A Phase Ii Open-Label Study of UM171-EXPANDED Cord Blood Transplantation in Patients with High and Very High-Risk Acute Leukemia/Myelodysplasia.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Excellthera Inc.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2024-11-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

ExCellThera inc

External identifiers

EU CT number

2024-517583-36-01

EudraCT number

2022-002458-26

ClinicalTrials.gov

NCT04103879

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

1) Examine the safety (including assessment of the rate of graft failure) and feasibility of infusing a single ECT-001-expanded cord blood in patients with high and very high-risk acute leukemia/myelodysplasia
2) Evaluate relapse-free survival at 1- and 2-years post-transplant in a group of patients with high-risk acute leukemia/myelodysplasia.

Secondary objectives 5

1. Determine the kinetics of hematologic engraftment (including neutrophil and platelet engraftment) following infusion of a single ECT-001-expanded cord blood.
2. Estimate the incidence of transplant related mortality at day 100 and 1-year post-transplant.
3. Determine incidence of acute and chronic GVHD by NIH criteria at 2 years post-transplant.
4. Determine incidence of grade 3 or higher infectious complications.
5. Determine incidence of pre-engraftment/engraftment syndrome requiring therapy.

Conditions and MedDRA coding

High risk and very high risk acute leukemia/myelodysplasia requiring an allogeneic haematopoietic stem cell transplantation

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 18-70 years old
2. Presence of a high and very high-risk hematologic malignancy
3. Availability of 2 CBs ≥ 4/6 HLA match when DRB1 is performed at the allele level and A, B at antigen resolution (intermediate resolution) and ≥ 4/8 HLA match when A, B, C and DRB1 are performed at the allele level. An acceptable alternative would be a 3/6 or 5/8 as long as there is no double DRB1 mismatch.
4. Karnofsky ≥70.
5. Left ventricular ejection fraction ≥ 40% (within 3 months unless patient has received chemotherapy or radiation therapy to the thorax since the last cardiac evaluation) or fractional shortening >22%
6. Forced vital capacity (FVC), forced expiratory volume in 1 second (FEV1) and diffusing capacity corrected for hemoglobin (DLCOc) ≥ 50% of predicted
7. Bilirubin < 2 x upper limit of normal (ULN) unless felt to be related to Gilbert’s disease or hemolysis; AST and ALT ≤ 2.5 x ULN; alkaline phosphatase ≤ 5 x ULN.
8. Adequate renal function defined as creatinine < 2.0 mg/dl (adults). All adults with a creatinine > 1.2 or a history of renal dysfunction must have estimated creatinine clearance > 50 ml/min.
9. Hematopoietic cell transplantation specific comorbidity index (HCT-CI) ≤3 if patients have ≥5% blasts in the bone marrow and HCT-CI ≤5 if 60-70 years old.

Exclusion criteria 13

1. Allogeneic myeloablative transplant within 6 months.
2. Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up, and tests.
3. Any abnormal condition or laboratory result that is considered by the principal investigator capable of altering patient condition or study outcome.
4. Active central nervous system involvement.
5. Chloroma > 2 cm.
6. Autologous hematopoietic stem cell transplant within 6 months.
7. Presence of a malignancy other than the one for which the UCB transplant is being performed and the expected survival related to the malignancy is estimated to be less than 75% at 5 years.
8. HIV positivity.
9. Hepatitis B or C infection with measurable viral load.
10. Liver cirrhosis.
11. Pregnancy, breastfeeding or unwillingness to use appropriate contraception.
12. Participation in a trial with an investigational agent within 30 days prior to entry in the study.
13. 3) Active or recent (prior 6 month) invasive fungal infection without ID consult and approval.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Evaluate relapse-free survival at 1- and 2-years post-transplant

Secondary endpoints 5

1. Kinetics of hematopoietic engraftment (Neutrophil engraftment and Platelet engraftment)
2. Transplant related mortality (TRM)
3. Incidence of acute and chronic GVHD
4. Incidence of grade 3 or higher infectious complications
5. Incidence of pre-engraftment and engraftment syndrome requiring therapy

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Excellthera Inc.

Sponsor organisation

Excellthera Inc.

Address

Marcelle-Coutu Pavilion, 2950 Chemin De Polytechnique 2950 Chemin De Polytechnique

City

Montreal

Postcode

H3T 1J4

Country

Canada

Scientific contact point

Organisation

Excellthera Inc.

Contact name

Pierre Caudrelier

Public contact point

Organisation

Excellthera Inc.

Contact name

Pierre Caudrelier

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 4 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications