CClinicalTrials.gg
Enrolling by invitationNCT01440218IDIOMUpdated Jan 16, 2025

Idiopathic Diseases of Man

An observational study in Rare Disease and Idiopathic Disease, sponsored by Scripps Translational Science Institute. Enrolling by invitation at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-01-16.

Sponsored by Scripps Translational Science Institute · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
10
Sex
All
01

Study summary

This research is being done to learn more about possible genetic causes of currently undiagnosed conditions, and to find out how the development of new technologies, such as DNA sequencing, can increase knowledge of the role genetic variants play in disorders and possibly how genetic variants may help de-termine the best treatment options.

The recent development of new technologies has increased our ability to understand how genetic mutations are associated with disease. Using these technologies to find the genetic variants responsible for rare diseases is a rapidly growing field and has already begun to transform the way conditions with unknown causes are diagnosed and treated.

Hypothesis: Identification of new genomic variants associated with idiopathic diseases and/or diseases of unknown etiology will advance medical knowledge about rare and common diseases.

02

Conditions studied

  • Rare Disease
  • Idiopathic Disease

Browse trials for

Keywords

  • Idiopathic diseases
  • family member of the affected individual
  • unknown etiology
03

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients with idiopathic diseases, referred by their physician.

Inclusion criteria

  1. Individual with rare disorder with previous unknown etiology.
  2. Individual with known disorder that does not respond to conventional treatment.
  3. Individual experienced a rare adverse event that was a result of the administration of a pharmacologic or biologic agent, immunization or device.
  4. Individual is a family member of the affected individual. -

Exclusion criteria

Exclusion Criteria:

  1. Unwilling or unable to grant informed consent if they do not have a legal guardian who has authority to sign a consent form on their behalf.
  2. Have a significant medical, affective, or psychiatric condition that in the Investigator's opinion may interfere with subject's study participation.
04

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
10 participants (estimated)
Biospecimen retention
Samples with dna

Groups and cohorts

  • Patients with idiopathic diseases

    Study population is limited to individuals with a rare severe illness, and/or their family members.

05

What researchers measure

Primary outcomes

  1. Genomic sequencing of tissue

    Generation of genomic information that may inform the diagnosis and/or treatment of idiopathic diseases and/or diseases of unknown etiology.

    Time frame: Day 1

Secondary outcomes

  1. Identification of modifying genomic alterations

    Identification of modifying genomic alterations that may indirectly exacerbate the condition.

    Time frame: Day 1

06

Study locations

1 site
  • Scripps Translational Science Institute
    La Jolla, California 92037, United States
07

Registry details

Key details

Study ID
NCT01440218
Lead sponsor
Scripps Translational Science Institute
Responsible party
Eric Topol, MD (Director, Scripps Translational Science Institute) — Principal investigator
First posted
Sep 26, 2011
Start date
Sep 2011
Primary completion
Dec 2025 (estimated)
Completion
Dec 2030 (estimated)
Last update
Jan 16, 2025

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Interested in this study?

Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.

No contact was published for this record. The registry link below has the sponsor’s details.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion