Clinical Trials Register

Summary
EudraCT Number:	2022-002458-26
Sponsor's Protocol Code Number:	ECT-001-CB.004
National Competent Authority:	Netherlands - Competent Authority
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-11-11
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Netherlands - Competent Authority

A.2

EudraCT number

2022-002458-26

A.3

Full title of the trial

A PHASE II OPEN-LABEL STUDY OF UM171-EXPANDED CORD BLOOD TRANSPLANTATION IN PATIENTS WITH HIGH AND VERY HIGH-RISK ACUTE LEUKEMIA/MYELODYSPLASIA.

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

Study of expanded Cord Blood transplantation in patients with high-risk blood cancers

A.4.1

Sponsor's protocol code number

ECT-001-CB.004

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04103879

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	ExCellThera inc.
B.1.3.4	Country	Canada
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	ExCellThera inc.
B.4.2	Country	Canada
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Erasmus mC
B.5.2	Functional name of contact point	Jurjen Versluis
B.5.3	Address:
B.5.3.1	Street Address	Dr. Molewaterplein 40
B.5.3.2	Town/ city	Rotterdam
B.5.3.3	Post code	3015 GD
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	00310107040704
B.5.6	E-mail	j.versluis.1@erasmusmc.nl

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	ECT-001-CB
D.3.2	Product code	ECT-001-CB
D.3.4	Pharmaceutical form	Solution for infusion in administration system
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	Yes
D.3.11.3.1	Somatic cell therapy medicinal product	Yes
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Yes
D.3.11.3.5.1	CAT classification and reference number	somatic cell therapy medicinal product (product ref.: H0005772)
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

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

E.1.1.1

Medical condition in easily understood language

blood cancer

E.1.1.2

Therapeutic area

Diseases [C] - Blood and lymphatic diseases [C15]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	22.0
E.1.2	Level	PT
E.1.2	Classification code	10067859
E.1.2	Term	Allogenic stem cell transplantation
E.1.2	System Organ Class	10042613 - Surgical and medical procedures

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

1) To 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.

E.2.2

Secondary objectives of the trial

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.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

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.

E.4

Principal exclusion criteria

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

E.5 End points

E.5.1

Primary end point(s)

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

E.5.1.1

Timepoint(s) of evaluation of this end point

1, and 2 years post-transplant.

E.5.2

Secondary end point(s)

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

E.5.2.1

Timepoint(s) of evaluation of this end point

Neutrophil engraftment: absolute neutrophil count (ANC) ≥ 0.5 x 109/L for 3 consecutive days after the 1st post-CBT nadir. Time to ANC ≥ 0.1 x109/L
Platelet engraftment: sustained platelet count ≥ 20 x 109/L with no platelet transfusion in the preceding 7 days as per CIBMTR standards
Time to achieving a platelet count ≥ 50 x 109/L
TRM will be monitored throughout the study and in particular at 100 days and 1 and 2 years post transplantation
The incidence of acute and chronic GvHD will be evaluated throughout the study, but particularly evaluated at 1-year post-transplant for aGVHD, 1 and 2 years post-transplant for cGVHD, using NIH criteria.
The incidence of grade 3 or higher infectious complications, pre-engraftment and engraftment syndrom will be evaluated throughout the study

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLV

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

F.3.3.1

Women of childbearing potential not using contraception