MedPath

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Phase 1
Conditions
Adult patients (n=10) requiring an allogeneic HSCT for the treatment of AML with an unfavorable prognosis according to international criteria in first complete remission or second complete remission following relapse and a Sorror score = 3.
MedDRA version: 21.0Level: PTClassification code 10000881Term: Acute myeloid leukaemia (in remission)System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Registration Number
EUCTR2019-004883-23-FR
Lead Sponsor
APHP
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
Not specified
Inclusion Criteria

•Adults (=18 and <55 years old) at the time of inclusion

•High risk AML in CR1 (with t(6;9), t(v; 11q23), t(9;22), Inv(3), t(3;3), abnormalities of 3q, monosomy 5, 7, 17, complex caryotype, monosomal caryotype, FLT3-ITD (Ratio =O.5) without NPM1 mutations)
or
•AML with any adverse genetic abnormality, secondary or therapy related AML excluding good risk genetic abnormalities, or any poor risk feature
or
•MRD+ patients after first line chemotherapy
or
•Relapsed AML in CR2
or
•>=CR2
•Absence of a matched – related sibling donor (MSD) or a matched unrelated donor (MUD) 10/10
•SORROR score = 3
•Presence of two UCB units with the following criteria*: HLA- matched 6/8, 7/8 or 8/8 for HLA- A, -B, and DRB1 locus, no mismatch at HLA- C locus
AND
•Presence of at least one UCB unit with the following criteria*: = 3 x 107 TNC/kg and = 1.5 105 CD34+/kg pre- freezing AND HLA- matched 6/8, 7/8 or 8/8 for HLA- A, -B, and DRB1 locus, no mismatch at HLA- C locus
* For the UCB taken into HTLP culture, the UCB with fewer CD34+ cells number will be chosen, as expansion during HTLP culture has been proven to ensure the appropriate number of CD7+ needed for each dose.
The non- cultured UCB will be chosen to have a higher CD34+ cell content in order to enable long- term hematopoietic engraftment
•No prior therapy with allogeneic stem cell transplantation
•No treatment with another investigational drug within one month before inclusion
•Patient affiliated to social security
•Written, informed consent of the patient

Are the trial subjects under 18? no
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 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•Active disease at the time of transplantation
•Previous allogeneic stem cell transplantation
•Any of the standard contraindications to allogeneic transplant
?Left ventricular ejection fraction <50%
?Abnormal biochemistry results (ALT/AST>10xULN, total bilirubin>2.5xULN, creatinin clearance <60ml/min)
?Lung: DLCO < 50%
•Inability to understand and provide informed consent
•Concomitant infectious disease: HTLV-I, HIV-I or HIV-II
•Pregnancy or breastfeeding for women of childbearing potential

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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