Complication of Transplanted Organ, NosPhase 2ASCOTTNCT02549586
Autologous Hematopoietic Stem Cell Transplantation for Allogeneic Organ Transplant Tolerance
A Phase 2 study testing Autologous Hematopoietic Stem Cell Transplant in people with Complication of Transplanted Organ, Nos, run by Gary A Levy, O. Ont. MD. FRCP AGAF at 2 sites in Canada.
Trial summary
This open-label, proof-of-principle two center cohort study will evaluate the ability of autologous hematopoietic stem cell transplantation to induce tolerance in recipients of deceased or live donor liver transplants (ASCOTT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Complication of Transplanted Organ, Nos
- What is being tested?
- Autologous Hematopoietic Stem Cell Transplant
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 2 sitesCanada
- Study dates
- Dec 2015to 30 Apr 2020
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants must be 18 years of age or older.
- Participants must be a minimum of 6 months post-transplant;
- Participants are recipients of a hepatic allograft for alcohol induced liver disease; or a genetic form of liver disease such as hemochromatosis or Wilson's disease; or an autoimmune liver disease including sclerosing cholangitis, autoimmune hepatitis, and/or primary biliary cirrhosis.
- Participants have a complication of transplantation that might be ameliorated by HSCT and/or withdrawal of immunosuppression such as: evidence of recurrent autoimmune disease in the graft; repeated episodes (minimum of 2) of acute cellular rejection; and/ or development of adverse events related to immune suppression which have not been well managed by conventional methods including drug dose reduction or substitution of other medications. Examples include progression of renal dysfunction, repeated infections, neurologic complications, cardiovascular complications, or post-transplant lymphoproliferative disease (PTLD) that has been in remission for at least 12 months. These complications must be deemed serious enough to warrant inclusion in the study by the investigator.
Who cannot join
- Participants < 18 yr.
- Participants with cardiac, renal, pulmonary, hepatic, or other organ impairment that would limit their ability to receive dose intensive chemotherapy;
- Participants with any active or chronic infection. Participants with previous reactivation of Epstein-Barr virus, cytomegalovirus , BK, human herpesvirus 6 or varicella-zoster virus would be considered eligible if the virus has returned to a latent state;
- Participants who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C;
- Participants with a previous history of a malignancy other than squamous or basal cell carcinoma of the skin, or post-transplant lymphoproliferative disease (PTLD);
- Participants whose life expectancy is severely limited by another co-morbid illness;
Show all 10Show fewer
- Participants with evidence of myelodysplasia, other non-autoimmune cytopenia, or an inherited immunodeficiency state;
- Pregnancy or Participants who are unwilling to practice two active forms of contraception during the time of chemotherapy administration. Participants must be willing to commit to not becoming pregnant from enrollment in the study until 2 years following their HSCT.
- Participants unable to comply with the medical treatment specified in the protocol;
- Participants unable to give written informed consent in accordance with research ethics board guidelines.
Trial locations and contacts
2 sites in 1 country.
Canada2 sites
The Ottawa Hopital
Ottawa, Ontario, K1H 8L6
Multi-Organ Transplant Program, Toronto General Hospital
Toronto, Ontario, M5G 2N2
About this study
This open-label, proof-of-principle two center cohort study will evaluate the ability of autologous hematopoietic stem cell transplantation to induce tolerance in recipients of deceased or live donor liver transplants (ASCOTT). A maximum of 10 participants will be entered at a minimum of 3 months post liver transplant. The participants will undergo autologous hematopoietic stem cell transplants (HSCT) to "re-educate" their immune systems to accept the graft without the need for long term immunosuppression (tolerance).
Read the detailed description
Although short-term results for liver transplantation are excellent, the need for immunosuppression limits quality of life and long-term survival.
Investigators plan to examine the utility and safety of autologous hematopoietic stem cell transplantation (HSCT) to allow withdrawal of immunosuppression in 10 liver transplant recipients who are at a high risk of developing recurrent liver damage from repeated bouts of rejection, or recurrent disease or who have a high likelihood of developing serious medical complications from complications of immune suppression.
Hematopoietic stem cells will be mobilized, purified and cryopreserved. Following a chemotherapy and Anti-thymocyte Globulin (ATG) based-regimen for immune ablation, the purified stem cells will be thawed and infused back into participants (autologous hematopoietic stem cell transplant - HSCT). Participants will be converted to everolimus, a mammalian target of rapamycin inhibitor (mTORi), which will be continued for 6 months and then withdrawn based on histologic evidence of graft acceptance.
Participants will be followed closely for a total of 24 months for any biochemical and histologic evidence of tolerance or rejection. Re-vaccination to common viral and bacterial antigens will be undertaken as required using a standard protocol for recipients of a hematopoietic stem cell transplant (HSCT).
- Conditions
- Complication of Transplanted Organ, Nos
- Being tested
- Autologous Hematopoietic Stem Cell Transplant
- Keywords
- solid organ transplant, immune tolerance, autologous stem cell transplant, blood and marrow transplant, autoimmune disease
- Medical subject headings
- Pyloric Stenosis, Hypertrophic, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Stem Cell TransplantDrug
Also called HSCT
Step 1: Participants will receive intravenous chemotherapy and cytokine-based treatment for mobilization of hematopoietic stem cells (HSC) into the circulation, followed by collection using peripheral vein leukopheresis. The HSC graft product will undergo ex vivo purification with CD34 selection using Miltenyil CliniMACS and cryopreserved.
Step 2: Intravenous busulphan, cyclophosphamide and anti-thymocyte globulin will be administered to participants to achieve immune ablation prior to the infusion of the participants' own thawed HSC graft product (HSC transplant). Routine supportive measures will be employed during the recovery from the chemotherapy and HSCT. Participants' immune suppression will be stopped at the time of immunoablative therapy and will be switched to everolimus which will be discontinued at 6 months post HSCT.
| Study group | Role | What they receive |
|---|---|---|
| Autologous HSCT | Experimental | Eligible participants will undergo an Autologous Hematopoietic Stem Cell Transplant (HSCT) as a two-step intervention. Receives: Drug: Autologous Hematopoietic Stem Cell Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients who develop tolerance post autologous HSCT
Number of patients who develop tolerance at 24 months post HSCT as defined clinically and histologically in the absence of any immunosuppression in liver transplant recipients.
Measured: 24 months post HSCT
Secondary outcomes (3)
HSCT mortality
Measured: 2 years post HSCT
HSCT related morbidity
Measured: at 2 years post HSCT
Rate of immune reconstitution
Measured: at 2 years post HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2015 (estimated)
- Study startDec 2015
- Primary completion30 Apr 2020
- Study completion30 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Complication of Transplanted Organ, NosStudies of the same condition, open and under way.
- 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
- Model description
- Pt are receiving everolimus in combination with autologous stem cell transplant to induce tolerance
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gary A Levy, O. Ont. MD. FRCP AGAF
- Sponsor type
- University, hospital or other
- Collaborators
- Ottawa Hospital Research Institute
- Responsible party
- Sponsor-investigator, Gary A Levy, O. Ont. MD. FRCP AGAF, University of Toronto
- Investigators
- Gary A Levy, MD FRCP AGAF, Principal investigator, University of Toronto Transplant Institute - Multi Organ Transplant ProgramHarold L Atkins, MD FRCP(C), Principal investigator, Ottawa Hospital Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02549586
- Sponsor's protocol ID
- Tol 001
- Acronym
- ASCOTT
- Status last verified
- Apr 2021
- First submitted
- 1 Sep 2015
- Last updated
- 28 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Sykes M, Levy G. Advances in transplantation. Semin Immunol. 2011 Aug;23(4):222-3. doi: 10.1016/j.smim.2011.08.013. Epub 2011 Sep 22. No abstract available.
PubMed 21943504Sayegh MH, Carpenter CB. Transplantation 50 years later--progress, challenges, and promises. N Engl J Med. 2004 Dec 23;351(26):2761-6. doi: 10.1056/NEJMon043418. No abstract available.
PubMed 15616214Azzi JR, Sayegh MH, Mallat SG. Calcineurin inhibitors: 40 years later, can't live without .. J Immunol. 2013 Dec 15;191(12):5785-91. doi: 10.4049/jimmunol.1390055.
PubMed 24319282Selzner N, Grant DR, Shalev I, Levy GA. The immunosuppressive pipeline: meeting unmet needs in liver transplantation. Liver Transpl. 2010 Dec;16(12):1359-72. doi: 10.1002/lt.22193.
PubMed 21117245Kaplan B, Qazi Y, Wellen JR. Strategies for the management of adverse events associated with mTOR inhibitors. Transplant Rev (Orlando). 2014 Jul;28(3):126-33. doi: 10.1016/j.trre.2014.03.002. Epub 2014 Mar 12.
PubMed 24685370Gotthardt DN, Bruns H, Weiss KH, Schemmer P. Current strategies for immunosuppression following liver transplantation. Langenbecks Arch Surg. 2014 Dec;399(8):981-8. doi: 10.1007/s00423-014-1191-9. Epub 2014 Apr 20.
PubMed 24748543Sachs DH, Kawai T, Sykes M. Induction of tolerance through mixed chimerism. Cold Spring Harb Perspect Med. 2014 Jan 1;4(1):a015529. doi: 10.1101/cshperspect.a015529.
PubMed 24384815Kawai T, Sachs DH, Sykes M, Cosimi AB; Immune Tolerance Network. HLA-mismatched renal transplantation without maintenance immunosuppression. N Engl J Med. 2013 May 9;368(19):1850-2. doi: 10.1056/NEJMc1213779. No abstract available.
PubMed 23656665Xie L, Ichimaru N, Morita M, Chen J, Zhu P, Wang J, Urbanellis P, Shalev I, Nagao S, Sugioka A, Zhong L, Nonomura N, Takahara S, Levy GA, Li XK. Identification of a novel biomarker gene set with sensitivity and specificity for distinguishing between allograft rejection and tolerance. Liver Transpl. 2012 Apr;18(4):444-54. doi: 10.1002/lt.22480.
PubMed 22162188Chruscinski A, Juvet S, Moshkelgosha S, Renner E, Lilly L, Selzner N, Bredeson C, Grant D, Adeyi O, Fischer S, Demetris AJ, Zhang J, Epstein M, Macarthur M, Clement AM, Khalili K, Allan D, Altouri S, Bence-Bruckler I, Cattral M, Fulcher J, Galvin Z, Ghanekar A, Greig P, Huebsch L, Humar A, Kew A, Kekre N, Kim TK, McDiarmid S, Martin L, McGilvray I, Sabloff M, Sapisochin G, Selzner M, Smith R, Tinckam K, Yi TJ, Levy G, Atkins H. Autologous Hematopoietic Stem Cell Transplantation for Liver Transplant Recipients With Recurrent Primary Sclerosing Cholangitis: A Pilot Study. Transplantation. 2022 Mar 1;106(3):562-574. doi: 10.1097/TP.0000000000003829.
PubMed 34049362
Study at a glance
From the registry record, last updated by the sponsor on 28 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Autologous Hematopoietic Stem Cell Transplantation for Allogeneic Organ Transplant Tolerance
- ClinicalTrials.gov ID
- NCT02549586
- Acronym / sponsor ID
- ASCOTT / Tol 001
- Conditions
- Complication of Transplanted Organ, Nos
- Keywords
- solid organ transplant, immune tolerance, autologous stem cell transplant, blood and marrow transplant, autoimmune disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Gary A Levy, O. Ont. MD. FRCP AGAFUniversity, hospital or other
- Collaborators
- Ottawa Hospital Research Institute
- Study start
- Dec 2015 (actual)
- Primary completion
- 30 Apr 2020 (actual)
- Study completion
- 30 Apr 2020 (actual)
- First posted
- 15 Sep 2015 (estimated)
- 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 open-label, proof-of-principle two center cohort study will evaluate the ability of autologous hematopoietic stem cell transplantation to induce tolerance in recipients of deceased or live donor liver transplants (ASCOTT). A maximum of 10 participants will be entered at a minimum of 3 months post liver transplant. The participants will undergo autologous hematopoietic stem cell transplants (HSCT) to "re-educate" their immune systems to accept the graft without the need for long term immunosuppression (tolerance).
Detailed description
Although short-term results for liver transplantation are excellent, the need for immunosuppression limits quality of life and long-term survival.
Investigators plan to examine the utility and safety of autologous hematopoietic stem cell transplantation (HSCT) to allow withdrawal of immunosuppression in 10 liver transplant recipients who are at a high risk of developing recurrent liver damage from repeated bouts of rejection, or recurrent disease or who have a high likelihood of developing serious medical complications from complications of immune suppression.
Hematopoietic stem cells will be mobilized, purified and cryopreserved. Following a chemotherapy and Anti-thymocyte Globulin (ATG) based-regimen for immune ablation, the purified stem cells will be thawed and infused back into participants (autologous hematopoietic stem cell transplant - HSCT). Participants will be converted to everolimus, a mammalian target of rapamycin inhibitor (mTORi), which will be continued for 6 months and then withdrawn based on histologic evidence of graft acceptance.
Participants will be followed closely for a total of 24 months for any biochemical and histologic evidence of tolerance or rejection. Re-vaccination to common viral and bacterial antigens will be undertaken as required using a standard protocol for recipients of a hematopoietic stem cell transplant (HSCT).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Pt are receiving everolimus in combination with autologous stem cell transplant to induce tolerance
| Arm | Type | Receives |
|---|---|---|
| Autologous HSCT | Experimental | Drug: Autologous Hematopoietic Stem Cell TransplantEligible participants will undergo an Autologous Hematopoietic Stem Cell Transplant (HSCT) as a two-step intervention. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Participants must be 18 years of age or older.
- Participants must be a minimum of 6 months post-transplant;
- Participants are recipients of a hepatic allograft for alcohol induced liver disease; or a genetic form of liver disease such as hemochromatosis or Wilson's disease; or an autoimmune liver disease including sclerosing cholangitis, autoimmune hepatitis, and/or primary biliary cirrhosis.
- Participants have a complication of transplantation that might be ameliorated by HSCT and/or withdrawal of immunosuppression such as: evidence of recurrent autoimmune disease in the graft; repeated episodes (minimum of 2) of acute cellular rejection; and/ or development of adverse events related to immune suppression which have not been well managed by conventional methods including drug dose reduction or substitution of other medications. Examples include progression of renal dysfunction, repeated infections, neurologic complications, cardiovascular complications, or post-transplant lymphoproliferative disease (PTLD) that has been in remission for at least 12 months. These complications must be deemed serious enough to warrant inclusion in the study by the investigator.
Key exclusion criteria (10 listed)
- Participants < 18 yr.
- Participants with cardiac, renal, pulmonary, hepatic, or other organ impairment that would limit their ability to receive dose intensive chemotherapy;
- Participants with any active or chronic infection. Participants with previous reactivation of Epstein-Barr virus, cytomegalovirus , BK, human herpesvirus 6 or varicella-zoster virus would be considered eligible if the virus has returned to a latent state;
- Participants who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C;
- Participants with a previous history of a malignancy other than squamous or basal cell carcinoma of the skin, or post-transplant lymphoproliferative disease (PTLD);
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 |
|---|---|---|
| Autologous Hematopoietic Stem Cell TransplantAlso: HSCTStep 1: Participants will receive intravenous chemotherapy and cytokine-based treatment for mobilization of hematopoietic stem cells (HSC) into the circulation, followed by collection using peripheral vein leukopheresis. The HSC graft product will undergo ex vivo purification with CD34 selection using Miltenyil CliniMACS and cryopreserved. Step 2: Intravenous busulphan, cyclophosphamide and anti-thymocyte globulin will be administered to participants to achieve immune ablation prior to the infusion of the participants' own thawed HSC graft product (HSC transplant). Routine supportive measures will be employed during the recovery from the chemotherapy and HSCT. Participants' immune suppression will be stopped at the time of immunoablative therapy and will be switched to everolimus which will be discontinued at 6 months post HSCT. | Drug | Autologous HSCT |
Primary outcome
Number of patients who develop tolerance post autologous HSCT
Time frame: 24 months post HSCT
Number of patients who develop tolerance at 24 months post HSCT as defined clinically and histologically in the absence of any immunosuppression in liver transplant recipients.
3 secondary outcomes
HSCT mortality
Time frame: 2 years post HSCT
HSCT related morbidity
Time frame: at 2 years post HSCT
Rate of immune reconstitution
Time frame: at 2 years post HSCT
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Apr 2021.
- Background: PubMed 21943504Sykes M, Levy G. Advances in transplantation. Semin Immunol. 2011 Aug;23(4):222-3. doi: 10.1016/j.smim.2011.08.013. Epub 2011 Sep 22. No abstract available.
- Background: PubMed 15616214Sayegh MH, Carpenter CB. Transplantation 50 years later--progress, challenges, and promises. N Engl J Med. 2004 Dec 23;351(26):2761-6. doi: 10.1056/NEJMon043418. No abstract available.
- Background: PubMed 24319282Azzi JR, Sayegh MH, Mallat SG. Calcineurin inhibitors: 40 years later, can't live without .. J Immunol. 2013 Dec 15;191(12):5785-91. doi: 10.4049/jimmunol.1390055.
- Background: PubMed 21117245Selzner N, Grant DR, Shalev I, Levy GA. The immunosuppressive pipeline: meeting unmet needs in liver transplantation. Liver Transpl. 2010 Dec;16(12):1359-72. doi: 10.1002/lt.22193.
- Background: PubMed 24685370Kaplan B, Qazi Y, Wellen JR. Strategies for the management of adverse events associated with mTOR inhibitors. Transplant Rev (Orlando). 2014 Jul;28(3):126-33. doi: 10.1016/j.trre.2014.03.002. Epub 2014 Mar 12.
- Background: PubMed 24748543Gotthardt DN, Bruns H, Weiss KH, Schemmer P. Current strategies for immunosuppression following liver transplantation. Langenbecks Arch Surg. 2014 Dec;399(8):981-8. doi: 10.1007/s00423-014-1191-9. Epub 2014 Apr 20.
- Background: PubMed 24384815Sachs DH, Kawai T, Sykes M. Induction of tolerance through mixed chimerism. Cold Spring Harb Perspect Med. 2014 Jan 1;4(1):a015529. doi: 10.1101/cshperspect.a015529.
- Background: PubMed 23656665Kawai T, Sachs DH, Sykes M, Cosimi AB; Immune Tolerance Network. HLA-mismatched renal transplantation without maintenance immunosuppression. N Engl J Med. 2013 May 9;368(19):1850-2. doi: 10.1056/NEJMc1213779. No abstract available.
- Background: PubMed 22162188Xie L, Ichimaru N, Morita M, Chen J, Zhu P, Wang J, Urbanellis P, Shalev I, Nagao S, Sugioka A, Zhong L, Nonomura N, Takahara S, Levy GA, Li XK. Identification of a novel biomarker gene set with sensitivity and specificity for distinguishing between allograft rejection and tolerance. Liver Transpl. 2012 Apr;18(4):444-54. doi: 10.1002/lt.22480.
- Derived from this study: PubMed 34049362Chruscinski A, Juvet S, Moshkelgosha S, Renner E, Lilly L, Selzner N, Bredeson C, Grant D, Adeyi O, Fischer S, Demetris AJ, Zhang J, Epstein M, Macarthur M, Clement AM, Khalili K, Allan D, Altouri S, Bence-Bruckler I, Cattral M, Fulcher J, Galvin Z, Ghanekar A, Greig P, Huebsch L, Humar A, Kew A, Kekre N, Kim TK, McDiarmid S, Martin L, McGilvray I, Sabloff M, Sapisochin G, Selzner M, Smith R, Tinckam K, Yi TJ, Levy G, Atkins H. Autologous Hematopoietic Stem Cell Transplantation for Liver Transplant Recipients With Recurrent Primary Sclerosing Cholangitis: A Pilot Study. Transplantation. 2022 Mar 1;106(3):562-574. doi: 10.1097/TP.0000000000003829.
Related trials for Complication of Transplanted Organ, Nos
Active, not recruitingPhase 1
Trial of Regulatory T-cells Plus Low-Dose Interleukin-2 for Steroid-Refractory Chronic Graft-versus-Host-Disease
Chronic Graft Versus Host Disease, Chronic GVHD
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.
Gary A Levy, MD FRCP AGAF principal investigator
University of Toronto Transplant Institute - Multi Organ Transplant Program
Harold L Atkins, MD FRCP(C) principal investigator
Ottawa Hospital Research Institute
2 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 Autologous Hematopoietic Stem Cell Transplant in people with Complication of Transplanted Organ, Nos, run by Gary A Levy, O. Ont. MD. FRCP AGAF at 2 sites in Canada.
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 4 inclusion and 10 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: finished, as of the sponsor's last update on 28 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Canada. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Number of patients who develop tolerance post autologous HSCT (time frame: 24 months post HSCT). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02549586, which also lists 10 linked publications.
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 28 Apr 2021.