Gene TherapyNCT04286815
Gene Therapy for X Linked Severe Combined Immunodeficiency
A study testing Lentiviral Vector Gene Therapy in people with Gene Therapy, run by Children's Hospital of Chongqing Medical University at 1 site in China.
Trial summary
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking… Read more about this study
- Who is it for?
- Ages Up to 18 years, men only
- Conditions the study is for
- Gene Therapy
- What is being tested?
- Lentiviral Vector Gene Therapy
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 1 May 2020to 1 May 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 Up to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- X-SCID patients diagnosed by IL2RG single gene mutation
- No HLA（human leukocyte antigen） matching donor
- Hematopoietic stem cell transplantation failed and the time from transplantation was more than 18 months
- Severe and persistent refractory infections
- Life expectancy of > : 4 months
- HIV PCR in peripheral blood was negative
- the children and their families signed informed consent and were willing to enter the clinical trial and complete follow-up
Who cannot join
- The patient has diagnosed with hematological malignant diseases
- Received chemotherapy within 3 months
- HIV infection or HBV（hepatitis B virus） infection
- The patient or his first-degree relative has developed a malignant tumor within the age of 18 or has been diagnosed with malignant tumor prone genes
- Although the patient with X-SCID was diagnosed as IL2RG single gene mutation , the clinical phenotype was not severe, so they could continue to wait for the donor search;
- Patients whose family members have no intention to continue the follow-up treatment in any link
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400014
Xiaodong Zhao, PhD, 18623070626, zhaoxd530@aliyun.com
About this study
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.
- Conditions
- Gene Therapy
- Being tested
- Lentiviral Vector Gene Therapy
- Keywords
- Gene Therapy, X Linked Severe Combined Immunodeficiency, Lentiviral Vector
- Medical subject headings
- X-Linked Combined Immunodeficiency Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lentiviral Vector Gene TherapyDevice
Lentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells
| Study group | Role | What they receive |
|---|---|---|
| Experimental Group | Experimental | a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency）. Receives: Device: Lentiviral Vector Gene Therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
1-year survival rate 1-year survival rate
1-year survival rate of 10 recruited patients
Measured: one year after gene therapy of last recruited patient
3-year survival rate
3-year survival rate of 10 recruited patients
Measured: three years after gene therapy of last recruited patient
5-year survival rate
5-year survival rate of 10 recruited patients
Measured: five years after gene therapy of last recruited patient
Secondary outcomes (8)
Growth velocity after gene therapy,weight in kilograms, height in meters
Body weight and height of patients will be assessed prior to (month 0) and post gene therapy,weight in kilograms, height in meters
Measured: through study completion, an average of 2 year
Vector marking (vector copy number per cell) in blood and bone marrow cells
vector marking in T cells, B cells, NK cells, myeloid cells, and bone marrow progenitors.
Measured: through study completion, an average of 1 year
Absolute numbers of peripheral-blood immune-cell subsets
Absolute numbers of peripheral-blood immune-cell subsets，as determined by means of standard flow cytometry
Measured: through study completion, an average of 1 year
Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood mononuclear cells
Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood ，as determined by means of quantitative polymerase chain reaction (PCR)
Measured: through study completion, an average of 1 year
Serum immunoglobulins levels
Serum immunoglobulins levels will be reported IgM（immunoglobulin M） in mg/dL Serum immunoglobulins levels will be reported IgM in mg/dL
Measured: through study completion, an average of 2 year
Number of patients without intravenous immune globulin supplementation
Number of patients without intravenous immune globulin supplementation after gene therapy
Measured: through study completion, an average of 2 year
Number of patients who has a response to vaccines
Number of patients who has a response to vaccines after gene therapy
Measured: through study completion, an average of 2 year
Number of patients who recovers from previous infection（virus and bacteria）
Number of patients who recovers from previous infection（virus and bacteria）after gene therapy
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Feb 2020
- Study start1 May 2020 (estimated)
- Primary completion1 May 2023 (estimated)
- Study completion1 May 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Gene TherapyStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Chongqing Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, mingfeng hu, Children's Hospital of Chongqing Medical University
- Investigators
- Xiaodong Zhao, PHD, Study director, Assistant President of Children's Hospital of Chongqing Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04286815
- Sponsor's protocol ID
- CHCMU gene therapy
- Status last verified
- Mar 2020
- First submitted
- 23 Feb 2020
- Last updated
- 27 Mar 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408.
PubMed 30995372Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debre M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010 Jul 22;363(4):355-64. doi: 10.1056/NEJMoa1000164.
PubMed 20660403De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL, Kardava L. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856.
PubMed 27099176
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gene Therapy for X Linked Severe Combined Immunodeficiency
- ClinicalTrials.gov ID
- NCT04286815
- Acronym / sponsor ID
- CHCMU gene therapy
- Conditions
- Gene Therapy
- Keywords
- Gene Therapy, X Linked Severe Combined Immunodeficiency, Lentiviral Vector
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Children's Hospital of Chongqing Medical UniversityUniversity, hospital or other
- Study start
- 1 May 2020 (estimated)
- Primary completion
- 1 May 2023 (estimated)
- Study completion
- 1 May 2025 (estimated)
- First posted
- 27 Feb 2020 (actual)
- 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.
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency ）.The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number per cell) in blood and bone marrow cells, immune reconstitution vector insertion-site patterns and so on.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental Group | Experimental | Device: Lentiviral Vector Gene Therapya lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCID（severe combined immune deficiency）. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- X-SCID patients diagnosed by IL2RG single gene mutation
- No HLA（human leukocyte antigen） matching donor
- Hematopoietic stem cell transplantation failed and the time from transplantation was more than 18 months
- Severe and persistent refractory infections
- Life expectancy of > : 4 months
Key exclusion criteria (6 listed)
- The patient has diagnosed with hematological malignant diseases
- Received chemotherapy within 3 months
- HIV infection or HBV（hepatitis B virus） infection
- The patient or his first-degree relative has developed a malignant tumor within the age of 18 or has been diagnosed with malignant tumor prone genes
- Although the patient with X-SCID was diagnosed as IL2RG single gene mutation , the clinical phenotype was not severe, so they could continue to wait for the donor search;
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 |
|---|---|---|
| Lentiviral Vector Gene TherapyLentiviral vector to transfer IL2RG complementary DNA to patients'bone marrow stem cells | Device | Experimental Group |
Primary outcome
1-year survival rate 1-year survival rate
Time frame: one year after gene therapy of last recruited patient
1-year survival rate of 10 recruited patients
3-year survival rate
Time frame: three years after gene therapy of last recruited patient
3-year survival rate of 10 recruited patients
5-year survival rate
Time frame: five years after gene therapy of last recruited patient
5-year survival rate of 10 recruited patients
8 secondary outcomes
Growth velocity after gene therapy,weight in kilograms, height in meters
Time frame: through study completion, an average of 2 year
Vector marking (vector copy number per cell) in blood and bone marrow cells
Time frame: through study completion, an average of 1 year
Absolute numbers of peripheral-blood immune-cell subsets
Time frame: through study completion, an average of 1 year
Quantity of DNA T-cell-receptor excision circles (TRECs) in peripheral-blood mononuclear cells
Time frame: through study completion, an average of 1 year
Serum immunoglobulins levels
Time frame: through study completion, an average of 2 year
Number of patients without intravenous immune globulin supplementation
Time frame: through study completion, an average of 2 year
Number of patients who has a response to vaccines
Time frame: through study completion, an average of 2 year
Number of patients who recovers from previous infection（virus and bacteria）
Time frame: through study completion, an average of 2 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2020.
- Background: PubMed 30995372Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408.
- Background: PubMed 20660403Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debre M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2010 Jul 22;363(4):355-64. doi: 10.1056/NEJMoa1000164.
- Background: PubMed 27099176De Ravin SS, Wu X, Moir S, Anaya-O'Brien S, Kwatemaa N, Littel P, Theobald N, Choi U, Su L, Marquesen M, Hilligoss D, Lee J, Buckner CM, Zarember KA, O'Connor G, McVicar D, Kuhns D, Throm RE, Zhou S, Notarangelo LD, Hanson IC, Cowan MJ, Kang E, Hadigan C, Meagher M, Gray JT, Sorrentino BP, Malech HL, Kardava L. Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency. Sci Transl Med. 2016 Apr 20;8(335):335ra57. doi: 10.1126/scitranslmed.aad8856.
- Individual participant dataSharing planned: No
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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.
Xiaodong Zhao, PHD study director
Assistant President of Children's Hospital of Chongqing Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lentiviral Vector Gene Therapy in people with Gene Therapy, run by Children's Hospital of Chongqing Medical University at 1 site in China.
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 7 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 27 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xiaodong Zhao, PHD (18623070626, zhaoxd530@aliyun.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1-year survival rate 1-year survival rate (time frame: one year after gene therapy of last recruited patient). The study lists 3 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 NCT04286815, which also lists 3 linked publications.
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 27 Mar 2020.