Clinical Trials Register

Summary
EudraCT Number:	2021-004324-14
Sponsor's Protocol Code Number:	WU-CART-007-1001
National Competent Authority:	France - ANSM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2022-12-15
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

France - ANSM

A.2

EudraCT number

2021-004324-14

A.3

Full title of the trial

A Phase 1/2 Dose-Escalation and Dose-Expansion Study of the Safety and Efficacy of Anti-CD7 Allogeneic CAR-T cells (WU-CART-007) in Patients with Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia (T-ALL)/Lymphoblastic Lymphoma (LBL)

A.3.1

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

Study of the Safety and Activity of WU-CART-007 in Patients with Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia

A.4.1

Sponsor's protocol code number

WU-CART-007-1001

A.5.4

Other Identifiers

Name:

IND

Number:

27758

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	Wugen, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Wugen, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Wugen, Inc.
B.5.2	Functional name of contact point	Eileen Mcnulty
B.5.3	Address:
B.5.3.1	Street Address	4260 Forest Park Ave, Suite 100
B.5.3.2	Town/ city	St. Louis
B.5.3.3	Post code	MO 63108-1110
B.5.3.4	Country	United States
B.5.6	E-mail	wucart007_clinops@wugen.com

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	Yes
D.2.5.1	Orphan drug designation number	DRU-2021-8497
D.3 Description of the IMP
D.3.1	Product name	WU-CART-007
D.3.2	Product code	WU-CART-007
D.3.4	Pharmaceutical form	Suspension for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	CAR+ve T cells
D.3.9.3	Other descriptive name	WU-CART-007
D.3.9.4	EV Substance Code	SUB259142
D.3.10	Strength
D.3.10.1	Concentration unit	million organisms/ml million organisms/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	20
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	No
D.3.11.3.2	Gene therapy medical product	Yes
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	No
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	Yes
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

Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia

E.1.1.1

Medical condition in easily understood language

Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia

E.1.1.2

Therapeutic area

Diseases [C] - Cancer [C04]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.0
E.1.2	Level	PT
E.1.2	Classification code	10042987
E.1.2	Term	T-cell type acute leukaemia
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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	10029547
E.1.2	Term	Non-Hodgkin's lymphoma
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

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

• To characterize the safety, tolerability, dose-limiting toxicities (DLT), and maximum tolerated dose (MTD) or maximum administered dose (MAD) (if no MTD is defined) and define the Recommended Phase 2 Dose (RP2D) of WU-CART-007 in T-ALL/LBL (Phase 1).
• To investigate the preliminary anti-tumor activity, as measured by complete response rate (CRR), objective response rate (ORR) and duration of response (DOR) of WU-CART-007 in relapsed/refractory (R/R) T-ALL/LBL patients (Phase 2). CRR is defined as proportion of patients that achieve a complete remission (CR) + CR with partial hematologic recovery (CRh) + CR with incomplete hematologic recovery (CRi). ORR is defined as proportion of patients that achieve CR), CRh, CRi, morphologic leukemia free state (MLFS), and partial response (PR) in patients with EMD only

E.2.2

Secondary objectives of the trial

• To investigate the preliminary effect on overall survival (OS) and progression-free survival (PFS).
• To evaluate preliminary anti-tumor activity as measured by Complete Remission (CR) rate defined as proportion of patients that achieve a CR.
• To determine rate of successful transition to hematopoietic cell transplant (HCT) in eligible patients.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Evidence of relapsed or refractory T-ALL or T-LBL, as defined by World Health Organization (WHO) classification with bone marrow with ≥ 5% lymphoblasts by morphologic assessment or evidence of extramedullary disease at screening.
• Relapsed or refractory disease defined as at least one of the following criteria:
a. Primary refractory: failure to achieve CR after induction chemotherapy, per investigator.
b. Early Relapse: relapsed disease within 12 months of initial diagnosis.
c. Late Relapse (relapsed refractory disease): relapsed disease after 12 months of initial diagnosis AND failure of re-induction therapy after disease recurrence.
d. Relapsed or refractory disease after allogeneic transplant, and meet the following criteria:
i. There must be histological confirmation of relapse after HSCT of T-ALL or T-LBL.
ii. Undergone allogeneic HSCT > 90 days prior to enrollment from a match related or unrelated donor, cord blood donor, haplo-identical, or autologous stem cells.
iii. Off all immunosuppressive medications for a minimum of 2 weeks with the exception of physiologic doses of corticosteroids,
iv. No prior history of Grade 2 or greater (per Cairo-Bishop) veno-occlusive disease/sinusoidal obstruction syndrome (VOD), or active graft versus host disease (GvHD) see Exclusion Criteria 8 below for exceptions.
• Adequate renal, hepatic, respiratory, and cardiovascular function, as defined in the body of the protocol.
• Life expectancy >12 weeks
• Age: Lower age limit of 12 years. Adolescent ages 12 to 17 will be eligible for enrollment beginning at Dose Level 3 of the dose-escalation phase, after review of safety, efficacy and cPK data and after consultation with the appropriate regulatory agencies.
• ECOG/Karnofsky performance status 0 or 1 at screening (Adults age >16) or Lansky Performance Status 60 and above (adolescents ≤ 16),
• Ability to understand the nature of this study, comply with protocol requirements, and give written informed consent. For minors, legal guardian willingness to give written informed consent with patient assent, where appropriate.
• Willing to participate in WUC-007-02 for long-term follow up.

E.4

Principal exclusion criteria

Patients will be excluded from study entry if:
• They have received previous treatment with any prior anti-CD7 therapy.
• Have not recovered from the effects of previous therapy.
• Wash-out period of at least 5 half-lives from the last dose of any investigational therapy prior to screening period.
• Have active or latent hepatitis B or active hepatitis C, any uncontrolled infection, or untreated HIV positive.
• Have any serious active infection at the time of treatment, or another serious underlying medical condition that would impair the ability of the patient to receive protocol treatment.
• Have Grade 2 to 4 acute or extensive chronic GvHD requiring systemic immunosuppression (steroids). Grade 1 GvHD not requiring immunosuppression is acceptable and grade 2 skin GvHD if treated with topical therapy only is acceptable.
• Have psychological, familial, sociological, or geographical conditions that do not permit compliance with the protocol.
• Pregnant or nursing (lactating) women
• Require prohibited medications or treatments, eg, steroids, or anti-neoplastic agents (fully defined in Section 6.2 criterion # 13.
• Treated with anti-T cell monoclonal antibodies

E.5 End points

E.5.1

Primary end point(s)

1. Assessment of safety and tolerability and DLT
2. Establishment of MTD or MAD
3. Investigate preliminary anti-tumor activity

E.5.1.1

Timepoint(s) of evaluation of this end point

No formal interim analysis is planned, as this is an open label trial.
The final analysis of the study will occur when the first of either 1 of the following circumstances occurs:
• The last patient completes treatment with a minimum of 3-month follow up;
• Three years following the enrollment of the first patient.

E.5.2

Secondary end point(s)

1. Preliminimary effect on overall survival (OS) and progression-free survival (PFS)
2. Preliminary anti-tumor activity as measured by Complete Remission
3. Determine rate of successful transition to HCT in eligible patients

E.5.2.1

Timepoint(s) of evaluation of this end point

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

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Tolerability, dose-limited toxicity & immunogenicity

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

Yes

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.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

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

Australia

United States

France

Netherlands

Germany

United Kingdom

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

LVLS

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

Yes

F.1.1

Number of subjects for this age range:

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)

Yes

F.1.1.6.1

Number of subjects for this age range:

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)

Yes

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

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

All patients will be asked to consent and enroll in a long-term follow-up protocol, WUC-007-02. This separate study will follow patients for up to 15 years after WU-CART-007 infusion to evaluate the persistence of the WU-CART-007 cell clones and to explore any unanticipated genetic consequences secondary to the introduction of genetically modified cells. Long term toxicity, subsequent anti-cancer therapy, and survival status will be monitored on this protocol.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2023-06-26

Ethics Committee Opinion of the trial application

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status	Completed

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