Immunodeficiency SyndromePhase 2NCT00358657
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
A Phase 2 study testing Allogeneic Bone Marrow Transplantation, Cyclophosphamide and Fludarabine Phosphate in people with Immunodeficiency Syndrome and Severe Aplastic Anemia, run by Fred Hutchinson Cancer Center at 3 sites in United States.
Trial summary
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus in treating patients with primary immunodeficiency disorders or noncancerous inherited… Read more about this study
- Who is it for?
- Ages Up to 55 years
- Conditions the study is for
- Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
- What is being tested?
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide and Fludarabine Phosphate
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 3 sitesUnited States
- Study dates
- 24 May 2006to 25 May 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 55 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Primary immunodeficiency disorder or other nonmalignant inherited disease (except Fanconi anemia) treatable by allogeneic HCT
- Patients with pre-existing medical conditions or other factors that renders them at high risk for regimen related toxicity or ineligible for conventional myeloablative HCT and who do not have HLA-matched related or unrelated donors
- Patients with a related donor who is identical for one HLA haplotype
- Acquired aplastic anemia: severe aplastic anemia (SAA) is defined as follows:
- Bone marrow cellularity < 25%, or marrow cellularity < 50% but with < 30% residual hematopoietic cells
- Two out of three of the following (in peripheral blood): neutrophils < 0.5 x 10^9/L; platelets < 20 x 10^9/L; reticulocytes < 20 x 10^9/L
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- SAA diagnostic criteria may be applied to assessment at initial diagnosis or follow-up assessments
- DONOR: Related donors who are identical for one HLA haplotype
- DONOR: Bone marrow will be the only allowed stem cell source
Who cannot join
- Fanconi anemia
- Suitably HLA-matched related or unrelated donors
- Patients with metabolic storage diseases who have severe central nervous system (CNS) involvement of disease, defined as intelligence quotient (IQ) score < 70
- Cardiac ejection fraction < 30% (or, if unable to obtain ejection fraction, shortening fraction < 26%) on multiple-gated acquisition (MUGA) scan or cardiac echocardiogram (echo), symptomatic coronary artery disease, or other cardiac failure requiring therapy; patients with a history of, or current cardiac disease should be evaluated with appropriate cardiac studies and/or cardiology consult; patients with a shortening fraction of < 26% must be seen by cardiology for approval
- Poorly controlled hypertension despite anti-hypertensive medications
- Patients with clinical or laboratory evidence of liver disease will need to be evaluated for the cause of the liver disease, its clinical severity in terms of liver function and the degree of portal hypertension; patients will be excluded if they are found to have fulminant liver failure, cirrhosis of the liver with evidence of portal hypertension, bridging fibrosis, alcoholic hepatitis, esophageal varices, a history of bleeding esophageal varices, hepatic encephalopathy, uncorrectable hepatic synthetic dysfunction evidenced by prolongation of the prothrombin time, ascites related to portal hypertension, bacterial or fungal liver abscess, biliary obstruction, chronic viral hepatitis with total serum bilirubin > 3 mg/dl, or symptomatic biliary disease
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- Positive for human immunodeficiency virus (HIV)
- Females who are pregnant (beta-human chorionic gonadotropin positive [beta-HCG+]) or breast-feeding
- Fertile men or women who are unwilling to use contraceptives during HCT and up to 12 months post-treatment
- Patients with fungal pneumonia with radiological progression after receipt of amphotericin formulation or mold-active azoles for greater than 1 month will not be eligible for this protocol (either regimen A or B)
- DONOR: Donor-recipient pairs in which the HLA-mismatch is only in the host-versus-graft (HVG) direction; patients are homozygous and donor is heterozygous
- DONOR: Donors who are not expected to meet the minimum target dose of marrow cells (1 x 10^8 nucleated cells/kg recipient ideal body weight)
- DONOR: HIV-positive donors
- DONOR: A positive anti-donor cytotoxic cross match is absolute donor exclusion
- DONOR: < 6 months old and > 75 years old
Trial locations and contacts
3 sites in 1 country.
United States3 sites
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109
About this study
Official title: HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus in treating patients with primary immunodeficiency disorders or noncancerous inherited disorders. Giving low doses of chemotherapy and total-body irradiation before a bone marrow transplant helps prepare the patient's body to accept the incoming donor's bone marrow and decrease the risk that the patient's immune system will reject the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells called graft versus host disease. Giving cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus after the transplant may help decrease this from happening.
Read the detailed description
PRIMARY OBJECTIVE:
- Determine safety of nonmyeloablative conditioning and hematopoietic cell transplantation (HCT) from human leukocyte antigen (HLA)-haploidentical related donors for patients with nonmalignant inherited disorders who do not have an HLA-matched related or unrelated donor.
SECONDARY OBJECTIVES:
- Determine whether nonmyeloablative conditioning and HCT from an HLA-haploidentical related donor graft can establish mixed chimerism (> 5% cluster of differentiation [CD]3+ donor T-cell chimerism) in patients with nonmalignant inherited disorders.
II. Transplant related mortality at day 100.
III. Incidence and severity of graft-versus-host disease (GHVD).
IV. Immune reconstitution.
- Infections during the first 200 days after HCT.
OUTLINE:
NONMYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) over 1 hour on days -6 to -2; cyclophosphamide IV over 1 hour on days -6 and -5; and undergo total body irradiation on day -1.
TRANSPLANTATION: Patients undergo allogeneic bone marrow transplant on day 0.
POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on days 3 and 4, and mycophenolate mofetil orally (PO) every 8 hours on days 5-30 then twice daily (BID) to day 40, and then if there is no evidence of active GVHD and donor engraftment is > 95% (or by principal investigator [PI] approval) taper until approximately day 96, or faster at discretion of PI. Patients also receive tacrolimus IV continuously over 22-24 hours starting on day 5 post-transplant and continue on tacrolimus through day 100 followed by a taper to approximately day 180 if there is no evidence of GVHD and their graft is doing well. Patients may convert to oral tacrolimus given BID or three times daily (TID) when the patient is able to take medications orally and has a therapeutic drug level. In addition, patients will receive sirolimus PO beginning on day 5 through day 180 followed by a taper to approximately day 210 if there is no evidence of GVHD and their graft is doing well.
After completion of study treatment, patients are followed up at 6, 12, 18, and 24 months, and then annually for 5 years.
- Being tested
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Mycophenolate Mofetil
- Medical subject headings
- Immunologic Deficiency Syndromes, Anemia, Aplastic, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic Bone Marrow TransplantationProcedure or surgery
Also called Allo BMT, Allogeneic BMT
Undergo allogeneic bone marrow transplantation
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida
Given IV
Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara
Given IV
Laboratory Biomarker AnalysisOther
Correlative studies
Also called Cellcept, MMF
Given PO
Nonmyeloablative Allogeneic Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called Non-myeloablative allogeneic transplant, Nonmyeloablative Stem Cell Transplantation, NST
Undergo allogeneic bone marrow transplantation
SirolimusDrug
Also called AY 22989, RAPA, Rapamune, Rapamycin
Given PO
TacrolimusDrug
Also called FK 506, Fujimycin, Hecoria, Prograf
Given IV or PO
Total-Body IrradiationRadiation
Also called TOTAL BODY IRRADIATION, Whole-Body Irradiation
Undergo total-body irradiation
| Study group | Role | What they receive |
|---|---|---|
| Treatment (chemo, total-body irradiation, transplant) | Experimental | See Detailed Description Receives: Procedure: Allogeneic Bone Marrow Transplantation; Drug: Cyclophosphamide; Drug: Fludarabine Phosphate; Other: Laboratory Biomarker Analysis; Drug: Mycophenolate Mofetil; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Drug: Sirolimus; Drug: Tacrolimus; Radiation: Total-Body Irradiation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Graft Rejection
Number of patients with graft rejection (CD3 donor chimerisms <5%).
Measured: Day 84
Graft Failure
Number of patients with graft failure (grade IV thrombocytopenia and neutropenia after day 21 that lasts > 2 weeks andn is refractory to growth factor support).
Measured: Day 84
Secondary outcomes (7)
Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism
Number of patients who achieve greater than 5% donor T-cell (CD3+) chimerisms
Measured: By day 84
Number of Patients With Transplant Related Mortality
The number of patients with transplant related mortality
Measured: Day 100 post transplant
Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
Number of patients diagnosed with overall GI/G2 acute GVHD by Day 100
Measured: Day 100 post transplant
Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
Number of patients diagnosed with overall GIII/IV acute GVHD by Day 100
Measured: Day 100 post transplant
Incidence of Chronic GVHD
Number of patients diagnosed with chronic GVHD by 1 year post transplant
Measured: 1 year post transplant
Immune Reconstitution
Number of patients with normal range CD3 @ 1 year post transplant
Measured: 1 year post transplant
Number of Patients With Infections
Number of patients with clinically significant infections requiring treatment within 200 days after HCT
Measured: Through day 200 after HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2006 (estimated)
- Study start24 May 2006
- Primary completion17 Aug 2018
- Study completion25 May 2019
Why it stopped: Low accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Immunodeficiency SyndromeStudies of the same condition, open and under way.
- More trials run by Fred Hutchinson Cancer CenterThe 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fred Hutchinson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kanwaldeep K Mallhi, Fred Hutchinson Cancer Center
- Investigators
- Kanwaldeep Mallhi, Principal investigator, Fred Hutch/University of Washington Cancer Consortium
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00358657
- Sponsor's protocol ID
- 2032.00
- Other IDs
- NCI-2010-00192, 2032, 2032.00, P01HL122173, P30CA015704, RG9213024
- Status last verified
- Jan 2021
- First submitted
- 28 Jul 2006
- Last updated
- 26 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide
- ClinicalTrials.gov ID
- NCT00358657
- Acronym / sponsor ID
- 2032.00
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low accrual
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Fred Hutchinson Cancer CenterUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI), National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 24 May 2006 (actual)
- Primary completion
- 17 Aug 2018 (actual)
- Study completion
- 25 May 2019 (actual)
- First posted
- 1 Aug 2006 (estimated)
- Results
- Posted 26 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus in treating patients with primary immunodeficiency disorders or noncancerous inherited disorders. Giving low doses of chemotherapy and total-body irradiation before a bone marrow transplant helps prepare the patient's body to accept the incoming donor's bone marrow and decrease the risk that the patient's immune system will reject the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells called graft versus host disease. Giving cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus after the transplant may help decrease this from happening.
Detailed description
PRIMARY OBJECTIVE:
- Determine safety of nonmyeloablative conditioning and hematopoietic cell transplantation (HCT) from human leukocyte antigen (HLA)-haploidentical related donors for patients with nonmalignant inherited disorders who do not have an HLA-matched related or unrelated donor.
SECONDARY OBJECTIVES:
- Determine whether nonmyeloablative conditioning and HCT from an HLA-haploidentical related donor graft can establish mixed chimerism (> 5% cluster of differentiation [CD]3+ donor T-cell chimerism) in patients with nonmalignant inherited disorders.
II. Transplant related mortality at day 100.
III. Incidence and severity of graft-versus-host disease (GHVD).
IV. Immune reconstitution.
- Infections during the first 200 days after HCT.
OUTLINE:
NONMYELOABLATIVE CONDITIONING REGIMEN: Patients receive fludarabine phosphate intravenously (IV) over 1 hour on days -6 to -2; cyclophosphamide IV over 1 hour on days -6 and -5; and undergo total body irradiation on day -1.
TRANSPLANTATION: Patients undergo allogeneic bone marrow transplant on day 0.
POST-TRANSPLANT IMMUNOSUPPRESSION: Patients receive cyclophosphamide IV over 1 hour on days 3 and 4, and mycophenolate mofetil orally (PO) every 8 hours on days 5-30 then twice daily (BID) to day 40, and then if there is no evidence of active GVHD and donor engraftment is > 95% (or by principal investigator [PI] approval) taper until approximately day 96, or faster at discretion of PI. Patients also receive tacrolimus IV continuously over 22-24 hours starting on day 5 post-transplant and continue on tacrolimus through day 100 followed by a taper to approximately day 180 if there is no evidence of GVHD and their graft is doing well. Patients may convert to oral tacrolimus given BID or three times daily (TID) when the patient is able to take medications orally and has a therapeutic drug level. In addition, patients will receive sirolimus PO beginning on day 5 through day 180 followed by a taper to approximately day 210 if there is no evidence of GVHD and their graft is doing well.
After completion of study treatment, patients are followed up at 6, 12, 18, and 24 months, and then annually for 5 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (chemo, total-body irradiation, transplant) | Experimental | Procedure: Allogeneic Bone Marrow Transplantation, Drug: Cyclophosphamide, Drug: Fludarabine Phosphate, Other: Laboratory Biomarker Analysis, Drug: Mycophenolate Mofetil, Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation, Drug: Sirolimus, Drug: Tacrolimus, Radiation: Total-Body IrradiationSee Detailed Description |
Who may qualify
- Ages
- Up to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Primary immunodeficiency disorder or other nonmalignant inherited disease (except Fanconi anemia) treatable by allogeneic HCT
- Patients with pre-existing medical conditions or other factors that renders them at high risk for regimen related toxicity or ineligible for conventional myeloablative HCT and who do not have HLA-matched related or unrelated donors
- Patients with a related donor who is identical for one HLA haplotype
- Acquired aplastic anemia: severe aplastic anemia (SAA) is defined as follows:
- DONOR: Related donors who are identical for one HLA haplotype
Key exclusion criteria (15 listed)
- Fanconi anemia
- Suitably HLA-matched related or unrelated donors
- Patients with metabolic storage diseases who have severe central nervous system (CNS) involvement of disease, defined as intelligence quotient (IQ) score < 70
- Cardiac ejection fraction < 30% (or, if unable to obtain ejection fraction, shortening fraction < 26%) on multiple-gated acquisition (MUGA) scan or cardiac echocardiogram (echo), symptomatic coronary artery disease, or other cardiac failure requiring therapy; patients with a history of, or current cardiac disease should be evaluated with appropriate cardiac studies and/or cardiology consult; patients with a shortening fraction of < 26% must be seen by cardiology for approval
- Poorly controlled hypertension despite anti-hypertensive medications
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 |
|---|---|---|
| Allogeneic Bone Marrow TransplantationAlso: Allo BMT, Allogeneic BMTUndergo allogeneic bone marrow transplantation | Procedure or surgery | Treatment (chemo, total-body irradiation, transplant) |
| CyclophosphamideAlso: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, CiclofosfamidaGiven IV | Drug | Treatment (chemo, total-body irradiation, transplant) |
| Fludarabine PhosphateAlso: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, FludaraGiven IV | Drug | Treatment (chemo, total-body irradiation, transplant) |
| Laboratory Biomarker AnalysisCorrelative studies | Other | Treatment (chemo, total-body irradiation, transplant) |
| Mycophenolate MofetilAlso: Cellcept, MMFGiven PO | Drug | Treatment (chemo, total-body irradiation, transplant) |
| Nonmyeloablative Allogeneic Hematopoietic Stem Cell TransplantationAlso: Non-myeloablative allogeneic transplant, Nonmyeloablative Stem Cell Transplantation, NSTUndergo allogeneic bone marrow transplantation | Procedure or surgery | Treatment (chemo, total-body irradiation, transplant) |
| SirolimusAlso: AY 22989, RAPA, Rapamune, RapamycinGiven PO | Drug | Treatment (chemo, total-body irradiation, transplant) |
| TacrolimusAlso: FK 506, Fujimycin, Hecoria, PrografGiven IV or PO | Drug | Treatment (chemo, total-body irradiation, transplant) |
| Total-Body IrradiationAlso: TOTAL BODY IRRADIATION, Whole-Body IrradiationUndergo total-body irradiation | Radiation | Treatment (chemo, total-body irradiation, transplant) |
Primary outcome
Graft Rejection
Time frame: Day 84
Number of patients with graft rejection (CD3 donor chimerisms <5%).
Graft Failure
Time frame: Day 84
Number of patients with graft failure (grade IV thrombocytopenia and neutropenia after day 21 that lasts > 2 weeks andn is refractory to growth factor support).
7 secondary outcomes
Proportion of Patients Who Achieve Greater Than 5% Donor T-cell Chimerism
Time frame: By day 84
Number of Patients With Transplant Related Mortality
Time frame: Day 100 post transplant
Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
Time frame: Day 100 post transplant
Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
Time frame: Day 100 post transplant
Incidence of Chronic GVHD
Time frame: 1 year post transplant
Immune Reconstitution
Time frame: 1 year post transplant
Number of Patients With Infections
Time frame: Through day 200 after HCT
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Kanwaldeep Mallhi principal investigator
Fred Hutch/University of Washington Cancer Consortium
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Allogeneic Bone Marrow Transplantation, Cyclophosphamide and Fludarabine Phosphate in people with Immunodeficiency Syndrome and Severe Aplastic Anemia, run by Fred Hutchinson Cancer Center at 3 sites 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 9 inclusion and 15 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: stopped early, as of the sponsor's last update on 26 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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?
Yes. Results are posted in the registry (first posted 26 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Graft Rejection (time frame: Day 84). The study lists 2 primary outcomes in all. 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 26 Jan 2021.