Combined ImmunodeficienciesPhase 2CD45RANCT02737384
Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID)
A Phase 2 study testing Depletion in CD45RA graft donor in people with Combined Immunodeficiencies, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to evaluate selective depletion of naïve CD45RA+ T cells from allogenic peripheral blood stem cell graft in children transplanted for combined immune deficiency. Read more about this study
- Who is it for?
- Ages 12 months to 18 years
- Conditions the study is for
- Combined Immunodeficiencies
- What is being tested?
- Depletion in CD45RA graft donor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteFrance
- Study dates
- 14 Jun 2016to 26 Oct 2017
- 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 12 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patient from 12 months to 18 years
- Combined immunodeficiencies with known molecular diagnosis or if unknown, corresponding of p-CID study's definition
- Hematopoietic stem cell Transplantation planned with one of the following donors :
- sibling with 1 or 2 HLA antigens mismatch
- parent 10/10 or 9/10 identical
- unrelated donor: 10/10 or 9/10 identical
Show all 9Show fewer
- Consent form signed by the child's legal guardian
- Patient using effectiveness contraception during this trial
- Affiliated or beneficiary of a health insurance regimen
Who cannot join
- Wiskott-Aldrich syndrome
- Ongoing pregnancy
- Positive HIV PCR
- Contraindication for hematopoetic stem cell transplantation
- Geno-identical donor in the siblings
- hematopoetic stem cell transplantation antecedent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID): Selective Depletion of Naive Cells From the Graft
The purpose of this study is to evaluate selective depletion of naïve CD45RA+ T cells from allogenic peripheral blood stem cell graft in children transplanted for combined immune deficiency. The aims of this procedure are to prevent graft versus host disease (GVHD) while preserving anti-infectious response from donor memory T lymphocytes.
Read the detailed description
Combined immunodeficiencies (CIDs) are an heterogeneous group of primitive immunodeficiency (PID), which affect T cells development, function or both. These inherited conditions can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). These procedures have a high risk of morbidity and mortality such a graft versus host disease (GVHD), rejection of the graft and serious infections, especially in this population of children with PID. GVHD is more frequent and severe if the donor is not an identical sibling and/or presents an HLA-mismatch. GVHD requires high immunosuppression as prevention and treatment, and therefore impedes immunity against infections.
In vitro and animal models suggest that GVHD is mediated by naïve T cells. The aim of this study is to decrease the rate and severity of GVHD after selective depletion of naïve CD45RA+ T cells from allogeneic hematopoietic stem cell grafts in patients with CIDs with high risk of severe GVHD, and to preserve immunity against pathogens in a population with high vulnerability to infections.
The project aims is, first, to show improvement of rejection-free and GVH-free survival 12 months post-transplant, and secondly, to show the decrease of viral infection, and assess immune reconstitution kinetic and quality and specific antiviral responses, after a engraftment with naïve cell depleted allograft.
- Conditions
- Combined Immunodeficiencies
- Being tested
- Depletion in CD45RA graft donor
- Keywords
- Combined immunodeficiencies, hematopoietic stem cell transplantation, CD45RA, primitive immunodeficiencies, pediatric transplantation, CD45RA depletion
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Depletion in CD45RA graft donorBiological
Experimental treatment: negative fraction after CD34+ selection from PBSC graft is depleted of naïve CD45RA+ cells. this fraction is reinjected to the recipient and is the experimental product.
Conditioning regimen:
Up-front ATG from D-14 toD-11 Busulphan IV from D-8 to -5 Fludarabine from D-7 to D-4 Thiothepa D-3 to D-2
Graft: CD34+ cells positively selected cells from PBSC of the donor
Post transplant immunosuppression: ciclosporin started at D-1 to D+100
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Not described Receives: Biological: Depletion in CD45RA graft donor |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of death
Measured: 12 months after the transplantation
Number of graft rejection
Measured: 12 months after the transplantation
Number of graft versus host disease (GVHD) grade III or IV
Measured: 12 months after the transplantation
Secondary outcomes (3)
Need of antiviral treatment
to assess viral infection
Measured: 12 months after the transplantation
T Lymphocyte proliferations to phytohemagglutinin (PHA)
to assess immune reconstitution
Measured: 12 months after the transplantation
Proportion of T CD4 and CD8 lymphocytes specific of cytomegalovirus, Epstein Barr virus and adenovirus
to assess specific antiviral response
Measured: 12 months after the transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2016 (estimated)
- Study start14 Jun 2016
- Primary completion26 Oct 2017
- Study completion26 Oct 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Combined ImmunodeficienciesStudies 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Responsible party
- Sponsor
- Investigators
- Marina CAVAZZANA, MD, PhD, Study chair, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02737384
- Sponsor's protocol ID
- P140317
- Other IDs
- 2015-A01256-43
- Acronym
- CD45RA
- Status last verified
- Mar 2026
- First submitted
- 8 Apr 2016
- Last updated
- 30 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Touzot F, Neven B, Dal-Cortivo L, Gabrion A, Moshous D, Cros G, Chomton M, Luby JM, Terniaux B, Magalon J, Picard C, Blanche S, Fischer A, Cavazzana M. CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study. J Allergy Clin Immunol. 2015 May;135(5):1303-9.e1-3. doi: 10.1016/j.jaci.2014.08.019. Epub 2014 Oct 3.
PubMed 25282016
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Stem Cells Transplantation in Children With Combined Immunodeficiency (CID): Selective Depletion of Naive Cells From the Graft
- ClinicalTrials.gov ID
- NCT02737384
- Acronym / sponsor ID
- CD45RA / P140317
- Conditions
- Combined Immunodeficiencies
- Keywords
- Combined immunodeficiencies, hematopoietic stem cell transplantation, CD45RA, primitive immunodeficiencies, pediatric transplantation, CD45RA depletion
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 14 Jun 2016 (actual)
- Primary completion
- 26 Oct 2017 (actual)
- Study completion
- 26 Oct 2017 (actual)
- First posted
- 13 Apr 2016 (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.
The purpose of this study is to evaluate selective depletion of naïve CD45RA+ T cells from allogenic peripheral blood stem cell graft in children transplanted for combined immune deficiency. The aims of this procedure are to prevent graft versus host disease (GVHD) while preserving anti-infectious response from donor memory T lymphocytes.
Detailed description
Combined immunodeficiencies (CIDs) are an heterogeneous group of primitive immunodeficiency (PID), which affect T cells development, function or both. These inherited conditions can only be cured by allogeneic hematopoietic stem cell transplantation (HSCT). These procedures have a high risk of morbidity and mortality such a graft versus host disease (GVHD), rejection of the graft and serious infections, especially in this population of children with PID. GVHD is more frequent and severe if the donor is not an identical sibling and/or presents an HLA-mismatch. GVHD requires high immunosuppression as prevention and treatment, and therefore impedes immunity against infections.
In vitro and animal models suggest that GVHD is mediated by naïve T cells. The aim of this study is to decrease the rate and severity of GVHD after selective depletion of naïve CD45RA+ T cells from allogeneic hematopoietic stem cell grafts in patients with CIDs with high risk of severe GVHD, and to preserve immunity against pathogens in a population with high vulnerability to infections.
The project aims is, first, to show improvement of rejection-free and GVH-free survival 12 months post-transplant, and secondly, to show the decrease of viral infection, and assess immune reconstitution kinetic and quality and specific antiviral responses, after a engraftment with naïve cell depleted allograft.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Biological: Depletion in CD45RA graft donor |
Who may qualify
- Ages
- 12 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Patient from 12 months to 18 years
- Combined immunodeficiencies with known molecular diagnosis or if unknown, corresponding of p-CID study's definition
- Hematopoietic stem cell Transplantation planned with one of the following donors :
- sibling with 1 or 2 HLA antigens mismatch
- parent 10/10 or 9/10 identical
Key exclusion criteria (6 listed)
- Wiskott-Aldrich syndrome
- Ongoing pregnancy
- Positive HIV PCR
- Contraindication for hematopoetic stem cell transplantation
- Geno-identical donor in the siblings
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 |
|---|---|---|
| Depletion in CD45RA graft donorExperimental treatment: negative fraction after CD34+ selection from PBSC graft is depleted of naïve CD45RA+ cells. this fraction is reinjected to the recipient and is the experimental product. Conditioning regimen: Up-front ATG from D-14 toD-11 Busulphan IV from D-8 to -5 Fludarabine from D-7 to D-4 Thiothepa D-3 to D-2 Graft: CD34+ cells positively selected cells from PBSC of the donor Post transplant immunosuppression: ciclosporin started at D-1 to D+100 | Biological | Treatment |
Primary outcome
Number of death
Time frame: 12 months after the transplantation
Number of graft rejection
Time frame: 12 months after the transplantation
Number of graft versus host disease (GVHD) grade III or IV
Time frame: 12 months after the transplantation
3 secondary outcomes
Need of antiviral treatment
Time frame: 12 months after the transplantation
T Lymphocyte proliferations to phytohemagglutinin (PHA)
Time frame: 12 months after the transplantation
Proportion of T CD4 and CD8 lymphocytes specific of cytomegalovirus, Epstein Barr virus and adenovirus
Time frame: 12 months after the transplantation
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2026.
- Background: PubMed 25282016Touzot F, Neven B, Dal-Cortivo L, Gabrion A, Moshous D, Cros G, Chomton M, Luby JM, Terniaux B, Magalon J, Picard C, Blanche S, Fischer A, Cavazzana M. CD45RA depletion in HLA-mismatched allogeneic hematopoietic stem cell transplantation for primary combined immunodeficiency: A preliminary study. J Allergy Clin Immunol. 2015 May;135(5):1303-9.e1-3. doi: 10.1016/j.jaci.2014.08.019. Epub 2014 Oct 3.
- Individual participant dataSharing planned: No
Related trials for Combined Immunodeficiencies
RecruitingObservational study
Genetic Basis of Immunodeficiency
Severe Combined Immunodeficiency
RecruitingPhase 1/2
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID)
X-linked Severe Combined Immunodeficiency (XSCID)
Not yet recruitingPhase 1/2
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
SCID, X-Linked
Not yet recruiting
Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
Adenosine DeAminase Severe Combined ImmunoDeficiency (ADA-SCID)
Enrolling by invitationObservational study
Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders
Primary Immunodeficiency, DOCK8
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.
Marina CAVAZZANA, MD, PhD study chair
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Depletion in CD45RA graft donor in people with Combined Immunodeficiencies, run by Assistance Publique - Hôpitaux de Paris at 1 site 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 9 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: stopped early, as of the sponsor's last update on 30 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 1 site 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.
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 was stopped early. Results are not available in the source record.
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 death (time frame: 12 months after the transplantation). The study lists 3 primary outcomes in all. 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 NCT02737384, 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 30 Mar 2026; AzTrials last refreshed its copy on 8 Nov 2026.