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Gene therapy offers hope for 'Bubble Boy' disease without a donor

NCT ID NCT04797260

Disease control Sponsor: Videja B.V. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused This study
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 13, 2026 · Last updated Jul 17, 2026 · Updated 3 times

Summary

This trial tests a gene therapy for infants under 2 years old with RAG1-deficient severe combined immunodeficiency (SCID), a life-threatening condition where the immune system barely works. The therapy uses the child's own blood stem cells, modified with a corrected gene, to try to rebuild a functioning immune system. It is designed for those who need a stem cell transplant but lack a matched donor. The study will monitor participants for at least 5 years to check safety and whether the treatment restores T and B cell immunity.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gene therapy (RAG1 LV CD34+ cells)
What this could lead to
If successful, this could provide a life-saving treatment option for infants with RAG1 SCID who have no matched donor, potentially restoring their immune system without lifelong medication.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply to all. Risks include adverse events, insertional mutagenesis, and the possibility that the therapy may not fully restore immunity.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2021

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

8 weeks to 24 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. RAG1-deficient SCID as confirmed by genetic analysis 2. Peripheral blood CD3+T cells \< 300/μL 3. Absence of peripheral blood naïve CD4+ T cells 4. Age \< 2 years 5. Age at least 8 weeks by the time of busulfan and fludarabine administration 6. Lack of an available HLA-identical sibling/family donor 7. Signed informed consent (parental or guardian) 8. Able to return to the local HSCT centre for follow-up (per protocol) during the 5-year trial and up to at least 15-year long-term follow-up after IMP administration Exclusion Criteria: 1. Omenn syndrome 2. Previous allogeneic HSCT 3. Significant organ dysfunction/co-morbidity (including but not limited to the ones listed below): 1. Mechanical ventilation 2. Shortening fraction on echocardiogram \<25% 3. Renal failure defined as dialysis dependence 4. Uncontrolled seizure disorder 4. Any other condition that the investigator considers is a contraindication to collection and/or infusion of trans-duced cells for that individual or indicate patient's inability to follow the protocol, for example contraindication f to busulfan, major congenital abnormalities, ineligible to receive anaesthesia, or documented refusal or inability of the family to return for scheduled visits. 5. Human immunodeficiency virus (HIV) infection or Human T-cell Leukemia Virus (HTLV) infection

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Conditions

The condition(s) this trial relates to.

severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-positive

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Severe combined immunodeficiency due to RAG1 deficiency

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites in 4 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Erciyes Üniversitesi TIP Fakültesi

    Kayseri, Turkey (Türkiye)

  • Hospital Universitari Vall d'Hebron

    Barcelona, 08035, Spain

  • Leiden University Medical Center

    Leiden, 2300RC, Netherlands

  • Wroclaw Medical University

    Wroclaw, 50-556, Poland

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