A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS.

Orchard Therapeutics Ltd.0 sites6 target enrollmentNovember 19, 2018

ConditionsWiskott-Aldrich Syndrome MedDRA version: 20.0Level: PTClassification code 10061598Term: ImmunodeficiencySystem Organ Class: 10021428 - Immune system disorders Therapeutic area: Diseases [C] - Immune System Diseases [C20]

DrugsBusilvex Fludarabina Accord MabThera Mozobil,MYELOSTIM

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Wiskott-Aldrich Syndrome
Sponsor: Orchard Therapeutics Ltd.
Enrollment: 6
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

November 19, 2018

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

Male

Investigators

Sponsor

Orchard Therapeutics Ltd.

Eligibility Criteria

Inclusion Criteria

•Age: up to 65 years.
•Diagnosis of WAS defined by genetic mutation and at least one of the following criteria:
•Severe WASP mutation;
•Absent WASP expression;
•Severe clinical score (Zhu clinical score \= 3\);
•Family member affected by WAS with life\-threatening or fatal clinical events.
•No human leukocyte antigen (HLA)\-identical related donor available.
•Parental/guardian/subject\-signed informed consent, and subject assent (if appropriate).
•For all subjects in the reproductive age range, agreement to use highly
•effective and adequate method of contraception (as detailed in Appendix 4: Contraceptive Guidance and Collection of Pregnancy Information) while receiving treatment and for at least 12 months following drug administration.

Exclusion Criteria

•1\. End\-organ dysfunction, severe active infection not responsive to treatment or other severe disease or clinical condition which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
•In addition to the potential infections tested per protocol, the PI should Tissue Directive as clinically appropriate and discuss the results with the medical monitor prior to cell harvest.
•Patients with ALT \>2x upper limit of normal (ULN) or total bilirubin \>1\.5 x ULN may be included only after discussed and agreed with the medical monitor and considered in the context of the criterion for excluding patients with other severe disease.
•Isolated elevation of total bilirubin \>1\.5 x ULN is acceptable if bilirubin is fractionated and direct bilirubin \<35% of total.
•2\. Malignant neoplasia (except local skin cancer) or a documented history of hereditary cancer syndrome . Patients with a prior successfully treated malignancy and a sufficient follow\-up to exclude recurrence (based on oncologist opinion) can be included after discussion and approval by the medical monitor.
•3\. Myelodysplasia, cytogenetic alterations characteristic of myelodysplastic syndrome and acute myeloid leukaemia , or other serious haematological disorders.
•4\. Prior allogeneic hematopoietic stem cell transplantation, with evidence of residual cells of donor origin.
•5\. Documented HIV infection (positive HIV RNA and/or anti\-p24 antibodies).
•6\. Current participation in other interventional clinical trials.
•7\. Previous gene therapy.