GVHDPhase 3NCT01295710
Study of US-ATG-F to Prevent Chronic Graft Versus Host Disease (GVHD)
A Phase 3 study testing US-ATG-F in people with GVHD and Adult Acute Myeloid Leukemia, run by Neovii Biotech at 28 sites in 2 countries.
Trial summary
The study objective is to compare the efficacy and safety of US-ATG-F as a supplement to standard of care prophylaxis versus standard of care prophylaxis alone in moderate to severe chronic GVHD-free survival. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- GVHD, Adult Acute Myeloid Leukemia, Adult Acute Lymphoid Leukemia
- What is being tested?
- US-ATG-F
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 260 participants
- Where
- 28 sitesin 2 countries
- Study dates
- 10 Oct 2011to 15 Oct 2015
- Phase
- Phase 3
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients designated to undergo allogeneic peripheral blood or bone marrow stem cell transplantation following the diagnosis of one of the primary diseases in early or intermediate disease status (i.e., acute myeloid leukemia, acute lymphoid leukemia, and myelodysplastic syndrome)
- Patients with an unrelated HLA-A,-B, -C and -DRBI matched donor
- Patients with a Karnofsky Performance Score ≥ 70%
Who cannot join
- Clinically significant concomitant diseases (i.e., cardiac, pulmonary, renal and CNS)
- Bacterial, viral, or fungal infections
- Known positive for Hepatitis B surfaces antigen, or Hepatitis C antibody, or who have been tested positive for HIV
- Patients with any concurrent malignancy. Cancer treated with curative intent < 5 years previously will not be allowed except for patients with resected basal cell carcinoma or treated cervical carcinoma in situ
- Known contraindications to the administration of rabbit immunoglobulin antibodies
- Hypersensitivity to methylprednisolone, tacrolimus, methotrexate or any excipients contains in these products
Trial locations and contacts
28 sites in 2 countries.
28 sites in 2 countries
United States26 sites
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Massachusetts General Hospital
Boston, Massachusetts, 02215
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27599
University of Chicago Medical Center
Chicago, Illinois, 60637
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
City of Hope
Duarte, California, 91019
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida Shands Cancer Center
Gainesville, Florida, 32610
Penn State Hershey Cancer Institute
Hershey, Pennsylvania, 17033
Loyola University Medical Center
Maywood, Illinois, 60153
Vanderbilt University Medical Center, Vanderbilt Ingram Cancer Center
Nashville, Tennessee, 37232
Tulane University Health Sciences Center
New Orleans, Louisiana, 70112
Weill Cornell Medical Center
New York, New York, 10065
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Abramson Cancer Center of the University at Perlman Center for Advanced Medicine
Philadelphia, Pennsylvania, 19104
Oregon Health and Science University
Portland, Oregon, 97239
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah School of Medicine
Salt Lake City, Utah, 84132
Texas Transplant Physician's Group
San Antonio, Texas, 78229
VA Puget Sound Healthcare System
Seattle, Washington, 98108
Fred Hutchinson Cancer Research Center
Seattle, Washington, 98109
Washington University Medical Center
St Louis, Missouri, 63110
Stanford University Medical Center, BMT
Stanford, California, 94305
Moffitt Cancer Center
Tampa, Florida, 33612
University of Kansas Medical Center
Westwood, Kansas, 66205
Australia2 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Royal Melbourne Hospital
Parkville, Victoria, 03050
About this study
Official title: Phase 3 Study of US-ATG-F to Prevent Moderate to Severe Chronic GVHD in Adult Acute Myeloid Leukemia, Acute Lymphoid Leukemia, and Myelodysplastic Syndrome Patients After Allogeneic Stem Cell Transplantation From Unrelated Donors
The study objective is to compare the efficacy and safety of US-ATG-F as a supplement to standard of care prophylaxis versus standard of care prophylaxis alone in moderate to severe chronic GVHD-free survival.
Read the detailed description
This study is randomized, prospective, double-blind, placebo-controlled, phase 3 study evaluating the prevention of moderate to severe chronic GVHD in patients undergoing bone marrow or peripheral blood stem cell transplantation from matched, unrelated donors for acute leukemia and myelodysplastic syndrome during the first year after transplant.
Patients meeting all the inclusion and none of the exclusion criteria will be randomized (1:1). All patients will receive premedication and study drug 3 days prior to transplantation.
- Conditions
- GVHD, Adult Acute Myeloid Leukemia, Adult Acute Lymphoid Leukemia, Myelodysplastic Syndrome
- Being tested
- US-ATG-F, Placebo
- Keywords
- adult acute myeloid leukemia, adult acute lymphoid leukemia, adult myelodysplastic syndrome, allogenic stem cell transplantation, unrelated donor, GVHD, US-ATG-F (Anti-human-T-lymphocyte Immune Globulin, Rabbit)
- Medical subject headings
- Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Anemia, Refractory, with Excess of Blasts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
US-ATG-FBiological
Also called Anti-human-T-lymphocyte Immune Globulin, Rabbit
20 mg/kg body weight per day, diluted in 250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation
PlaceboBiological
250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation
| Study group | Role | What they receive |
|---|---|---|
| US-ATG-F | Active comparator | 20 mg/kg body weight per day, diluted in 250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation Receives: Biological: US-ATG-F |
| Placebo | Placebo comparator | 250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With First Occurrence of Moderate to Severe Chronic GVHD According to 2005 NIH Criteria as Determined by the Independent Endpoint Committee or Death From Any Cause After Allogeneic Stem Cell Transplantation
Participants with first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Independent Endpoint Committee or death from any cause after allogeneic stem cell transplantation, with a target of 124 total events of moderate or severe chronic GVHD, or death from any cause
Measured: Time from first study drug administration until the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Independent Endpoint Committee, or death from any cause, assessed up to 48 months
Secondary outcomes (11)
Overall Survival
Incidence of death from any cause
Measured: Time from first study drug administration until the occurrence of death from any cause, assessed up to 48 months
Number of Participants With Chronic GVHD Mild to Severe
Participants with the first occurrence of mild to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of mild to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Chronic GVHD Moderate to Severe
Participants with the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Chronic GVHD Severe
Participants with the first occurrence of severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade I-IV
Participants with the first occurrence of acute GVHD grade I-IV as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of acute GVHD grade I-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade II-IV
Participants with the first occurrence of acute GVHD grade II-IV as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of acute GVHD grade II-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade III-IV
Participants with the first occurrence of acute GVHD grade III-IV as determined by the Investigators, with death and re transplantation as competing risks
Measured: Time from first study drug administration until the first occurrence of acute GVHD grade III-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Relapse
Participants with relapse or disease recurrence, with death as competing risk
Measured: Time from first study drug administration until the occurrence of relapse, with death as competing risk, assessed up to 48 months
Disease-free Survival
Incidence of relapse or death
Measured: Time from first study drug administration until the occurrence of relapse or death, assessed up to 48 months
Number of Participants With Transplant Related Mortality
Participants with transplant related mortality
Measured: Time from first study drug administration until the occurrence of transplant related mortality, assessed up to 48 months
Systemic Immunosuppressive Medication for Treatment of Moderate to Severe Chronic GVHD
Participants who started on systemic immunosuppressive medicine for treatment of moderate to severe chronic GVHD as determined by the Investigator, with death and re-transplantation as competing risks
Measured: Time from first study drug administration until start of systemic immunosuppressive medicine for treatment of moderate to severe chronic GVHD as determined by the Investigator, with death and re-transplantation as competing risks, assessed up to 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Feb 2011 (estimated)
- Study start10 Oct 2011
- Primary completion15 Oct 2015
- Study completion15 Oct 2015
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GVHDStudies 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 3
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 260 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neovii Biotech
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Anne Kuan, Study director, Neovii Biotech
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01295710
- Sponsor's protocol ID
- IV-ATG-SCT-01
- Status last verified
- Mar 2019
- First submitted
- 9 Feb 2011
- Last updated
- 9 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Chakupurakal G, Freudenberger P, Skoetz N, Ahr H, Theurich S. Polyclonal anti-thymocyte globulins for the prophylaxis of graft-versus-host disease after allogeneic stem cell or bone marrow transplantation in adults. Cochrane Database Syst Rev. 2023 Jun 21;6(6):CD009159. doi: 10.1002/14651858.CD009159.pub3.
PubMed 37341189
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Study of US-ATG-F to Prevent Moderate to Severe Chronic GVHD in Adult Acute Myeloid Leukemia, Acute Lymphoid Leukemia, and Myelodysplastic Syndrome Patients After Allogeneic Stem Cell Transplantation From Unrelated Donors
- ClinicalTrials.gov ID
- NCT01295710
- Acronym / sponsor ID
- IV-ATG-SCT-01
- Conditions
- GVHD, Adult Acute Myeloid Leukemia, Adult Acute Lymphoid Leukemia, Myelodysplastic Syndrome
- Keywords
- adult acute myeloid leukemia, adult acute lymphoid leukemia, adult myelodysplastic syndrome, allogenic stem cell transplantation, unrelated donor, GVHD, US-ATG-F (Anti-human-T-lymphocyte Immune Globulin, Rabbit)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 260 participants (actual)
- Lead sponsor
- Neovii BiotechIndustry (pharma, biotech, devices)
- Study start
- 10 Oct 2011 (actual)
- Primary completion
- 15 Oct 2015 (actual)
- Study completion
- 15 Oct 2015 (actual)
- First posted
- 14 Feb 2011 (estimated)
- Results
- Posted 20 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study objective is to compare the efficacy and safety of US-ATG-F as a supplement to standard of care prophylaxis versus standard of care prophylaxis alone in moderate to severe chronic GVHD-free survival.
Detailed description
This study is randomized, prospective, double-blind, placebo-controlled, phase 3 study evaluating the prevention of moderate to severe chronic GVHD in patients undergoing bone marrow or peripheral blood stem cell transplantation from matched, unrelated donors for acute leukemia and myelodysplastic syndrome during the first year after transplant.
Patients meeting all the inclusion and none of the exclusion criteria will be randomized (1:1). All patients will receive premedication and study drug 3 days prior to transplantation.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| US-ATG-F | Active comparator | Biological: US-ATG-F20 mg/kg body weight per day, diluted in 250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation |
| Placebo | Placebo comparator | Biological: Placebo250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients designated to undergo allogeneic peripheral blood or bone marrow stem cell transplantation following the diagnosis of one of the primary diseases in early or intermediate disease status (i.e., acute myeloid leukemia, acute lymphoid leukemia, and myelodysplastic syndrome)
- Patients with an unrelated HLA-A,-B, -C and -DRBI matched donor
- Patients with a Karnofsky Performance Score ≥ 70%
Key exclusion criteria (6 listed)
- Clinically significant concomitant diseases (i.e., cardiac, pulmonary, renal and CNS)
- Bacterial, viral, or fungal infections
- Known positive for Hepatitis B surfaces antigen, or Hepatitis C antibody, or who have been tested positive for HIV
- Patients with any concurrent malignancy. Cancer treated with curative intent < 5 years previously will not be allowed except for patients with resected basal cell carcinoma or treated cervical carcinoma in situ
- Known contraindications to the administration of rabbit immunoglobulin antibodies
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 |
|---|---|---|
| US-ATG-FAlso: Anti-human-T-lymphocyte Immune Globulin, Rabbit20 mg/kg body weight per day, diluted in 250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation | Biological | US-ATG-F |
| Placebo250 mL normal saline, IV infusion over 6-16 hours 3 days prior to transplantation | Biological | Placebo |
Primary outcome
Number of Participants With First Occurrence of Moderate to Severe Chronic GVHD According to 2005 NIH Criteria as Determined by the Independent Endpoint Committee or Death From Any Cause After Allogeneic Stem Cell Transplantation
Time frame: Time from first study drug administration until the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Independent Endpoint Committee, or death from any cause, assessed up to 48 months
Participants with first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Independent Endpoint Committee or death from any cause after allogeneic stem cell transplantation, with a target of 124 total events of moderate or severe chronic GVHD, or death from any cause
11 secondary outcomes
Overall Survival
Time frame: Time from first study drug administration until the occurrence of death from any cause, assessed up to 48 months
Number of Participants With Chronic GVHD Mild to Severe
Time frame: Time from first study drug administration until the first occurrence of mild to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Chronic GVHD Moderate to Severe
Time frame: Time from first study drug administration until the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Chronic GVHD Severe
Time frame: Time from first study drug administration until the first occurrence of severe chronic GVHD according to 2005 NIH criteria as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade I-IV
Time frame: Time from first study drug administration until the first occurrence of acute GVHD grade I-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade II-IV
Time frame: Time from first study drug administration until the first occurrence of acute GVHD grade II-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Acute GVHD Grade III-IV
Time frame: Time from first study drug administration until the first occurrence of acute GVHD grade III-IV as determined by the Investigators, with death and re transplantation as competing risks, assessed up to 48 months
Number of Participants With Relapse
Time frame: Time from first study drug administration until the occurrence of relapse, with death as competing risk, assessed up to 48 months
Disease-free Survival
Time frame: Time from first study drug administration until the occurrence of relapse or death, assessed up to 48 months
Number of Participants With Transplant Related Mortality
Time frame: Time from first study drug administration until the occurrence of transplant related mortality, assessed up to 48 months
Systemic Immunosuppressive Medication for Treatment of Moderate to Severe Chronic GVHD
Time frame: Time from first study drug administration until start of systemic immunosuppressive medicine for treatment of moderate to severe chronic GVHD as determined by the Investigator, with death and re-transplantation as competing risks, assessed up to 48 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 37341189Chakupurakal G, Freudenberger P, Skoetz N, Ahr H, Theurich S. Polyclonal anti-thymocyte globulins for the prophylaxis of graft-versus-host disease after allogeneic stem cell or bone marrow transplantation in adults. Cochrane Database Syst Rev. 2023 Jun 21;6(6):CD009159. doi: 10.1002/14651858.CD009159.pub3.
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Graft vs Host Disease
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.
Anne Kuan study director
Neovii Biotech
28 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing US-ATG-F in people with GVHD and Adult Acute Myeloid Leukemia, run by Neovii Biotech at 28 sites in 2 countries.
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 3 inclusion and 6 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 9 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 28 sites in United States, Australia. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 20 Mar 2019). 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?
Number of Participants With First Occurrence of Moderate to Severe Chronic GVHD According to 2005 NIH Criteria as Determined by the Independent Endpoint Committee or Death From Any Cause After Allogeneic Stem Cell Transplantation (time frame: Time from first study drug administration until the first occurrence of moderate to severe chronic GVHD according to 2005 NIH criteria as determined by the Independent Endpoint Committee, or death from any cause, assessed up to 48 months). 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 NCT01295710, which also lists 1 linked publication.
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 9 Apr 2019.