Pediatric PatientsPhase 1/2HTLP NeckerNCT03879876
Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
A Phase 1/2 study testing Human T Lymphoid Progenitor (HTLP) injection in people with Pediatric Patients and Any Type of Severe Combined Immunodeficiency (SCID), run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Pediatric Patients, Any Type of Severe Combined Immunodeficiency (SCID), Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- What is being tested?
- Human T Lymphoid Progenitor (HTLP) injection
- 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
- 13 May 2020to 24 Apr 2024
- Phase
- Phase 1/2
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Pediatric patients affected by any type of SCID confirmed by clinical, immunological and/or molecular diagnosis and eligible for an allogeneic HSCT
- Absence of a matched sibling donor or a matched unrelated donor (MUD) 10/10
- Clinical conditions incompatible with the search of a MUD
- Written, informed consent of parents/ legal representative (child)
- Age ≤ 2 years at the time of screening
- No prior therapy with allogeneic stem cell transplantation
- No treatment with another investigational drug within one month before inclusion
- Patient affiliated to social security
Who cannot join
- Presence of an HLA genoidentical donor
- Absence of written parental consent
- Treatment with another investigational drug within one month before inclusion
- Positive for HIV infection by genome PCR
- Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)
Trial locations and contacts
1 site in 1 country.
France1 site
Unité d'Immunologie Hématologie Rhumatologie Pédiatrique (UIHR),
Paris, Île-de-France Region, 75015
About this study
Official title: A Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients.
Read the detailed description
Not provided
- Conditions
- Pediatric Patients, Any Type of Severe Combined Immunodeficiency (SCID), Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Being tested
- Human T Lymphoid Progenitor (HTLP) injection
- Keywords
- SCID, hematopoietic stem cell transplantation, Human T Lymphoid Progenitor
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human T Lymphoid Progenitor (HTLP) injectionDrug
Injection of progenitors derived from HTLP culture at Day 8-Day 12 after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients (Day7 of culture)
| Study group | Role | What they receive |
|---|---|---|
| Human T Lymphoid Progenitor (HTLP) injection | Experimental | Human T Lymphoid Progenitor cells (HTLPs) obtained after a brief period of ex vivo culture in the presence of the fusion protein DL-4, Retronectin® and a combination of cytokines Receives: Drug: Human T Lymphoid Progenitor (HTLP) injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose limiting toxicity (DLT)
to evaluate the procedure safety
Measured: 3 months post-transplant
reconstitution of the CD3+ TCRαβ+ cell compartment
determined by the presence of ≥ 300/µL total, circulating CD3+ TCRαβ+ T cells to evaluate the efficacy
Measured: Month 3
Secondary outcomes (10)
Time course of reconstitution of the different T cell subpopulations
time necessary to reach a normal number of naïve CD4+ and CD8+ T cells
Measured: Month 3, month 6, month 12
presence of recent thymic emigrants
To evaluate the active thymopoiesis
Measured: Month 3, month 6, month 12
T-cell receptor excision circles (TREC ) number in peripheral blood
To evaluate the active thymopoiesis
Measured: Month 3, month 6, month 12
TCR rearrangements
By NGS analysis
Measured: Month 3, month 6, month 12
B-cell reconstitution
number and phenotype for naïve IgD+CD27-, marginal zone IgD+CD27+, switched memory IgD-CD27+, and IgD-CD27- cells
Measured: Month 6, month 12
Immunoglobulin (Ig) levels
Measured: Month 6, month 12
NK cell numbers
Measured: Month 6, month 12
Cumulative incidence of infections
Measured: 12 months post-transplant
Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD)
Measured: 24 months post-transplant
Overall survival
Measured: 2 years post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2019
- Study start13 May 2020
- Primary completion3 Sep 2023
- Study completion24 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric PatientsStudies 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 1/2
- 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
- Isabelle ANDRE, PhD, Study director, Institut National de la Santé Et de la Recherche Médicale, FranceDespina MOSHOUS, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris and Université Paris Descartes
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03879876
- Sponsor's protocol ID
- P150949J
- Other IDs
- 2018-001029-14, 2018
- Acronym
- HTLP Necker
- Status last verified
- May 2026
- First submitted
- 15 Mar 2019
- Last updated
- 20 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID Patients
- ClinicalTrials.gov ID
- NCT03879876
- Acronym / sponsor ID
- HTLP Necker / P150949J
- Conditions
- Pediatric Patients, Any Type of Severe Combined Immunodeficiency (SCID), Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
- Keywords
- SCID, hematopoietic stem cell transplantation, Human T Lymphoid Progenitor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- URC-CIC Paris Descartes Necker Cochin
- Study start
- 13 May 2020 (actual)
- Primary completion
- 3 Sep 2023 (actual)
- Study completion
- 24 Apr 2024 (actual)
- First posted
- 19 Mar 2019 (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.
The purpose of this study is to evaluate the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients.
Detailed description
Not provided
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Human T Lymphoid Progenitor (HTLP) injection | Experimental | Drug: Human T Lymphoid Progenitor (HTLP) injectionHuman T Lymphoid Progenitor cells (HTLPs) obtained after a brief period of ex vivo culture in the presence of the fusion protein DL-4, Retronectin® and a combination of cytokines |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Pediatric patients affected by any type of SCID confirmed by clinical, immunological and/or molecular diagnosis and eligible for an allogeneic HSCT
- Absence of a matched sibling donor or a matched unrelated donor (MUD) 10/10
- Clinical conditions incompatible with the search of a MUD
- Written, informed consent of parents/ legal representative (child)
- Age ≤ 2 years at the time of screening
Key exclusion criteria (5 listed)
- Presence of an HLA genoidentical donor
- Absence of written parental consent
- Treatment with another investigational drug within one month before inclusion
- Positive for HIV infection by genome PCR
- Contra-indication to allogeneic transplantation or conditioning therapy (except SCID patients with DNA repair deficiency)
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 |
|---|---|---|
| Human T Lymphoid Progenitor (HTLP) injectionInjection of progenitors derived from HTLP culture at Day 8-Day 12 after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients (Day7 of culture) | Drug | Human T Lymphoid Progenitor (HTLP) injection |
Primary outcome
Dose limiting toxicity (DLT)
Time frame: 3 months post-transplant
to evaluate the procedure safety
reconstitution of the CD3+ TCRαβ+ cell compartment
Time frame: Month 3
determined by the presence of ≥ 300/µL total, circulating CD3+ TCRαβ+ T cells to evaluate the efficacy
10 secondary outcomes
Time course of reconstitution of the different T cell subpopulations
Time frame: Month 3, month 6, month 12
presence of recent thymic emigrants
Time frame: Month 3, month 6, month 12
T-cell receptor excision circles (TREC ) number in peripheral blood
Time frame: Month 3, month 6, month 12
TCR rearrangements
Time frame: Month 3, month 6, month 12
B-cell reconstitution
Time frame: Month 6, month 12
Immunoglobulin (Ig) levels
Time frame: Month 6, month 12
NK cell numbers
Time frame: Month 6, month 12
Cumulative incidence of infections
Time frame: 12 months post-transplant
Cumulative incidence of acute and chronic episodes of graft versus host disease (GVHD)
Time frame: 24 months post-transplant
Overall survival
Time frame: 2 years post-transplant
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 20 May 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Isabelle ANDRE, PhD study director
Institut National de la Santé Et de la Recherche Médicale, France
Despina MOSHOUS, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris and Université Paris Descartes
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Human T Lymphoid Progenitor (HTLP) injection in people with Pediatric Patients and Any Type of Severe Combined Immunodeficiency (SCID), 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 8 inclusion and 5 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 20 May 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose limiting toxicity (DLT) (time frame: 3 months post-transplant). The study lists 2 primary outcomes in all. 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 20 May 2026.