SCID, X-LinkedPhase 1/2NCT03217617
SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
A Phase 1/2 study testing Direct intravenous injection of ivlv-X1 lentiviral vector in people with SCID, X-Linked, run by Shenzhen Geno-Immune Medical Institute at 1 site in China.
Trial summary
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. Read more about this study
- Who is it for?
- Ages 1 month to 1 year, men only
- Conditions the study is for
- SCID, X-Linked
- What is being tested?
- Direct intravenous injection of ivlv-X1 lentiviral vector
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 30 Jun 2027to 31 Dec 2029
- 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 1 month to 1 year
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of SCID-X1 based on:
- A proven mutation in the common interleukin-2 receptor gamma chain gene as defined by direct sequencing of patient DNA.
- T-cell immune deficiency defined as one or more of the following: CD3+ autologous T cells < 300/ul, or less than 50% of normal value for in vitro mitogen stimulation, or absent proliferation in vitro to antigen stimulation.
- No available HLA identical related donor.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
Show all 10Show fewer
- No prior allogeneic stem cell transplantation.
- Life expectancy ≥ 3 months.
- Documented to be negative for HIV infection.
- Written, informed consent obtained prior to any study-specific procedures.
Who cannot join
- No available molecular diagnosis confirming SCID-X1.
- Existence of an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
- Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
- Patients with evidence of infection with HIV-1 or 2.
- Presence of a medical condition indicating that survival will be less than 4 weeks such as the requirement for mechanical ventilation, severe failure of a major organ system, or evidence of a serious, progressive infection that is refractory to medical treatment.
- Current treatment with any immunosuppressive agent, excluding corticosteroids.
- Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, 518000
Lung-Ji Chang, PhD, +86 0755-86573763, c@mkgimi.com
About this study
Official title: Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Via Direct Intravenous Injection of Lentiviral Vector (Ivlv-X1)
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
Read the detailed description
Important Regulatory Notice:
This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China.
ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities.
X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder caused by defect in the common cytokine receptor chain, normally on the surface of lymphocytes. Individuals with SCID-X1 lack the normal development of a functional immune system and so have difficulty fighting infections, which may lead to chronic or severe illness and death. X-SCID patients are normally rescued by a bone marrow transplant from a healthy donor. This trial aims to treat SCID-X1 using a self-inactivating lentiviral vector (LV) carrying a functional gene to correct the genetic defect. By direct intravenous (iv) injection of the LV (ivlv-X1) to modify immune cells in the body, this treatment may establish normal healthy immune cells and overcome the immunodeficiency.
The primary objectives are to evaluate the safety of the self-inactivating ivlv-X1-LV, the in vivo gene transfer protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of iv LV gene transfer efficiency, and finally the long-term correction of immunodeficiency.
- Conditions
- SCID, X-Linked
- Being tested
- Direct intravenous injection of ivlv-X1 lentiviral vector
- Keywords
- SCID-X1, lentiviral vector, intravenous gene transfer
- 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.
Direct intravenous injection of ivlv-X1 lentiviral vectorBiological
ivlv-X1 LV intravenous injection at a dose of ~1x10e9/kg body weight.
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | In vivo LV gene transfer to treat SCID-X1 Receives: Biological: Direct intravenous injection of ivlv-X1 lentiviral vector |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Measured: 1 year
Overall immune reconstitution
T and B cell recovery
Measured: 1 year
Change of infection status
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jul 2017
- Study start30 Jun 2027 (estimated)
- Primary completion31 Dec 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SCID, X-LinkedStudies of the same condition, open and under way.
- More trials run by Shenzhen Geno-Immune Medical InstituteThe sponsor's other registered studies.
- 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
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shenzhen Geno-Immune Medical Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Lung-Ji Chang, Shenzhen Geno-Immune Medical Institute
- Investigators
- Lung-Ji Chang, Ph.D, Principal investigator, Shenzhen Geno-Immune Medical Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03217617
- Sponsor's protocol ID
- GIMI-IRB-17014
- Status last verified
- Nov 2026
- First submitted
- 3 Jul 2017
- Last updated
- 3 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) Via Direct Intravenous Injection of Lentiviral Vector (Ivlv-X1)
- ClinicalTrials.gov ID
- NCT03217617
- Acronym / sponsor ID
- GIMI-IRB-17014
- Conditions
- SCID, X-Linked
- Keywords
- SCID-X1, lentiviral vector, intravenous gene transfer
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Shenzhen Geno-Immune Medical InstituteUniversity, hospital or other
- Study start
- 30 Jun 2027 (estimated)
- Primary completion
- 31 Dec 2028 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 14 Jul 2017 (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.
This is a Phase I/II gene therapy trial treating X-linked severe combined immunodeficiency (SCID-X1) using a self-inactivating lentiviral vector (ivlv-X1) to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct intravenous lentiviral gene transfer protocol.
Detailed description
Important Regulatory Notice:
This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China.
ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities.
X-linked severe combined immunodeficiency (SCID-X1) is a genetic disorder caused by defect in the common cytokine receptor chain, normally on the surface of lymphocytes. Individuals with SCID-X1 lack the normal development of a functional immune system and so have difficulty fighting infections, which may lead to chronic or severe illness and death. X-SCID patients are normally rescued by a bone marrow transplant from a healthy donor. This trial aims to treat SCID-X1 using a self-inactivating lentiviral vector (LV) carrying a functional gene to correct the genetic defect. By direct intravenous (iv) injection of the LV (ivlv-X1) to modify immune cells in the body, this treatment may establish normal healthy immune cells and overcome the immunodeficiency.
The primary objectives are to evaluate the safety of the self-inactivating ivlv-X1-LV, the in vivo gene transfer protocol and the efficacy of immune reconstitution in patients overcoming frequent infections present at the time of treatment, assessment of iv LV gene transfer efficiency, and finally the long-term correction of immunodeficiency.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Biological: Direct intravenous injection of ivlv-X1 lentiviral vectorIn vivo LV gene transfer to treat SCID-X1 |
Who may qualify
- Ages
- 1 month to 1 year
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Diagnosis of SCID-X1 based on:
- No available HLA identical related donor.
- With severe infections, including but not limited to: pneumonitis; protracted diarrhea requiring total parenteral nutrition; infection with herpes viruses or adenovirus; disseminated BCG infection.
- No cytogenetic abnormalities (medullary karyotype) and no detection of main rearrangements associated with acute leukemia.
- No prior allogeneic stem cell transplantation.
Key exclusion criteria (8 listed)
- No available molecular diagnosis confirming SCID-X1.
- Existence of an available HLA-identical related donor.
- Diagnosis of active malignant disease other than EBV-associated lymphoproliferative disease.
- Current treatment with any chemotherapeutic agent (becomes eligible if not on treatment for at least 1 month).
- Patients with evidence of infection with HIV-1 or 2.
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 |
|---|---|---|
| Direct intravenous injection of ivlv-X1 lentiviral vectorivlv-X1 LV intravenous injection at a dose of ~1x10e9/kg body weight. | Biological | Single arm |
Primary outcome
Overall survival
Time frame: 1 year
Overall immune reconstitution
Time frame: 1 year
T and B cell recovery
Change of infection status
Time frame: 1 year
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 Nov 2026.
- Individual participant dataSharing planned: No
Related trials for SCID, X-Linked
Enrolling by invitationPhase 1/2
Base-Edited Hematopoietic Stem/Progenitor Cell X-Linked Severe Combined Immunodeficiency Gene Therapy
X-linked Severe Combined Immunodeficiency, X-SCID
Active, not recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
SCID, Omenn's Syndrome
Enrolling by invitationObservational study
Natural History Study of SCID Disorders
Severe Combined Immunodeficiency (SCID), Leaky SCID
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.
Lung-Ji Chang, Ph.D principal investigator
Shenzhen Geno-Immune Medical Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Direct intravenous injection of ivlv-X1 lentiviral vector in people with SCID, X-Linked, run by Shenzhen Geno-Immune Medical Institute 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 10 inclusion and 8 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: opening soon, as of the sponsor's last update on 3 Nov 2026. 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.
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?
Contact Lung-Ji Chang, Ph.D (+86 0755-86573763, c@mkgimi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: 1 year). 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?
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 Nov 2026; AzTrials last refreshed its copy on 8 Nov 2026.