uncontrollable transplant associated thrombotic microangiopathy
- Patient not cleared by transplant provider to process
- Donor unable to donate peripheral blood stem cells
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD34+ cell enriched stem cell graftPatients will receive a peripheral blood stem cell graft that has been CD34+ cell enriched using the Miltenyi CliniMACS Prodigy® system. | Biological | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jordan Milner, MD principal investigator
University of Florida
Questions about this trial
What is this clinical trial studying?
A study testing CD34+ cell enriched stem cell graft in people with Severe Combined Immunodeficiency and Fanconi Anemia, run by University of Florida.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Giselle Moore-Higgs, PhD, APRN ((352) 273-9050, mooregj@ufl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2026.