NCT02333760

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.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
78mo left

Started Sep 2014

Longer than P75 for phase_1

Geographic Reach
2 countries

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress65%
Sep 2014Oct 2032

Study Start

First participant enrolled

September 1, 2014

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

October 28, 2014

Completed
2 months until next milestone

First Posted

Study publicly available on registry

January 7, 2015

Completed
17.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2032

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2032

Last Updated

June 3, 2021

Status Verified

May 1, 2021

Enrollment Period

18.1 years

First QC Date

October 28, 2014

Last Update Submit

May 31, 2021

Conditions

Wiskott-Aldrich Syndrome

Outcome Measures

Primary Outcomes (8)

  • Incidence and type of SAEs

    Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality

    yearly from 3 years to 15 years

  • Lentiviral integration sites

    Presence of lentiviral integration sites in different cells sub-populations

    yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)

  • Vector copy numbers

    Quantification of vector copy numbers on sorted cells population by q-PCR

    yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)

  • Replication competent lentivirus (RCL)

    Presence of RCL

    yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)

  • Change in medical conditions

    Weight and complete clinical exam

    yearly from 3 years to 10 years

  • Key medical events related to WAS

    Eczema status, infections, bleeding symptoms, autoimmune manifestation

    yearly from 3 years to 10 years

  • Hematological reconstitution

    CBC including platelets count and size

    yearly from 3 years to 10 years

  • Reconstitution of cell mediated and humoral immunity

    Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene

    yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )

Secondary Outcomes (3)

  • Need for associated treatments

    yearly from 3 years to 15 years

  • Representation of TCR families

    yearly from 3 years to 5 years

  • Bone marrow content

    yearly from 3 years to 5 years (optional)

Interventions

Autologous CD34+ cells transduced with WASP lentiviral vectorGENETIC

Follow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene

Eligibility Criteria

Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hopital Necker - Enfants Malades

Paris, 75743, France

Location

UCL Institute of Child Health

London, WC1N 1EH, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 35075289DERIVED

MeSH Terms

Conditions

Wiskott-Aldrich Syndrome

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesLymphopeniaLeukopeniaCytopeniaHemorrhagic DisordersLeukocyte DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedPrimary Immunodeficiency DiseasesImmunologic Deficiency SyndromesImmune System Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
OTHER
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 28, 2014

First Posted

January 7, 2015

Study Start

September 1, 2014

Primary Completion (Estimated)

October 1, 2032

Study Completion (Estimated)

October 1, 2032

Last Updated

June 3, 2021

Record last verified: 2021-05

Locations

GB(1)FR(1)