AzTrials
AzTrials

Allogeneic DiseasePhase 1HAPLO-GELNCT05855707

Wharton Jelly Mesenchymal Stromal Cells as GVHD Prophylaxis

A Phase 1 study testing WJ-MSC infusion in people with Allogeneic Disease and GVHD,Acute, run by Central Hospital, Nancy, France.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
Search all trials

Trial summary

Despite progress in chemotherapy, targeted therapy and immunotherapy, allogeneic hematopoietic stem cell transplantation (allo-SCT) is still the only curative procedure for some hematological malignancies. Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Allogeneic Disease, GVHD,Acute, Graft Failure
What is being tested?
WJ-MSC infusion
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
12 participants
Where
No sites listed yet
Study dates
Sep 2023to Sep 2026
Phase
Phase 1
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 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • With AML/ALL/SMD/SMP or lymphoid neoplasm requiring allogeneic stem cell transplantation
  • In complete response (CR) for AML/ALL or CR,partial response (PR) or non pre-treated for SMD/SMP and lymphoid neoplasm
  • Without a HLA matched related donor available and with identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
  • With usual criteria for HSCT:
  • ECOG ≤ 2
  • No severe and uncontrolled infection
Show all 13Show fewer
  • Cardiac function compatible with high dose of cyclophosphamide
  • Adequate organ function: ASAT and ALAT ≤ 2N, total bilirubin ≤ 1.5N, creatinine clearance ≥30ml/min (except if those abnormalities are linked to the hematological disease)
  • Requiring a RIC or non myeloablative conditioning:
  • >50 years old; (ii) heavily pre-treated; (iii) Comoribidities according to Sorror et al. Blood 2005;106(8):2912-9, notamment HCT/CI≥ 3 (JAMA. 2011 Nov 2;306(17):1874-83).
  • 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 and using effective contraceptive methods during treatment and within 12 months for women of childbearing age and 6 months for men of childbearing age after the last dose of cyclophosphamide

Who cannot join

  • History of Cancer in the last 5 years
  • Uncontrolled infection: Seropositivity for HIV or HTLV-1 or active hepatitis B or C defined by a positive PCR HBV or HCV and hepatic cytolysis due to HBV
  • Uncontrolled coronary insufficiency, recent myocardial infarction <6 month, current manifestations of heart failure, uncontrolled cardiac rhythm disorders, ventricular ejection fraction <50%
  • Pulmonary failure with DLCO<50%
  • Addition of immunosuppressant treatment for GVHD prophylaxis (except immunosuppressant allowed per protocol)
  • Renal failure with creatinine clearance <50ml / min
Show all 10Show fewer
  • Pregnancy (β-HCG positive) or breast-feeding
  • Any debilitating medical or psychiatric illness which would preclude the realization of the SCT or the understanding of the protocol
  • Under protection by law (tutorship or curatorship)
  • Unwilling or unable to comply with the protocol

About this study

Official title: Wharton's Jelly Mesenchymal Stromal Cell (WG-MSC) Injections as GVHD Prophylaxis in Hematopoietic Allogeneic Stem Cell Transplantation With an Haplo-identical Donor : a Dose Escalation Study

Despite progress in chemotherapy, targeted therapy and immunotherapy, allogeneic hematopoietic stem cell transplantation (allo-SCT) is still the only curative procedure for some hematological malignancies. The probability of finding a matched sibling donor (MSD) is estimated under the classical 30%, because of the age of patients and their relatives, and a matched unrelated donor (MUD) can take time to identify. Currently in France, 25% of the allo-SCT are performed with an haplo-identical related donor. The Baltimore group developed an approach using haploidentical related donors, RIC, T-replete bone marrow and post-transplant high dose cyclophosphamide (PTCy) in patients with advanced hematological malignancies. PTCy has shown to eradicate alloreactive donor and host T-cells, activated by respective antigens, thereby reducing the incidence of graft versus host disease (GvHD) but delaying hematopoietic recovery. Therefore, the main source of graft is peripheral blood stem cells (PBSC) mobilized by G-CSF in France. Unfortunately, with PBSC we observe a higher cumulative incidence of GvHD (around 50%) and a higher toxicity-related mortality (TRM), especially for recipients >50 years old. The co-transplantation of Mesenchymal Stem Cells (MSC) at the time of transplantation has previously shown a double interest in GvHD immunomodulation and hematopoiesis support. Pre-clinical studies (in mice) have shown that mesenchymal stromal cells (MSCs) from Wharton's Jelly reduce the incidence of GvHD when the infusions are weekly repeated. We propose a phase I clinical trial to find the maximum tolerated dose (MTD) of a weekly infusion of WJ-MSC administered as GvHD prophylaxis and as a support for a faster hematological reconstitution after haplo-identical allo-SCT.

Being tested
WJ-MSC infusion

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • WJ-MSC infusionBiological

    cellular therapy

Study groups in NCT05855707
Study groupRoleWhat they receive
maximum tolerated doseExperimental1. x10e6 WJ-MSC/kg/infusion for 3 weekly infusions 1.5x10e6 WJ-MSC/kg/infusion for 3 weekly infusions 2. x10e6 WJ-MSC/kg/infusion for 3 weekly infusions
Receives: Biological: WJ-MSC infusion

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Maximum Tolerated Dose

    The maximum tolerated dose (MTD) will be defined by the highest dose (highest level) where no patient out of 3, or only 1 patient out of 6 presents with dose-limiting toxicity (DLT).

    The occurrence, within 7 days following one of the three injections, of any adverse event (AE) reasonably related to the injection of CSM-GW grade 3 to 5 according to the NCI-CTCAE classification version 5.0, or part of the "Important Medical Event list", or having a severity criterion

    The maximum tolerated dose (MTD) will be defined by the highest dose (highest level) where no patient out of 3, or only 1 patient out of 6 presents with dose-limiting toxicity (DLT).

    The occurrence, within 7 days following one of the three injections, of any adverse event (AE) reasonably related to the injection of CSM-GW grade 3 to 5 according to the NCI-CTCAE classification version 5.0, or part of the "Important Medical Event list", or having a severity criterion

    Measured: 7 days

Secondary outcomes (6)

  1. acute and chronic GVHD incidence

    Measured: 12 months

  2. toxicity-related mortality (TRM)

    Measured: 12 months

  3. relapse incidence (RI)

    Measured: 12 months

  4. overall surival (OS)

    Measured: 12 months

  5. GvHD and relapse free survival (GRFS)

    Measured: 12 months

  6. poor graft function

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 May 2023
  2. Study startSep 2023 (estimated)
  3. Primary completionSep 2025 (estimated)
  4. Study completionSep 2026 (estimated)

Resources

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
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05855707
Sponsor's protocol ID
2023-504268-40-00
Other IDs
2023
Acronym
HAPLO-GEL
Status last verified
May 2023
First submitted
4 May 2023
Last updated
11 May 2023
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing WJ-MSC infusion in people with Allogeneic Disease and GVHD,Acute, run by Central Hospital, Nancy, 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 13 inclusion and 10 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: status not confirmed, as of the sponsor's last update on 11 May 2023. 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 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.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

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?

Maximum Tolerated Dose (time frame: 7 days). 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 NCT05855707.

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 11 May 2023.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in