A medication for rare immune disorders caused by specific gene changes
Safety and Efficacy of Itacitinib in treatment of JAK/STAT pathway disorders with activating mutations
['FUNDING_OTHER'] · BAYLOR COLLEGE OF MEDICINE · NIH-11170731
This project is testing a medication called Itacitinib to see if it can help people with rare immune disorders caused by specific genetic changes in the JAK/STAT pathway.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | BAYLOR COLLEGE OF MEDICINE (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-11170731 on ClinicalTrials.gov |
What this research studies
Many rare immune disorders are caused by specific gene defects, and it's often hard to develop new treatments for them due to the small number of patients. This project aims to address this challenge by using a special type of clinical trial, called a 'basket trial,' to test one drug, Itacitinib, across several different rare immune conditions. These conditions, including STAT1-GOF, STAT3-GOF, STAT5b-GOF, and JAK1-GOF, all involve overactive immune pathways due to specific genetic mutations. We hope to gather important information on whether Itacitinib is safe and effective for these conditions, which could lead to better treatment options.
Who could benefit from this research
Good fit: Ideal candidates are individuals diagnosed with rare immune disorders such as STAT1-GOF, STAT3-GOF, STAT5b-GOF, or JAK1-GOF, which are caused by dominant activating mutations.
Not a fit: Patients whose immune disorders are not related to activating mutations in the JAK/STAT pathway, or who do not have one of the specific conditions being studied, may not receive benefit from this particular treatment.
Why it matters
Potential benefit: If successful, this medication could offer a new, targeted treatment option for patients with specific rare immune disorders that currently have limited therapeutic choices.
How similar studies have performed: While targeted therapies for immune pathways exist for other conditions, this approach of using a 'basket trial' for rare immune disorders with Itacitinib is a novel strategy to gather evidence for these specific conditions.
Where this research is happening
HOUSTON, UNITED STATES
- BAYLOR COLLEGE OF MEDICINE — HOUSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: FORBES, LISA — BAYLOR COLLEGE OF MEDICINE
- Study coordinator: FORBES, LISA
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.