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Etablering av fibroblasthärledda cellinjer från hudbiopsier av patienter med immunbrist eller immunoregleringsstörningar

Etablering av fibroblasthärledda cellinjer från hud-/vävnadsbiopsier av patienter med immunbrist eller immunoregleringsstörningar

Background:

  • National Institutes of Health (NIH) researchers have been studying immune cells (white blood cells) to better understand how the human body s defense system works and adjusts or regulates itself, and how changes in this system can make a person sick.
  • To study the cells of patients who have problems with their immune systems, researchers would like to collect samples of skin cells from patients with immune system disorders and compare them with skin cells taken from healthy volunteers. By studying these cells, researchers hope to determine whether these cells can be modified to create a new kind of personalized gene therapy that would attempt to cure immune diseases in the future.

Objectives:

  • To obtain skin cells from patients with immune system disorders and from healthy volunteers for research and comparison purposes.

Eligibility:

  • Patients between the ages of 2 and 85 who have immune system disorders.
  • Healthy volunteers between the ages of 18 and 85.
  • Both groups will be selected from the eligible participants of existing NIH studies into immune system disorders.

Design:

  • Researchers may take up to two biopsies from participants arms, legs, abdomen, or back.
  • The biopsy site will be numbed with local anesthetic and cleaned before the sample is taken.
  • The punch skin biopsy needle will be inserted into the skin and rotated to remove a small circle of skin (approximately 1/4 to 3/8 of an inch across). The area will be closed with bandages or stitches, and then covered with a dressing. Any stitches will be removed in 7 to 10 days.
  • Tissue samples collected in the study will be stored for future research.

Studieöversikt

Status

Anmälan via inbjudan

Betingelser

Detaljerad beskrivning

This protocol is designed as an adjunct to other National Institutes of Health (NIH) IRB approved protocols that allow for genetic testing, which may include those that are screening protocols for patients with rare primary immunodeficiency or immunodysregulation disorders. Patients deemed of sufficient research interest after review of outside medical records, clinical evaluation, and testing, may be invited to participate in this study. Healthy volunteers and relatives of patients will also be invited to participate as a source of control samples for research testing. After consent and enrollment into this study, skin punch biopsies will be obtained to establish dermal fibroblast cell lines for research studies directed at understanding the genetic and biochemical bases of these diseases. Cell lines will also be used to investigate the utility of induced pluripotent stem cells (iPS) for lymphocyte derivation and targeted gene correction. The nasal epithelial scrapings will be used for research purposes to grow out primary nasal respiratory epithelial cell lines. These cell lines will be used for functional studies by testing virus replication in them. These functional studies will allow us to identify new primary immunodeficiencies that may present primarily as recurrent or persistent lower respiratory tract virus infections. Results with the potential to impact medical care will be relayed to the referring physicians and where applicable patients will be referred to other appropriate NIH protocols for additional clinical evaluation and treatment.

The study will enroll up to 200 patients and healthy volunteers over the next 27 years.

Studietyp

Observationell

Inskrivning (Beräknad)

200

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studieorter

    • Maryland
      • Bethesda, Maryland, Förenta staterna, 20892
        • National Institutes of Health Clinical Center

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

2 år till 85 år (Barn, Vuxen, Äldre vuxen)

Tar emot friska volontärer

Ja

Kön som är behöriga för studier

Allt

Testmetod

Icke-sannolikhetsprov

Studera befolkning

Up to 150 patients with poorly defined, rare inherited immunodeficiency or immunodysregulation disorders, and up to 50 healthy volunteers as controls. Patients and healthy volunteers, who are first evaluated under another NIAID protocol (such as 05-I-0213 or 06-I-0015) and others, may be offered the opportunity to participate in this protocol.

Beskrivning

  • INCLUSION CRITERIA:

Patients:

  1. To be enrolled in this study, a patient must be >=2 years of age but not >85 years of age,
  2. have a known diagnosis of primary immunodeficiency or immunodysregulation (or be a blood relative of such as patient),
  3. be concurrently enrolled on an NIH IRB approved NIAID protocol that includes genetic testing for disease of the immune system, such as but not limited to 05-I-0213 or 06-I-0015

Patient Relative: To be enrolled in this study, a patient relative must be:

  1. A biological relative of a participant being studied under this protocol. Relatives may be biological mother, father, sibling, children, grandparents, aunts, uncles and first cousins.

    a. A minor relative of the proban participant must demonstrate that they are asymptomatic carriers or are at risk for the disease

  2. be concurrently enrolled on an NIH IRB approved protocol that includes genetic testing for disease of the immune system, such as but not limited to 05-I-0213 or 06-I-0015.

  3. Greater than or equal to 8 years of age but not greater than 85 years of age,

Healthy Volunteers:

To be enrolled in this study, a normal volunteer must fulfill all of the following criteria:

  1. Be enrolled on protocol 05-I-0213.
  2. Be a healthy adult of either sex and between ages of 18 years and 85 years

EXCLUSION CRITERIA:

Patients or the Patient Relative are not eligible to be in this trial if::

  1. Platelet count less than 20,000/microL
  2. The individual is hemodynamically unstable because of acute bleeding.
  3. Any condition that, in the investigator s opinion, places the patient at undue risk by participating in the study or limits the utility of the specimen to be obtained.
  4. For Nasal Scraping: a history of turbinectomy or significant nasal pathology that would preclude obtaining mucosal scrape biopsies.

A Healthy Volunteer is not eligible to be in this trial if they:

  1. Areless than 18 years old or older than 85 years
  2. Weighs less than 110 pounds
  3. Are pregnant or breastfeeding
  4. Are receiving a chemotherapeutic agent(s) or has a malignancy
  5. Cannot avoid taking aspirin or non-steroidal anti-inflammatory medications during the 7 days preceding skin biopsy
  6. Have history of heart, lung, kidney disease, bleeding disorders, diabetes mellitus, chronic peripheral arterial or venous insufficiency, chronic diffuse skin conditions without uninvolved areas suitable for skin biopsy, poor skin healing, or keloid formation.
  7. Have been diagnosed as having viral hepatitis (B or C), human immunodeficiency virus (HIV), or a carrier for methicillin-resistant Staphylococcus aureus (MRSA)
  8. Hemoglobin measurement is less than 12.0 g/dL
  9. Platelet count less than 150,000/(micro)L
  10. PT greater than 15.0 seconds or PTT greater than 40 seconds
  11. Have a history of intravenous injection drug use or of engaging in high-risk activities for exposure to the acquired immunodeficiency syndrome (AIDS) virus.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Observationsmodeller: COHORT
  • Tidsperspektiv: CROSS_SECTIONAL

Kohorter och interventioner

Grupp / Kohort
Healthy Volunteers

Up to 50 subjects as healthy controls

Immunodeficiency

Up to 150 subjects with poorly defined, rare inherited immunodeficiency or immunodysregulation disorders

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Generate fibroblast, dermal, or other skin-resident cell lines
Tidsram: Over the lifetime of the study

Obtain skin punch biopsies to generate fibroblast, dermal, or other skin-resident cell lines in patients who previously underwent HSCT. Cells may also be used for somatic cell hybridization, cell complementation, assessing fibroblast-specific innate immune responses, or other genetic techniques.

Over the lifetime of the study

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Utredare

  • Huvudutredare: Helen C Su, M.D., National Institute of Allergy and Infectious Diseases (NIAID)

Publikationer och användbara länkar

Den som ansvarar för att lägga in information om studien tillhandahåller frivilligt dessa publikationer. Dessa kan handla om allt som har med studien att göra.

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

10 juni 2009

Studieregistreringsdatum

Först inskickad

7 maj 2009

Först inskickad som uppfyllde QC-kriterierna

7 maj 2009

Första postat (Beräknad)

8 maj 2009

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

27 oktober 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

26 oktober 2026

Senast verifierad

2 oktober 2026

Mer information

Termer relaterade till denna studie

Andra studie-ID-nummer

  • 090133
  • 09-I-0133

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

NEJ

IPD-planbeskrivning

There isn't any data generated on this study that is relevant or shareable to any individual participant. Data obtained from iPS line growth is not medically actionable and therefor will not be reported back or shared with the participant.

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Nej

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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