Bethesda, MDNCT01212055Now EnrollingIRB Ready

LAD-1 Clinical Trial in Bethesda, MD

Access cutting-edge lad-1 treatment through this clinical trial at a research site in Bethesda. Study-provided care at no cost to qualified participants.

Sponsored by National Cancer Institute (NCI)

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Expert Care in Bethesda

Access lad-1 specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related lad-1 treatment provided free

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Check if you qualify for this lad-1 clinical trial in Bethesda, MD

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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 Bethesda

    Convenient for MD 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 Bethesda site if eligible
  4. 4Begin participation

About This LAD-1 Study in Bethesda

Background: \- Gene therapy is being investigated as a possible treatment for individuals with immunodeficiency diseases or other conditions that make it difficult to fight off infection. Gene therapy avoids problems with donor identification and possible rejection of bone marrow transplant by using the patient s own modified blood cells to help treat the disease. Researchers are interested in collecting stem cells from the blood of individuals with immunodeficiency diseases in order to use the cells to develop potential gene therapy treatments. Objectives: \- To collect blood stem cells from patients with immunodeficiency diseases tto test our ability to correct the defects of these cells in the test tube. Eligibility: * Individuals between 18 and 40 years of age with immunodeficiency diseases. * Individuals with human immunodeficiency virus (HIV) will not be able to participate in this study. Design: * Participants will provide an initial blood sample for disease screening (such as hepatitis B and C, syphilis, or viruses like the Epstein-Barr virus, herpes simplex virus, or toxoplasmosis) and to check kidney and liver function. * Starting 5 days before blood donation, participants will receive daily injections of a drug called G-CSF (granulocyte colony stimulating factor, or filgrastim), which pushes stem cells out of the bone marrow and into the bloodstream. Participants will receive the injections at the National Institutes of Health Clinical Center. * On day 5, participants will have a single leukapheresis procedure to collect the stem cells from the blood. * No additional treatment will be provided as part of this protocol. The cells that are collected will be used fore experiments in the lab and will not be used to treat individuals with these diseases.

Sponsor: National Cancer Institute (NCI)

Who Can Participate

Inclusion Criteria

Individuals age 18-40 years.
Diagnosis of DOCK8 deficiency, LAD-1, or GATA2 Deficiency: -DOCK8 deficiency
Homozygous or compound heterozygous mutations in the DOCK8 gene. -LAD-1
Less than 10% CD18 expression on the neutrophil surface. -GATA2 Deficiency
Deleterious mutation of GATA2 Gene
Serum creatinine \<1.5 mg/dL.
Total Bilirubin \< 3mg/dl, ALT and AST \< 5X upper limit of normal.
Ability to give informed consent.
Adequate venous access for peripheral apheresis, or consent to use a temporary central venous catheter for apheresis.
Individuals of childbearing age must have a negative urine pregnancy test within one week prior to beginning filgrastim or filgrastim biosimilar administration.

Exclusion Criteria

HIV infection.
Chronic hepatitis B or hepatitis C virus infection.
History of psychiatric disorder which may compromise compliance with protocol, or which does not allow for appropriate informed consent.
Active infection that is not responding to antimicrobial therapy.
Pregnant Individuals may not participate per OHSRP SOP.
Any Individual who is breastfeeding as the effects of filgrastim or filgrastim biosimilar product on infants is not known.
Sexually active individuals capable of becoming pregnant who are unable or unwilling to use effective form(s) of contraception during the 10 days surrounding filgrastim or filgrastim biosimilar product administration and apheresis procedure(s). Effective forms of contraception include one or more of the following: intrauterine device (IUD), hormonal (birth control pills, injections, or implants), tubal ligation/hysterectomy, partner s vasectomy, barrier methods, (condom, diaphragm, or cervical cap), or abstinence. Males on the protocol must use an effective form of contraception at study entry.
Presence of active malignancy in another organ system other than the hematopoietic system.
Individuals with active pulmonary disease.
History of hypertension that is not controlled by medication, stroke, or severe heart disease. Individuals with symptomatic angina will be considered to have severe heart disease and will not be eligible.
Other medical contraindications to stem cell donation (i.e., severe atherosclerosis, autoimmune disease, iritis or episcleritis, deep venous thrombosis, cerebrovascular accident).
Thrombocytopenia (platelets less than 50,000 per microliter) at baseline evaluation.
Individuals receiving experimental therapy or investigational agents.
Sensitivity to filgrastim, to E. Coli derived products, or to a biosimilar recombinant protein to filgrastim.
Individuals must test negative for transfusion-transmissible infectious agents, including hepatitis B (HBsAg), hepatitis C (anti-HCV), HIV (anti-HIV-1/2).

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 Bethesda?

Yes, this clinical trial (NCT01212055) has an active research site in Bethesda, MD 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.

LAD-1 Treatment Options in Bethesda, MD

If you're searching for lad-1 treatment options in Bethesda, MD, this clinical trial (NCT01212055) 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 Bethesda research site is actively enrolling participants for this clinical trial. You'll receive care from experienced lad-1 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 lad-1 clinical trials near you to find additional studies recruiting in your area.

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