Clinical trial NCT02333760: Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
Official title: Long Term Safety Follow up of Patients Enrolled in the Phase I/II Clinical Trial of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome (GTG002-07 and GTG003-08).
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE1
PHASE2
Conditions: Wiskott-Aldrich Syndrome
Lead sponsor: Genethon
Sponsor class: OTHER
First posted: 2015-01-07
Enrollment: 10
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

Brief summary: An open follow up study of patients enrolled in the Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome and treated with autologous CD34+ cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector.


Eligibility criteria: Inclusion Criteria:

* Patients enrolled in the initial phase I/II WAS conducted in France and United Kingdom (GTG002.07 and GTG003.08).
* Parents, guardians or patient signed informed consent, guardians or patient signed informed consent

Exclusion Criteria:

• Parents, guardians, patients unwilling to return for the follow up study period.

Primary outcomes:
measure: Incidence and type of SAEs
timeFrame: yearly from 3 years to 15 years
measure: Lentiviral integration sites
timeFrame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
measure: Vector copy numbers
timeFrame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
measure: Replication competent lentivirus (RCL)
timeFrame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
measure: Change in medical conditions
timeFrame: yearly from 3 years to 10 years
measure: Key medical events related to WAS
timeFrame: yearly from 3 years to 10 years
measure: Hematological reconstitution
timeFrame: yearly from 3 years to 10 years
measure: Reconstitution of cell mediated and humoral immunity
timeFrame: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Secondary outcomes:
measure: Need for associated treatments
timeFrame: yearly from 3 years to 15 years
measure: Representation of TCR families
timeFrame: yearly from 3 years to 5 years
measure: Bone marrow content
timeFrame: yearly from 3 years to 5 years (optional)
Other outcomes:
Locations:
city: Paris
country: France
facility: Hopital Necker - Enfants Malades
city: London
country: United Kingdom
facility: UCL Institute of Child Health
Study officials:
References:
citation: Magnani A, Semeraro M, Adam F, Booth C, Dupre L, Morris EC, Gabrion A, Roudaut C, Borgel D, Toubert A, Clave E, Abdo C, Gorochov G, Petermann R, Guiot M, Miyara M, Moshous D, Magrin E, Denis A, Suarez F, Lagresle C, Roche AM, Everett J, Trinquand A, Guisset M, Bayford JX, Hacein-Bey-Abina S, Kauskot A, Elfeky R, Rivat C, Abbas S, Gaspar HB, Macintyre E, Picard C, Bushman FD, Galy A, Fischer A, Six E, Thrasher AJ, Cavazzana M. Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott-Aldrich syndrome. Nat Med. 2022 Jan;28(1):71-80. doi: 10.1038/s41591-021-01641-x. Epub 2022 Jan 24.