TSRA-196 for Alpha-1 Antitrypsin Deficiency
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called TSRA-196, an experimental therapy for individuals with severe alpha-1 antitrypsin deficiency (AATD), a genetic condition that can lead to lung and liver problems. The study aims to determine the safety, effectiveness, and behavior of TSRA-196 in the body. It targets individuals with the PiZZ genotype of AATD who experience lung issues like emphysema or liver problems with fibrosis (scarring). Participants must not have smoked for at least six months and must meet specific criteria for lung or liver conditions. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.
Do I have to stop taking my current medications for the trial?
The trial requires that participants either have never received AAT treatments or have stopped taking any investigational or approved treatments that modify AAT levels for a period of 5 half-lives or at least 4 weeks, whichever is longer, before starting the trial medication.
Is there any evidence suggesting that TSRA-196 is likely to be safe for humans?
Research has shown that TSRA-196 is designed to safely address the genetic issue causing alpha-1 antitrypsin deficiency (AATD). This new treatment aims to help affected individuals by restoring normal protein function. Although detailed safety information from human trials remains limited, the FDA has approved its development, indicating a positive outlook for its safety.
The FDA has granted TSRA-196 special designations, such as Fast Track and Orphan Drug status. These designations suggest the treatment holds promise for AATD, and early research indicates it might be safe and well-tolerated.
As TSRA-196 is in the early stages of clinical trials, it continues to gather safety data. While specific human safety results are not yet available, the trial focuses on assessing the treatment's safety for people, including monitoring reactions and noting any unwanted side effects.12345Why do researchers think this study treatment might be promising for alpha-1 antitrypsin deficiency?
Most treatments for Alpha-1 Antitrypsin Deficiency, like augmentation therapy, work by replacing the missing protein in the body. However, TSRA-196 is unique because it targets the underlying genetic cause of the condition, potentially offering a more lasting solution. Researchers are excited about TSRA-196 because it uses a novel mechanism to modify the gene responsible for producing alpha-1 antitrypsin, which could lead to improved lung function and fewer symptoms. This approach could provide a significant advancement over current treatments, which mainly focus on symptom management rather than addressing the root cause.
What evidence suggests that TSRA-196 might be an effective treatment for alpha-1 antitrypsin deficiency?
Research has shown that TSRA-196 is designed to address the genetic issue causing alpha-1 antitrypsin deficiency (AATD). In early studies, nine patients demonstrated promising results, with increased levels of the beneficial protein and decreased levels of the harmful one. Animal tests showed the treatment's effectiveness even at low doses, suggesting potential efficacy for humans as well. TSRA-196 has received Fast Track and Orphan Drug status, highlighting its potential to significantly enhance AATD treatment.12345
Are You a Good Fit for This Trial?
This trial is for adults aged 18–70 with severe alpha-1 antitrypsin deficiency (PiZZ genotype) and related lung or mild liver disease. Participants must have low AAT levels, be nonsmokers for at least 6 months, and not currently on treatments that affect AAT levels.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive escalating doses of TSRA-196 to evaluate safety and tolerability
Dose Expansion
Participants receive expanded doses of TSRA-196 to further assess efficacy and PK/PD parameters
Single Repeat Dose
Participants receive a single repeat dose of TSRA-196 to evaluate long-term effects
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- TSRA-196
Trial Overview
The study is testing a new drug called TSRA-196 in people with PiZZ alpha-1 antitrypsin deficiency. It will look at different doses to check safety, how well it works, and how the body processes the drug.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Find a Clinic Near You
Who Is Running the Clinical Trial?
Tessera Therapeutics, Inc.
Lead Sponsor
Citations
A Study of TSRA-196 in Adults With PiZZ Alpha-1 ...
The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure.
2.
investor.regeneron.com
investor.regeneron.com/news-releases/news-release-details/regeneron-and-tessera-therapeutics-jointly-develop-tsra-196Regeneron and Tessera Therapeutics to Jointly Develop ...
TSRA-196 is designed to precisely correct the genetic mutation underlying AATD, with the goal of restoring production of functional alpha-1 ...
Tessera Therapeutics Announces FDA Clearance of IND ...
Alpha-1 antitrypsin deficiency remains a serious genetic disease with significant unmet medical need, and we believe TSRA-196 has the potential ...
Advances in Precision Editing Therapies for Alpha-1 ...
In this review, we summarize the current preclinical and clinical efforts for AATD, with an emphasis on genome and RNA editing strategies. AAT ...
TSRA-196 Gets FDA Fast Track, Orphan Drug ...
Fast Track and Orphan designations allow us to accelerate the development of TSRA-196 to dramatically improve the lives of people living with ...
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