Hematologic DiseasesPhase 2HaploRescueNCT05126186
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Rescuing Patients With Graft Failure
A Phase 2 study testing haplo-SCT with PTCy in people with Hematologic Diseases and Graft Failure, run by Assistance Publique - Hôpitaux de Paris.
Trial summary
Prognosis of patients with graft failure is dismal, and re-transplantation is the sole option for long-term survival. Currently, there is no consensus concerning therapeutic options in patients with primary or secondary (within the 60 days post-transplantation) graft failure and finding a new donor within an… Read more about this study
- Who is it for?
- Ages 3 to 70 years
- Conditions the study is for
- Hematologic Diseases, Graft Failure, Allogeneic Hematopoietic Stem Cell Transplantation
- What is being tested?
- haplo-SCT with PTCy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- No sites listed yet
- Study dates
- 1 Dec 2021to 1 Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Aged from 3 to 70 years
- All hematological diseases
- Suffering from primary or secondary (within the 60 days post-transplantation) graft failure after a 1st allo-SCT
- With usual criteria for allo-SCT:
- ECOG ≤ 2
- No severe and uncontrolled infection
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- Cardiac function compatible with high dose of cyclophosphamide
- Adequate organ function: ASAT and ALAT ≤ 2.5N, total bilirubin ≤ 2N, creatinine clearance ≥30ml / min
- With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
- Absence of donor specific antibody (DSA) detected in the patient with a MFI ≥ 1500 (antibodies directed towards the distinct haplotype between donor and recipient)
- With health insurance coverage (bénéficiaire ou ayant droit).
- Understand informed consent or optimal treatment and follow-up.
- Contraception methods must be prescribed during all the duration of the research. Women and men of childbearing age must use contraceptive methods within 12 months and 6 months after the last dose of cyclophosphamide, respectively.
- Having signed a written informed consent (2 parents for patients aged less than 18)
Who cannot join
- Aged< 3 years old and >70 years old
- With uncontrolled infection
- With Seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and associated hepatic cytolysis
- Yellow fever vaccine within 2 months before transplantation
- Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
- Uncontrolled coronary insufficiency, recent myocardial infarction <6 month, current manifestations of heart failure, uncontrolled cardiac rhythm disorders, ventricular ejection fraction <50%
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- Heart failure according to NYHA (II or more)
- Preexisting acute hemorrhagic cystitis
- Renal failure with creatinine clearance < 30ml / min
- Urinary tract obstruction
- Pregnant (β-HCG positive) or breast-feeding
- Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up
- COVID vaccination or recent COVID disease <3 months
- Tutorship or curatorship
- Contraindications to treatments used during the research
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Rescuing Patients With Graft Failure: a Phase II Study
Prognosis of patients with graft failure is dismal, and re-transplantation is the sole option for long-term survival. Currently, there is no consensus concerning therapeutic options in patients with primary or secondary (within the 60 days post-transplantation) graft failure and finding a new donor within an acceptable delay is challenging. Literature is poor on the subject while the overall survival of such patients is about 30% at 1 year. This situation thus represents today a very challenging unmet medical need.
Recently, haploidentical (haplo) related donor Stem Cell Transplantation (haplo-SCT) have improved dramatically outcomes using T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy, which targets alloreactive T cells generated early after an HLA-mismatched transplant, sparing regulatory T cells and leaving unaffected the non-dividing hematopoietic stem cells) and standard post-transplant immune suppression with a calcineurin inhibitor (CNI) and mycophenolate mofetil. Our group re-transplanted a patient who experienced two consecutive graft failures and was successfully managed through a third haplo-SCT from her son using PTCy. We then retrospectively collected and analyzed data from 26 primary graft failure patients transplanted between 2011 and 2017 in 15 centers on behalf of French Society for Stem Cell Transplantation and Cell Therapy (SFGM-TC). The study population consisted mainly of patients with primary or secondary (within the 60 days post-transplantation) graft failure who underwent haplo-SCT and received PTCy as graft-versus-host-disease prophylaxis. The 1-year overall survival was about 60% suggesting that this approach might be a valid option in this particular poor clinical situation but now need validation through a phase II multicenter, national, prospective cohort study.
- Being tested
- haplo-SCT with PTCy
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
haplo-SCT with PTCyOther
Conditioning regimen Fludarabine (30mg/m2/day from day -6 to day -4), Cyclophosphamide (14.5 mg/kg/day at day -6 and day -5) except for patients who received a total dose of Cyclophosphamide >100mg/Kg during the first Bone Marrow Transplantation Total Body Irradiation (2 Gray on day -1).
Source of stem cell source Peripheral blood stem cell Minimal target dose of 4.106 CD34+ cells/kg of recipient
GvHD prophylaxis Cyclophosphamide 50 mg/Kg/day at D+3 and D+4 Ciclosporine from day+5 (residual 200 à 300ng/l) Mycophenolate mofetyl at 15mg/Kg x2/day from day+5
Prevention of EBV reactivation Rituximab : 150mg/m2 intravenously at Day+5 post Haplo-SCT Each infusion of Rituximab will be preceded by administration of anti-pyretic and an antihistaminic.
| Study group | Role | What they receive |
|---|---|---|
| haplo-SCT with PTCy | Experimental | haploidentical (haplo) related donor Stem Cell Transplantation (haplo-SCT) with administration of post-transplantation cyclophosphamide (PTCy, which targets alloreactive T cells generated early after an HLA-mismatched transplant, sparing regulatory T cells and leaving unaffected the non-dividing hematopoietic stem cells) Receives: Other: haplo-SCT with PTCy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival
Measured: at one year
Secondary outcomes (54)
Graft failure incidence
Measured: at 3 months
Neutrophils engraftment
3 consecutive days with neutrophiles >0.5 G/L
Measured: at day 100
Platelets engraftment
7 consecutive days with platelets >20 G/L
Measured: at day 100
Absolute numbers of neutrophils
Measured: at 1 month
Absolute numbers of neutrophils
Measured: at 2 months
Absolute numbers of neutrophils
Measured: at 3 months
Absolute numbers of neutrophils
Measured: at 6 months
Absolute numbers of neutrophils
Measured: at 12 months
Absolute numbers of neutrophils
Measured: through study completion, an average of 6 months
Absolute number of platelets
Measured: at one month
Absolute number of platelets
Measured: at 2 months
Absolute number of platelets
Measured: at 3 months
Absolute number of platelets
Measured: at 6 months
Absolute number of platelets
Measured: at 12 months
Absolute number of platelets
Measured: through study completion, an average of 6 months
Incidence of use of growth factors for poor hematopoietic reconstitution
Measured: at 3 months
Acute GvHD incidence
Measured: at 3 months
Chronic GvHD incidence
Measured: at 24 months
Relapse incidence
Measured: at 12 months
Relapse incidence
Measured: at 24 months
Progression free survival
Measured: at 12 months
Progression free survival
Measured: at 24 months
Incidence of CMV infection
Measured: at 12 months
Incidence of EBV infection
Measured: at 12 months
Incidence of severe infections
Severe infections are defined as CTAE grade of 3 or 4
Measured: at 3 months
Incidence of severe infections
Severe infections are defined as CTAE grade of 3 or 4
Measured: at 6 months
Incidence of severe infections
Severe infections are defined as CTAE grade of 3 or 4
Measured: at 12 months
Incidence of severe infections
Severe infections are defined as CTAE grade of 3 or 4
Measured: at 24 months
Incidence of veino-occlusive disease (VOD)
Measured: at 3 months
Severity of veino-occlusive disease (VOD)
Measured: at 3 months
Non-relapse mortality
Measured: at 24 months
Incidence of cardiac toxicities
Measured: at 12 months
Overall survival
Measured: at 24 months
Interval between first allo-SCT and rescue haplo-SCT
Measured: at 60 days
Quality of life for adults
Quality of life will be assessed for adults using "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" EORTC QLQ-C30-V3 questionnaire.The QLQ-C30 is composed of both multi-item scales and single-item measures. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.
Measured: at 3 months
Quality of life for adults
Quality of life will be assessed for adults using "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" EORTC QLQ-C30-V3 questionnaire.The QLQ-C30 is composed of both multi-item scales and single-item measures. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.
Measured: at 6 months
Quality of life for adults
Quality of life will be assessed for adults using "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" EORTC QLQ-C30-V3 questionnaire.The QLQ-C30 is composed of both multi-item scales and single-item measures. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.
Measured: at 12 months
Quality of life for adults
Quality of life will be assessed for adults using "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" EORTC QLQ-C30-V3 questionnaire.The QLQ-C30 is composed of both multi-item scales and single-item measures. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level.
Measured: at 24 months
Quality of life for minors
Quality of life will be assessed for minor using The Pediatric Quality of Life Inventory™ (PedsQL™) The 36-item PedsQL™ Family Impact Module is a parent-report instrument designed to assess the impact of pediatric chronic health conditions on parents and the family. It includes 6 subscales measuring parents' self-reported functioning. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Higher scores indicate better functioning
Measured: at 3 months
Quality of life for minors
Quality of life will be assessed for minor using The Pediatric Quality of Life Inventory™ (PedsQL™) The 36-item PedsQL™ Family Impact Module is a parent-report instrument designed to assess the impact of pediatric chronic health conditions on parents and the family. It includes 6 subscales measuring parents' self-reported functioning. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Higher scores indicate better functioning
Measured: at 6 months
Quality of life for minors
Quality of life will be assessed for minor using The Pediatric Quality of Life Inventory™ (PedsQL™) The 36-item PedsQL™ Family Impact Module is a parent-report instrument designed to assess the impact of pediatric chronic health conditions on parents and the family. It includes 6 subscales measuring parents' self-reported functioning. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Higher scores indicate better functioning
Measured: at 12 months
Quality of life for minors
Quality of life will be assessed for minor using The Pediatric Quality of Life Inventory™ (PedsQL™) The 36-item PedsQL™ Family Impact Module is a parent-report instrument designed to assess the impact of pediatric chronic health conditions on parents and the family. It includes 6 subscales measuring parents' self-reported functioning. The scale has five Likert response options, 'never', 'almost never', 'sometimes', 'often' and 'almost always' (corresponding to scores of 100, 75, 50, 25 and 0). Higher scores indicate better functioning
Measured: at 24 months
Proportion of patients with a donor chimerism of 90% or more
Measured: at 1 month
Proportion of patients with a donor chimerism of 90% or more
Measured: at 3 months
Proportion of patients with a donor chimerism of 90% or more
Measured: at 6 months
Proportion of patients with a donor chimerism of 90% or more
Measured: at 12 months
Immune reconstitution
Immune reconstitution will be defined by analyzing T, B, NK, regulatory T cell levels in the peripheral blood
Measured: at 3 months post-transplantation
Immune reconstitution
Immune reconstitution will be defined by analyzing T, B, NK, regulatory T cell levels in the peripheral blood
Measured: at 6 months post-transplantation
Immune reconstitution
Immune reconstitution will be defined by analyzing T, B, NK, regulatory T cell levels in the peripheral blood
Measured: at 12 months post-transplantation
Immune reconstitution
Immune reconstitution will be defined by analyzing T, B, NK, regulatory T cell levels in the peripheral blood
Measured: at 24 months post-transplantation
Iron overload estimation
Measured: at 3 months
Iron overload estimation
Measured: at 6 months
Iron overload estimation
Measured: at 12 months
Iron overload estimation
Measured: at 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2021
- Study start1 Dec 2021 (estimated)
- Primary completion1 Dec 2025 (estimated)
- Study completion1 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic DiseasesStudies 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 2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05126186
- Sponsor's protocol ID
- APHP200129
- Acronym
- HaploRescue
- Status last verified
- Nov 2021
- First submitted
- 8 Nov 2021
- Last updated
- 18 Nov 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide for Rescuing Patients With Graft Failure: a Phase II Study
- ClinicalTrials.gov ID
- NCT05126186
- Acronym / sponsor ID
- HaploRescue / APHP200129
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 35 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 1 Dec 2021 (estimated)
- Primary completion
- 1 Dec 2025 (estimated)
- Study completion
- 1 Dec 2026 (estimated)
- First posted
- 18 Nov 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Prognosis of patients with graft failure is dismal, and re-transplantation is the sole option for long-term survival. Currently, there is no consensus concerning therapeutic options in patients with primary or secondary (within the 60 days post-transplantation) graft failure and finding a new donor within an acceptable delay is challenging. Literature is poor on the subject while the overall survival of such patients is about 30% at 1 year. This situation thus represents today a very challenging unmet medical need.
Recently, haploidentical (haplo) related donor Stem Cell Transplantation (haplo-SCT) have improved dramatically outcomes using T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy, which targets alloreactive T cells generated early after an HLA-mismatched transplant, sparing regulatory T cells and leaving unaffected the non-dividing hematopoietic stem cells) and standard post-transplant immune suppression with a calcineurin inhibitor (CNI) and mycophenolate mofetil. Our group re-transplanted a patient who experienced two consecutive graft failures and was successfully managed through a third haplo-SCT from her son using PTCy. We then retrospectively collected and analyzed data from 26 primary graft failure patients transplanted between 2011 and 2017 in 15 centers on behalf of French Society for Stem Cell Transplantation and Cell Therapy (SFGM-TC). The study population consisted mainly of patients with primary or secondary (within the 60 days post-transplantation) graft failure who underwent haplo-SCT and received PTCy as graft-versus-host-disease prophylaxis. The 1-year overall survival was about 60% suggesting that this approach might be a valid option in this particular poor clinical situation but now need validation through a phase II multicenter, national, prospective cohort study.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| haplo-SCT with PTCy | Experimental | Other: haplo-SCT with PTCyhaploidentical (haplo) related donor Stem Cell Transplantation (haplo-SCT) with administration of post-transplantation cyclophosphamide (PTCy, which targets alloreactive T cells generated early after an HLA-mismatched transplant, sparing regulatory T cells and leaving unaffected the non-dividing hematopoietic stem cells) |
Who may qualify
- Ages
- 3 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- Aged from 3 to 70 years
- All hematological diseases
- Suffering from primary or secondary (within the 60 days post-transplantation) graft failure after a 1st allo-SCT
- With usual criteria for allo-SCT:
- With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
Key exclusion criteria (15 listed)
- Aged< 3 years old and >70 years old
- With uncontrolled infection
- With Seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and associated hepatic cytolysis
- Yellow fever vaccine within 2 months before transplantation
- Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
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 |
|---|---|---|
| haplo-SCT with PTCyConditioning regimen Fludarabine (30mg/m2/day from day -6 to day -4), Cyclophosphamide (14.5 mg/kg/day at day -6 and day -5) except for patients who received a total dose of Cyclophosphamide >100mg/Kg during the first Bone Marrow Transplantation Total Body Irradiation (2 Gray on day -1). Source of stem cell source Peripheral blood stem cell Minimal target dose of 4.106 CD34+ cells/kg of recipient GvHD prophylaxis Cyclophosphamide 50 mg/Kg/day at D+3 and D+4 Ciclosporine from day+5 (residual 200 à 300ng/l) Mycophenolate mofetyl at 15mg/Kg x2/day from day+5 Prevention of EBV reactivation Rituximab : 150mg/m2 intravenously at Day+5 post Haplo-SCT Each infusion of Rituximab will be preceded by administration of anti-pyretic and an antihistaminic. | Other | haplo-SCT with PTCy |
Primary outcome
Overall Survival
Time frame: at one year
54 secondary outcomes
Graft failure incidence
Time frame: at 3 months
Neutrophils engraftment
Time frame: at day 100
Platelets engraftment
Time frame: at day 100
Absolute numbers of neutrophils
Time frame: at 1 month
Absolute numbers of neutrophils
Time frame: at 2 months
Absolute numbers of neutrophils
Time frame: at 3 months
Absolute numbers of neutrophils
Time frame: at 6 months
Absolute numbers of neutrophils
Time frame: at 12 months
Absolute numbers of neutrophils
Time frame: through study completion, an average of 6 months
Absolute number of platelets
Time frame: at one month
Absolute number of platelets
Time frame: at 2 months
Absolute number of platelets
Time frame: at 3 months
Absolute number of platelets
Time frame: at 6 months
Absolute number of platelets
Time frame: at 12 months
Absolute number of platelets
Time frame: through study completion, an average of 6 months
Incidence of use of growth factors for poor hematopoietic reconstitution
Time frame: at 3 months
Acute GvHD incidence
Time frame: at 3 months
Chronic GvHD incidence
Time frame: at 24 months
Relapse incidence
Time frame: at 12 months
Relapse incidence
Time frame: at 24 months
Progression free survival
Time frame: at 12 months
Progression free survival
Time frame: at 24 months
Incidence of CMV infection
Time frame: at 12 months
Incidence of EBV infection
Time frame: at 12 months
Incidence of severe infections
Time frame: at 3 months
Incidence of severe infections
Time frame: at 6 months
Incidence of severe infections
Time frame: at 12 months
Incidence of severe infections
Time frame: at 24 months
Incidence of veino-occlusive disease (VOD)
Time frame: at 3 months
Severity of veino-occlusive disease (VOD)
Time frame: at 3 months
Non-relapse mortality
Time frame: at 24 months
Incidence of cardiac toxicities
Time frame: at 12 months
Overall survival
Time frame: at 24 months
Interval between first allo-SCT and rescue haplo-SCT
Time frame: at 60 days
Quality of life for adults
Time frame: at 3 months
Quality of life for adults
Time frame: at 6 months
Quality of life for adults
Time frame: at 12 months
Quality of life for adults
Time frame: at 24 months
Quality of life for minors
Time frame: at 3 months
Quality of life for minors
Time frame: at 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2021.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing haplo-SCT with PTCy in people with Hematologic Diseases and Graft Failure, run by Assistance Publique - Hôpitaux de Paris.
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 14 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: opening soon, as of the sponsor's last update on 18 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Régis Peffault de Latour (+33142385073, regis.peffaultdelatour@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (time frame: at one year). 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 18 Nov 2021.