Wiskott-Aldrich SyndromePhase 1/2WASFUPNCT02333760
Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome
A Phase 1/2 study testing Autologous CD34+ cells transduced with WASP lentiviral vector in people with Wiskott-Aldrich Syndrome, run by Genethon at 2 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Wiskott-Aldrich Syndrome
- What is being tested?
- Autologous CD34+ cells transduced with WASP lentiviral vector
- How many people
- 10 participants
- Where
- 2 sitesin 2 countries
- Study dates
- Sep 2014to Oct 2032
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- Parents, guardians, patients unwilling to return for the follow up study period.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
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).
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.
- Conditions
- Wiskott-Aldrich Syndrome
- Being tested
- Autologous CD34+ cells transduced with WASP lentiviral vector
- Medical subject headings
- Wiskott-Aldrich Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and type of SAEs
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Measured: yearly from 3 years to 15 years
Lentiviral integration sites
Presence of lentiviral integration sites in different cells sub-populations
Measured: 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
Measured: 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
Measured: 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
Measured: yearly from 3 years to 10 years
Key medical events related to WAS
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Measured: yearly from 3 years to 10 years
Hematological reconstitution
CBC including platelets count and size
Measured: 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
Measured: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Secondary outcomes (3)
Need for associated treatments
Immunoglobulins, antibacterial, antifungal, antiviral drugs, transfusions
Measured: yearly from 3 years to 15 years
Representation of TCR families
Representation of TCR families by PCR TREC (TCR excision circle) and TCR V beta panel
Measured: yearly from 3 years to 5 years
Bone marrow content
Numbers and type of cells in bone marrow
Measured: yearly from 3 years to 5 years (optional)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2015 (estimated)
- Study startSep 2014
- Primary completionOct 2032 (estimated)
- Study completionOct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Wiskott-Aldrich SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Genethon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02333760
- Sponsor's protocol ID
- GNT-WAS-03
- Acronym
- WASFUP
- Status last verified
- May 2021
- First submitted
- 28 Oct 2014
- Last updated
- 3 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 35075289
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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).
- ClinicalTrials.gov ID
- NCT02333760
- Acronym / sponsor ID
- WASFUP / GNT-WAS-03
- Conditions
- Wiskott-Aldrich Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- GenethonUniversity, hospital or other
- Study start
- Sep 2014
- Primary completion
- Oct 2032 (estimated)
- Study completion
- Oct 2032 (estimated)
- First posted
- 7 Jan 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- Parents, guardians, patients unwilling to return for the follow up study period.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous CD34+ cells transduced with WASP lentiviral vectorFollow up of ex vivo gene therapy transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing human WASP gene | Genetic | – |
Primary outcome
Incidence and type of SAEs
Time frame: yearly from 3 years to 15 years
Incidence and nature of delayed events such as malignancies, hematologic, autoimmune events, mortality
Lentiviral integration sites
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Presence of lentiviral integration sites in different cells sub-populations
Vector copy numbers
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Quantification of vector copy numbers on sorted cells population by q-PCR
Replication competent lentivirus (RCL)
Time frame: yearly from 3 years to 15 years (from 11 to 15 yearly time points, only in case of Advers Events of Special Interest)
Presence of RCL
Change in medical conditions
Time frame: yearly from 3 years to 10 years
Weight and complete clinical exam
Key medical events related to WAS
Time frame: yearly from 3 years to 10 years
Eczema status, infections, bleeding symptoms, autoimmune manifestation
Hematological reconstitution
Time frame: yearly from 3 years to 10 years
CBC including platelets count and size
Reconstitution of cell mediated and humoral immunity
Time frame: yearly from 3 years to 10 years (from 3 years to 5 years for PHA and candida )
Immunophenotyping panel, whole blood lymphocytes proliferation assays, restoration of antibody production, humoral response to antigene
3 secondary outcomes
Need for associated treatments
Time frame: yearly from 3 years to 15 years
Representation of TCR families
Time frame: yearly from 3 years to 5 years
Bone marrow content
Time frame: yearly from 3 years to 5 years (optional)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2021.
- Derived from this study: PubMed 35075289Magnani 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Autologous CD34+ cells transduced with WASP lentiviral vector in people with Wiskott-Aldrich Syndrome, run by Genethon at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 3 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in France, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and type of SAEs (time frame: yearly from 3 years to 15 years). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02333760, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2021.