San Francisco, CANCT03538899Now EnrollingIRB Ready

Severe Combined Immunodeficiency Clinical Trial in San Francisco, CA

Access cutting-edge severe combined immunodeficiency treatment through this clinical trial at a research site in San Francisco. Study-provided care at no cost to qualified participants.

Sponsored by University of California, San Francisco

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Expert Care in San Francisco

Access severe combined immunodeficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related severe combined immunodeficiency treatment provided free

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Check if you qualify for this severe combined immunodeficiency clinical trial in San Francisco, CA

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Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Local to San Francisco

    Convenient for CA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit San Francisco site if eligible
  4. 4Begin participation

About This Severe Combined Immunodeficiency Study in San Francisco

This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene. This method involves transferring a normal copy of the DCLRE1C gene into stem cells of an affected patient. Participants will receive an infusion of stem cells transduced with a self-inactivating lentiviral vector that contains a normal copy of the DCLRE1C gene. Prior to the infusion they will receive sub-ablative, dose-targeted busulfan conditioning. The study will investigate if the procedure is safe, whether it can be done according to the methods described in the protocol, and whether the procedure will provide a normal immune system for the patient. A total of 24 newly diagnosed patients will be enrolled at the University of California San Francisco in this single-site trial and will be followed for 15 years post-infusion. It is hoped that this type of gene transfer may offer improved outcomes for ART-SCID patients who lack a brother or sister who can be used as a donor for stem cell transplantation or who have failed to develop a functioning immune system after a previous stem cell transplant.

Sponsor: University of California, San Francisco

Who Can Participate

Inclusion Criteria

≥2.0 months of age at initiation of busulfan conditioning
New diagnosis of typical or minimally leaky ART-SCID, as defined by the criteria below:
Artemis deficiency with bi-allelic pathogenic or likely pathogenic mutations in DCLRE1C; AND
CD3 count \< 50 autologous cells/µL (typical ART-SCID) OR spontaneous maternal chimerism, OR CD3 count \>50/µL and \<300/uL and with restricted T cell receptor Vb diversity; AND
CD45 cell response to mitogens (PHA) \< 50% of the lower limit of normal range for the lab (leaky ART-SCID).
No medically eligible HLA-identical sibling with a normal immune system who could serve as an allogeneic bone marrow donor (applies to newly diagnosed patients only).

Exclusion Criteria

Presence of a medically eligible HLA-matched sibling
Evidence of HIV infection by polymerase chain reaction or p24 antigen testing.
Unable to tolerate general anesthesia and/or marrow harvest or insertion of central venous catheter.
Any one of liver function tests AST, ALT, gamma-glutamyl transpeptidase (GGT) \>5X the upper limit of normal for lab and/or total bilirubin \>2.0 mg/dl (not due to Gilbert's) at the time of planned initiation of busulfan conditioning unless the elevated LFTs are considered to be due to medication, a viral infection for which there is no treatment other than reconstituting T cell immunity, or maternal GVHD.
Presence of any severe medical conditions making a patient unsuitable for busulfan administration
Presence of a recognized second gene mutation that results in an autosomal dominant or recessive disorder intrinsic to hematopoietic cells and that could be treated by an allogeneic HCT.
Presence of a medical condition indicating that survival is predicted to be less than 4 months, 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 therapy.
A social situation indicating that the family may not be able to comply with protocol procedures and recommended medical care and follow-up.
Other conditions which in the opinion of the Principal Investigator and/or co-investigators, contra-indicate the infusion of transduced cells or study participation.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in San Francisco?

Yes, this clinical trial (NCT03538899) has an active research site in San Francisco, CA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Severe Combined Immunodeficiency Treatment Options in San Francisco, CA

If you're searching for severe combined immunodeficiency treatment options in San Francisco, CA, this clinical trial (NCT03538899) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our San Francisco research site is actively enrolling participants for this clinical trial. You'll receive care from experienced severe combined immunodeficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all severe combined immunodeficiency clinical trials near you to find additional studies recruiting in your area.

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