Fanconi AnemiaPhase 2NCT03579875
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
A Phase 2 study testing Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1) and Fludarabine (FLU) in people with Fanconi Anemia and Severe Aplastic Anemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and… Read more about this study
- Who is it for?
- Ages Up to 65 years
- Conditions the study is for
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes
- What is being tested?
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1) and Fludarabine (FLU)
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Nov 2018to 5 Jan 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 Up to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- For FA patients:
- Diagnosis of Fanconi anemia
- Age <65 years of age
- Has one of the following risk factors:
- Severe aplastic anemia (SAA)
- Myelodysplastic features
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- High risk genotype
- Immunodeficiency associated with history of recurrent infections
- Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients <16 years of age
- Adequate pulmonary, cardiac and liver function
- Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
- For TBD patients:
- Diagnosis of TBD
- Age <70 years of age
- Has one of the following risk factors:
- Severe aplastic anemia (SAA)
- Myelodysplastic features
- Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score
- ≥ 50% for patients <16 years of age
- Adequate pulmonary, cardiac and liver function
- Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
Who cannot join
- Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
- Active, uncontrolled infection within 1 week prior to starting study therapy
- Malignant solid tumor cancer within previous 2 years
- Donor Selection (Inclusion Criteria): meets one of the following match criteria:
- an HLA-A, B, DRB1 matched sibling donor (matched sibling)
- an HLA-A, B, DRB1 matched related donor (other than sibling)
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- a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen
- 7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.
- Body weight of at least 40 kilograms and at least 12 years of age
- Willing and able to undergo mobilized peripheral blood apheresis
- In general good health as determined by the medical provider
- Adequate organ function defined as:
- Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)
- Hepatic: ALT < 2 x upper limit of normal
- Renal: serum creatinine < 1.8 mg/dl
- Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1/2 Antibody, HTLVA 1/2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B
- Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start
- Voluntary written consent (parent/guardian and minor assent, if < 18 years) prior to the performance of any research related procedure
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Masonic Cancer Center at University of Minnesota
Minneapolis, Minnesota, 55455
Lisa Burke, RN, 612-273-8482, lburke3@Fairview.org
About this study
Official title: MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure
- Being tested
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU), Methylprednisolone (MP), Donor mobilized PBSC infusion
- Medical subject headings
- Fanconi Anemia, Anemia, Aplastic, Myelodysplastic Syndromes, Bone Marrow Failure Disorders, Dyskeratosis Congenita
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Total Body Irradiation (TBI) (Plan 1)Drug
Also called TBI
300 cGy with thymic shielding on day -6
Cyclophosphamide (CY) (Plan 1)Drug
Also called CY
10 mg/kg IV daily on days -5, -4, -3, and -2
Fludarabine (FLU)Drug
Also called FLU
35 mg/m2 IV daily on days -5, -4, -3, and -2
Methylprednisolone (MP)Drug
Also called MP
1 mg/kg IV q12h on days -5, -4, -3, -2, and -1
Donor mobilized PBSC infusionDevice
Also called PBSC
T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation on day 0
G-CSFDrug
Initiate G-CSF 5mcg/kg per day IV on day +1 (continue until ANC >2.5 x 10^9/L for 3 consecutive days or single day ANC >3000 Arm 1 and Arm 3)
Cyclophosphamide (CY) (Plan 2)Drug
Also called CY
5 mg/kg IV daily on days -5, -4, -3, and -2
RituximabDrug
200 mg/m2 IV once on day -1
BusulfanDrug
Also called BU
Busulfan 0.6 mg/kg if > 4 years old and/or >12 kg (0.8 mg/kg IV if ≤ 4 years old and/or ≤ 12 kg) is given IV over 2 hours every 12 hours for 2 days.
AlemtuzumabDrug
Alemtuzumab 0.2 mg/kg is given IV over 2 hours daily for 5 days (total dose 1 mg/kg)
MelphalanDrug
Also called MEL
If available, MEL dosing will be model-based using Bayesian methodology. If Bayesian methodology is unavailable, MEL dosing will be weight-based: MEL 70 mg/m2 for patients ≥10 kg (2.35 mg/kg for patients <10 kg^) IV for one dose over 30 minutes.
RituximabDrug
Rituximab will be given once on treatment plans 1-3 on day -1.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia | Experimental | Given to:
* Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type OR
* Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia Receives: Drug: Total Body Irradiation (TBI) (Plan 1); Drug: Cyclophosphamide (CY) (Plan 1); Drug: Fludarabine (FLU); Drug: Methylprednisolone (MP); Device: Donor mobilized PBSC infusion; Drug: G-CSF; Drug: Rituximab |
| Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia | Experimental | Given to:
• An HLA-identical sibling donor recipients with single or multi- lineage hematopoietic failure Receives: Drug: Fludarabine (FLU); Drug: Methylprednisolone (MP); Device: Donor mobilized PBSC infusion; Drug: G-CSF; Drug: Cyclophosphamide (CY) (Plan 2); Drug: Rituximab |
| Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia | Experimental | Given to:
* Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type who cannot tolerate TBI
* Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia who cannot tolerate TBI
* Biallelic mutations in FANCD1/BRCA2 who cannot receive TBI
* Per treating physician preference Receives: Drug: Cyclophosphamide (CY) (Plan 1); Drug: Fludarabine (FLU); Drug: Methylprednisolone (MP); Drug: Rituximab; Drug: Busulfan |
| Treatment Plan 4: CY, FLU, and alemtuzumab | Experimental | given to TBD patients with:
* Bone marrow failure AND
* Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 3 patients would be treated per year. Statistical outcomes will be descriptive. Receives: Drug: Cyclophosphamide (CY) (Plan 1); Drug: Fludarabine (FLU); Drug: Alemtuzumab |
| Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. | Experimental | given to TBD patients with:
* Early myelodysplastic features (with or without cytogenetic abnormalities) AND
* Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 2 patients would be treated per year. Statistical outcomes will be descriptive. Receives: Drug: Cyclophosphamide (CY) (Plan 1); Drug: Fludarabine (FLU); Drug: Alemtuzumab; Drug: Melphalan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Grade II-IV acute graft versus host disease (GVHD)
incidence of grade II-IV acute graft versus host disease (GVHD)
Measured: Day 100
Secondary outcomes (8)
Neutrophil engraftment
Rate of neutrophil engraftment (defined as the first of three consecutive days after HCT that the patient's absolute neutrophil counts is ≥ 0.5x109 per liter)
Measured: Day 42
Platelet engraftment
Time to platelet engraftment (First of three consecutive days after HCT that the patient's platelet count ≥ 20x10^9 per liter)
Measured: Day 42
Acute graft versus host disease (aGVHD)
Incidence of grade III-IV acute graft versus host disease
Measured: Day 100
Chronic graft versus host disease (cGVHD)
Incidence of chronic graft versus host disease after transplant
Measured: 1 Year after transplant
Regimen related toxicity
Incidence of regimen related toxicity based on CTCAE v5
Measured: 30 Days after transplant
Bacterial, viral and fungal infections
Incidence of bacterial, viral and fungal infections
Measured: 1 Year after transplant
Opportunistic infections
Incidence of opportunistic infections
Measured: 100 Days after transplant
Overall survival (OS)
Incidence of overall survival
Measured: 1 Year after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2018
- Study start13 Nov 2018
- Primary completion1 Jan 2027 (estimated)
- Study completion5 Jan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Margaret MacMillan, MD, Msc, FRCPC, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03579875
- Sponsor's protocol ID
- 2016LS161
- Other IDs
- MT2017-17
- Status last verified
- Jan 2026
- First submitted
- 25 May 2018
- Last updated
- 30 Jan 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
- ClinicalTrials.gov ID
- NCT03579875
- Acronym / sponsor ID
- 2016LS161
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion, Telomere Biology Disorder, Bone Marrow Failure
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 13 Nov 2018 (actual)
- Primary completion
- 1 Jan 2027 (estimated)
- Study completion
- 5 Jan 2029 (estimated)
- First posted
- 9 Jul 2018 (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.
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia | Experimental | Drug: Total Body Irradiation (TBI) (Plan 1), Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Methylprednisolone (MP), Device: Donor mobilized PBSC infusion, Drug: G-CSF, Drug: RituximabGiven to: * Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type OR * Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia |
| Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia | Experimental | Drug: Fludarabine (FLU), Drug: Methylprednisolone (MP), Device: Donor mobilized PBSC infusion, Drug: G-CSF, Drug: Cyclophosphamide (CY) (Plan 2), Drug: RituximabGiven to: • An HLA-identical sibling donor recipients with single or multi- lineage hematopoietic failure |
| Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia | Experimental | Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Methylprednisolone (MP), Drug: Rituximab, Drug: BusulfanGiven to: * Patients with an unrelated donor or HLA mismatched related donor, regardless of disease type who cannot tolerate TBI * Patients with an HLA- identical sibling donor recipient and myelodysplastic features, MDS, or acute leukemia who cannot tolerate TBI * Biallelic mutations in FANCD1/BRCA2 who cannot receive TBI * Per treating physician preference |
| Treatment Plan 4: CY, FLU, and alemtuzumab | Experimental | Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Alemtuzumabgiven to TBD patients with: * Bone marrow failure AND * Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 3 patients would be treated per year. Statistical outcomes will be descriptive. |
| Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. | Experimental | Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Alemtuzumab, Drug: Melphalangiven to TBD patients with: * Early myelodysplastic features (with or without cytogenetic abnormalities) AND * Any donor type including haploidentical (4/8) to 8/8-HLA matched related donor, or 7-8/8 HLA-matched unrelated donor Based on historical numbers, it is expected approximately 2 patients would be treated per year. Statistical outcomes will be descriptive. |
Who may qualify
- Ages
- Up to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (21 listed)
- Diagnosis of Fanconi anemia
- Has one of the following risk factors:
- Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients <16 years of age
- Diagnosis of TBD
- Age <70 years of age
Key exclusion criteria (18 listed)
- Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
- Active, uncontrolled infection within 1 week prior to starting study therapy
- Malignant solid tumor cancer within previous 2 years
- an HLA-A, B, DRB1 matched sibling donor (matched sibling)
- an HLA-A, B, DRB1 matched related donor (other than sibling)
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 |
|---|---|---|
| Total Body Irradiation (TBI) (Plan 1)Also: TBI300 cGy with thymic shielding on day -6 | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia |
| Cyclophosphamide (CY) (Plan 1)Also: CY10 mg/kg IV daily on days -5, -4, -3, and -2 | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia, Treatment Plan 4: CY, FLU, and alemtuzumab, Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. |
| Fludarabine (FLU)Also: FLU35 mg/m2 IV daily on days -5, -4, -3, and -2 | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia, Treatment Plan 4: CY, FLU, and alemtuzumab, Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. |
| Methylprednisolone (MP)Also: MP1 mg/kg IV q12h on days -5, -4, -3, -2, and -1 | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia |
| Donor mobilized PBSC infusionAlso: PBSCT cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation on day 0 | Device | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia |
| G-CSFInitiate G-CSF 5mcg/kg per day IV on day +1 (continue until ANC >2.5 x 10^9/L for 3 consecutive days or single day ANC >3000 Arm 1 and Arm 3) | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia |
| Cyclophosphamide (CY) (Plan 2)Also: CY5 mg/kg IV daily on days -5, -4, -3, and -2 | Drug | Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia |
| Rituximab200 mg/m2 IV once on day -1 | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia |
| BusulfanAlso: BUBusulfan 0.6 mg/kg if > 4 years old and/or >12 kg (0.8 mg/kg IV if ≤ 4 years old and/or ≤ 12 kg) is given IV over 2 hours every 12 hours for 2 days. | Drug | Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia |
| AlemtuzumabAlemtuzumab 0.2 mg/kg is given IV over 2 hours daily for 5 days (total dose 1 mg/kg) | Drug | Treatment Plan 4: CY, FLU, and alemtuzumab, Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. |
| MelphalanAlso: MELIf available, MEL dosing will be model-based using Bayesian methodology. If Bayesian methodology is unavailable, MEL dosing will be weight-based: MEL 70 mg/m2 for patients ≥10 kg (2.35 mg/kg for patients <10 kg^) IV for one dose over 30 minutes. | Drug | Treatment Plan 5: CY, FLU, melphalan (MEL), and alemtuzumab. |
| RituximabRituximab will be given once on treatment plans 1-3 on day -1. | Drug | Treatment Plan 1: TBI 300 , CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 2: CY, FLU, MP, Rituximab in patients with Fanconi Anemia, Treatment Plan 3: BU, Cy, FLU, MP and Rituximab in patients with Fanconi Anemia |
Primary outcome
Grade II-IV acute graft versus host disease (GVHD)
Time frame: Day 100
incidence of grade II-IV acute graft versus host disease (GVHD)
8 secondary outcomes
Neutrophil engraftment
Time frame: Day 42
Platelet engraftment
Time frame: Day 42
Acute graft versus host disease (aGVHD)
Time frame: Day 100
Chronic graft versus host disease (cGVHD)
Time frame: 1 Year after transplant
Regimen related toxicity
Time frame: 30 Days after transplant
Bacterial, viral and fungal infections
Time frame: 1 Year after transplant
Opportunistic infections
Time frame: 100 Days after transplant
Overall survival (OS)
Time frame: 1 Year after transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jan 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.
Margaret MacMillan, MD, Msc, FRCPC principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1) and Fludarabine (FLU) in people with Fanconi Anemia and Severe Aplastic Anemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
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 21 inclusion and 18 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: looking for participants now, as of the sponsor's last update on 30 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Margaret MacMillan, MD, Msc, FRCPC (612-626-2961, macmi002@umn.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Grade II-IV acute graft versus host disease (GVHD) (time frame: Day 100). It is what the study was designed to measure, not a finding.
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 30 Jan 2026.