Allografted With Myeloablative ConditioningNCT01520623
Complement and Graft-versus-host Disease
A study testing Serum concentration /Serum inflammatory in people with Allografted With Myeloablative Conditioning, run by Assistance Publique - Hôpitaux de Paris at 3 sites in France.
Trial summary
Allogeneic haematopoietic stem cell transplantation (HSCT) often remains the only curative treatment for haematological malignancies. The anti-leukaemic effect of allogeneic HSCT, called the GvL (Graf-versus-Leukemia) effect, is often associated to the development of an immune response against healthy recipient cells… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Allografted With Myeloablative Conditioning
- What is being tested?
- Serum concentration /Serum inflammatory
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 3 sitesFrance
- Study dates
- Apr 2010to Jul 2013
- Phase
- Interventional
- 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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Allografted patients with myeloablative conditioning for an haematological malignancy
- Age > 18 years old and < 65 years.
- The patient must have access to social insurance according to local regulations.
- Patient must give a written informed consent (personally signed and dated) before completing any study related procedure
Who cannot join
- Age < 18 years old and > 65 years
- Patient with active infection HIV, HTLV1, Hepatite B ou C
- Uncontrolled infection(s), (i.e. documented bacterial, parasitical, or fungal infection).
- Patient with lupus
- Patient with transaminases > 5N, TP<30% with Facteur V < 30% before allogreffe
- Creatinine clearance < 50ml/min
- Absence of any psychological condition potentially hampering signing informed consent
- Patient refused to sign informed consent
Trial locations and contacts
3 sites in 1 country.
France3 sites
Saint Louis Hospital
Paris, 75010
Saint Antoine Hospital
Paris, 75012
Necker Hospital
Paris, 75
About this study
Official title: Role of Complement System in Human Allogeneic Haematopoietic Stem Cell Transplantation
Allogeneic haematopoietic stem cell transplantation (HSCT) often remains the only curative treatment for haematological malignancies. The anti-leukaemic effect of allogeneic HSCT, called the GvL (Graf-versus-Leukemia) effect, is often associated to the development of an immune response against healthy recipient cells leading to a graft-versus-host disease (GvHD) in 20 to 70% of allogeneic HSCT. Acute GvHD, that usually targets the skin, the gastrointestinal (GI) tract and the liver, is an important cause of morbidity and mortality after allogeneic HSCT, particularly in the case of GI GvHD. The main goal of the research in the field of allogeneic HSCT is to determine strategies that could decrease the risk of GvHD without affecting the GvL effect. According to GVHD experimental models, it is likely that GvL but not GvHD may occur in the absence of inflammatory signals induced by the transplant-associated conditioning. Based on this hypothesis, we have chosen to analyse the role of Complement system in patients who received allogeneic HSCT. Indeed, Complement system is a major actor of inflammation and in the generation of tissue destruction, both of which are involved in the physiopathology of GVHD. Furthermore, it might be a potential target of some available inhibitory drugs (purified C1-Inhibitor, anti-C5 antibodies) in a preventive or curative manner in such patients. Preliminary data obtained from 34 allografted patients in our institution suggest that Complement activation by the classical pathway is correlated to the occurrence of GI GVHD. The goal of our current project, in order to confirm these preliminary results in a larger series, is to explore Complement system activation in patients who received allogeneic HSCT in three Adult Hematology departments in Paris fot two years and to correlate the biological results to the clinical events occurring after HSCT.
Read the detailed description
The study will be performed in allografted patients with myeloablative conditioning for an haematological malignancy from 3 adult transplant units.
Patients will be followed for at least12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze:
- serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59.
- serum inflammatory cytokine levels
In addition, patients with clinical signs of gut GVHD will be explored by gastrointestinal endoscopy to perform gut biopsies. C5b9 deposure will be then analyzed by immunohistochemistry on GVHD lesions.
Activation of complement system will be defined by a decrease of complement factor levels of 50% and values under lower physiological limits. The clinical evolution and the inflammatory cytokine profile of patients with such an activation profile will be compared to that of those without complement activation.
A data base containing biological and clinical data will be established. Biological results will be correlated to post-transplant clinical events, in particular the occurrence of gut GVHD but also non relapse mortality and overall survival by adapted statistical tests (comparison of percentages by Chi-2 of Pearson, comparison of survival curves by logrank, multivariate analysis by logistic regression test or cox model).
The number of required patients will be established by comparison of the percentage of gut GVHD in the patients with or without complement activation. Based on our preliminary results, we hypothesize that 2/3 patients will not have complement activation among whose 20% will develop acute gut GVHD. We expect an increase of acute gut GVHD up to 60% of the patients with complement activation that would represent 1/3 of the cohort.
With a bilateral alpha risk of 5% and a power of 80%, the number of required patients is 23 in the activated group and 46 in the non activated group, thus a total of 69 patients.
- Being tested
- Serum concentration /Serum inflammatory
- Keywords
- Allogeneic haematopoietic stem cell transplantation (HSCT), Myeloablative conditioning for an haematological malignancy, Graft-versus-host disease (GvHD), Activation of complement system, Gut GVHD
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Serum concentration /Serum inflammatoryOther
Allografted patients with myeloablative conditioning for an haematological malignancy. Patients will be followed for at least 12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze the serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59 and the serum inflammatory cytokine levels
| Study group | Role | What they receive |
|---|---|---|
| Allografted patients | Other | Allografted patients with myeloablative conditioning for an haematological malignancy. Patients will be followed for at least 12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze the serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59 and the serum inflammatory cytokine levels Receives: Other: Serum concentration /Serum inflammatory |
What is being measured
The primary outcome is the main question the study is designed to answer.
Activation of the complement system and the development of acute gut GvHD
Assessment of the activation of the complement system after human allogeneic stem cell transplantation and of its potential correlation with the development of acute gut GvHD
Measured: 12 weeks
Secondary outcomes (2)
Overall survival
Overall Survival at 3, 6, 9 , 12 and 24-month Post HCST
Measured: 2 years
Overall survival without relapse
Relapse
Measured: 2 Years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2012 (estimated)
- Study startApr 2010
- Primary completionFeb 2013
- Study completionJul 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Allografted With Myeloablative ConditioningStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 N/A
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Clinical haematology and BMT unit,Necker Hospital, Paris, Clinical haematology and BMT unit,Saint Antoine Hospital, Paris, Clinical haematology and BMT unit,Saint Louis Hospital,Paris, European Georges Pompidou Hospital, URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Marie-Thérèse RUBIO, MD, PhD, Principal investigator, Saint Antoine Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01520623
- Sponsor's protocol ID
- CRC 08025
- Status last verified
- Mar 2026
- First submitted
- 26 Dec 2011
- Last updated
- 5 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Rubio MT, Bouillie M, Bouazza N, Coman T, Trebeden-Negre H, Gomez A, Suarez F, Sibon D, Brignier A, Paubelle E, Nguyen-Khoc S, Cavazzana M, Lantz O, Mohty M, Urien S, Hermine O. Pre-transplant donor CD4- invariant NKT cell expansion capacity predicts the occurrence of acute graft-versus-host disease. Leukemia. 2017 Apr;31(4):903-912. doi: 10.1038/leu.2016.281. Epub 2016 Oct 14.
PubMed 27740636Notarantonio AB, D'aveni-Piney M, Pagliuca S, Ashraf Y, Galimard JE, Xhaard A, Marcais A, Suarez F, Brissot E, Feugier P, Urien S, Bouazza N, Jacquelin S, Meatchi T, Bruneval P, Fremeaux-Bacchi V, Peffault De Latour R, Hermine O, Durey-Dragon MA, Rubio MT. Systemic complement activation influences outcomes after allogeneic hematopoietic cell transplantation: A prospective French multicenter trial. Am J Hematol. 2023 Oct;98(10):1559-1570. doi: 10.1002/ajh.27030. Epub 2023 Jul 22.
PubMed 37483161
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Complement System in Human Allogeneic Haematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT01520623
- Acronym / sponsor ID
- CRC 08025
- Keywords
- Allogeneic haematopoietic stem cell transplantation (HSCT), Myeloablative conditioning for an haematological malignancy, Graft-versus-host disease (GvHD), Activation of complement system, Gut GVHD
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Clinical haematology and BMT unit,Necker Hospital, Paris, Clinical haematology and BMT unit,Saint Antoine Hospital, Paris, Clinical haematology and BMT unit,Saint Louis Hospital,Paris, European Georges Pompidou Hospital, URC-CIC Paris Descartes Necker Cochin
- Study start
- Apr 2010
- Primary completion
- Feb 2013 (actual)
- Study completion
- Jul 2013 (actual)
- First posted
- 30 Jan 2012 (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.
Allogeneic haematopoietic stem cell transplantation (HSCT) often remains the only curative treatment for haematological malignancies. The anti-leukaemic effect of allogeneic HSCT, called the GvL (Graf-versus-Leukemia) effect, is often associated to the development of an immune response against healthy recipient cells leading to a graft-versus-host disease (GvHD) in 20 to 70% of allogeneic HSCT. Acute GvHD, that usually targets the skin, the gastrointestinal (GI) tract and the liver, is an important cause of morbidity and mortality after allogeneic HSCT, particularly in the case of GI GvHD. The main goal of the research in the field of allogeneic HSCT is to determine strategies that could decrease the risk of GvHD without affecting the GvL effect. According to GVHD experimental models, it is likely that GvL but not GvHD may occur in the absence of inflammatory signals induced by the transplant-associated conditioning. Based on this hypothesis, we have chosen to analyse the role of Complement system in patients who received allogeneic HSCT. Indeed, Complement system is a major actor of inflammation and in the generation of tissue destruction, both of which are involved in the physiopathology of GVHD. Furthermore, it might be a potential target of some available inhibitory drugs (purified C1-Inhibitor, anti-C5 antibodies) in a preventive or curative manner in such patients. Preliminary data obtained from 34 allografted patients in our institution suggest that Complement activation by the classical pathway is correlated to the occurrence of GI GVHD. The goal of our current project, in order to confirm these preliminary results in a larger series, is to explore Complement system activation in patients who received allogeneic HSCT in three Adult Hematology departments in Paris fot two years and to correlate the biological results to the clinical events occurring after HSCT.
Detailed description
The study will be performed in allografted patients with myeloablative conditioning for an haematological malignancy from 3 adult transplant units.
Patients will be followed for at least12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze:
- serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59.
- serum inflammatory cytokine levels
In addition, patients with clinical signs of gut GVHD will be explored by gastrointestinal endoscopy to perform gut biopsies. C5b9 deposure will be then analyzed by immunohistochemistry on GVHD lesions.
Activation of complement system will be defined by a decrease of complement factor levels of 50% and values under lower physiological limits. The clinical evolution and the inflammatory cytokine profile of patients with such an activation profile will be compared to that of those without complement activation.
A data base containing biological and clinical data will be established. Biological results will be correlated to post-transplant clinical events, in particular the occurrence of gut GVHD but also non relapse mortality and overall survival by adapted statistical tests (comparison of percentages by Chi-2 of Pearson, comparison of survival curves by logrank, multivariate analysis by logistic regression test or cox model).
The number of required patients will be established by comparison of the percentage of gut GVHD in the patients with or without complement activation. Based on our preliminary results, we hypothesize that 2/3 patients will not have complement activation among whose 20% will develop acute gut GVHD. We expect an increase of acute gut GVHD up to 60% of the patients with complement activation that would represent 1/3 of the cohort.
With a bilateral alpha risk of 5% and a power of 80%, the number of required patients is 23 in the activated group and 46 in the non activated group, thus a total of 69 patients.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Allografted patients | Other | Other: Serum concentration /Serum inflammatoryAllografted patients with myeloablative conditioning for an haematological malignancy. Patients will be followed for at least 12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze the serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59 and the serum inflammatory cytokine levels |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Allografted patients with myeloablative conditioning for an haematological malignancy
- Age > 18 years old and < 65 years.
- The patient must have access to social insurance according to local regulations.
- Patient must give a written informed consent (personally signed and dated) before completing any study related procedure
Key exclusion criteria (8 listed)
- Age < 18 years old and > 65 years
- Patient with active infection HIV, HTLV1, Hepatite B ou C
- Uncontrolled infection(s), (i.e. documented bacterial, parasitical, or fungal infection).
- Patient with lupus
- Patient with transaminases > 5N, TP<30% with Facteur V < 30% before allogreffe
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 |
|---|---|---|
| Serum concentration /Serum inflammatoryAllografted patients with myeloablative conditioning for an haematological malignancy. Patients will be followed for at least 12 months after transplantation and blood samples drawn before conditioning and once a week for 12 weeks after transplantation to analyze the serum concentration of Complement factors (C3, C4, B factor), Complement regulatory proteins (C1-inhibitor, I and H Factors) and analysis of the surface expression of Complement regulatory molecules such as CD46, CD55 and CD59 and the serum inflammatory cytokine levels | Other | Allografted patients |
Primary outcome
Activation of the complement system and the development of acute gut GvHD
Time frame: 12 weeks
Assessment of the activation of the complement system after human allogeneic stem cell transplantation and of its potential correlation with the development of acute gut GvHD
2 secondary outcomes
Overall survival
Time frame: 2 years
Overall survival without relapse
Time frame: 2 Years
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 5 Mar 2026.
- Results publication: PubMed 27740636Rubio MT, Bouillie M, Bouazza N, Coman T, Trebeden-Negre H, Gomez A, Suarez F, Sibon D, Brignier A, Paubelle E, Nguyen-Khoc S, Cavazzana M, Lantz O, Mohty M, Urien S, Hermine O. Pre-transplant donor CD4- invariant NKT cell expansion capacity predicts the occurrence of acute graft-versus-host disease. Leukemia. 2017 Apr;31(4):903-912. doi: 10.1038/leu.2016.281. Epub 2016 Oct 14.
- Derived from this study: PubMed 37483161Notarantonio AB, D'aveni-Piney M, Pagliuca S, Ashraf Y, Galimard JE, Xhaard A, Marcais A, Suarez F, Brissot E, Feugier P, Urien S, Bouazza N, Jacquelin S, Meatchi T, Bruneval P, Fremeaux-Bacchi V, Peffault De Latour R, Hermine O, Durey-Dragon MA, Rubio MT. Systemic complement activation influences outcomes after allogeneic hematopoietic cell transplantation: A prospective French multicenter trial. Am J Hematol. 2023 Oct;98(10):1559-1570. doi: 10.1002/ajh.27030. Epub 2023 Jul 22.
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.
Marie-Thérèse RUBIO, MD, PhD principal investigator
Saint Antoine Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Serum concentration /Serum inflammatory in people with Allografted With Myeloablative Conditioning, run by Assistance Publique - Hôpitaux de Paris at 3 sites in France.
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 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Mar 2026. 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 France. 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.
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?
Activation of the complement system and the development of acute gut GvHD (time frame: 12 weeks). 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 NCT01520623, which also lists 2 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 5 Mar 2026.