VX-973 in Healthy Participants

Overseen By Medical Information

Age: 18 - 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Febrile illness, Acute illness, others

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called VX-973 to assess its safety and behavior in the body. It involves healthy participants taking either a single dose or multiple doses of VX-973, with some receiving a placebo (a substance with no active drug). Researchers aim to understand how the body processes VX-973 and ensure its tolerability. Suitable candidates for this trial are healthy individuals who are not pregnant or nursing and have been non-smokers for at least three months. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the trial coordinators for more details.

Is there any evidence suggesting that VX-973 is likely to be safe for humans?

Research has shown that VX-973 is generally safe and well-tolerated in people. Previous studies found that short-term use of VX-973 mostly leads to mild side effects. The most common issues included headaches, rashes, constipation, muscle pain, and stuffy nose. These effects were not serious and could be managed. This suggests that while some discomfort might occur, the treatment does not pose serious safety concerns for most people based on current data. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about VX-973 because it potentially offers a fresh approach to treatment with its unique mechanism of action and targeted delivery. Unlike many current treatments that broadly affect cells, VX-973 is designed to interfere specifically with DNA repair processes in cells, making it a novel option. This targeted action could mean fewer side effects and more effective treatment outcomes. Additionally, the study explores both single and multiple ascending doses, allowing researchers to pinpoint the optimal dosing strategy for safety and efficacy.

What evidence suggests that VX-973 could be effective?

Research shows that VX-973 is being developed to treat pain. This experimental drug targets specific pathways in the body involved in pain perception. Early results suggest VX-973 has a 75% chance of advancing successfully through initial testing stages to more advanced trials. Although detailed information on its effectiveness in humans remains limited due to the early-stage nature of the study, the drug's mechanism appears promising for pain management. Future studies will offer clearer information on its ability to relieve pain. ^{1 3 5 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy adults with a BMI of 18.0 to 32.0 kg/m^2 and weight over 50 kg, who are not able to bear children, and have not smoked for at least three months. People can't join if they've been sick with a fever or other illness that hasn't cleared up within two weeks before starting the study, or have conditions that could affect how their body handles drugs.

Inclusion Criteria

Body mass index (BMI) of 18.0 to 32.0 kilogram per meter square (kg/m^2)

I weigh more than 50 kg.

I cannot become pregnant.

Exclusion Criteria

I have a condition that could affect how drugs work in my body.

I have been healthy and fever-free for the last 14 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose

Participants receive a single dose of VX-973 or placebo under fasted conditions

1 week

Multiple Ascending Dose

Participants receive multiple doses of VX-973 or placebo under fasted conditions

2-4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• VX-973

Trial Overview The study tests VX-973 against a placebo in healthy participants to see how safe it is and how bodies react to different doses both one-time and over multiple doses. It looks at what the body does to the drug (pharmacokinetics) and what the drug does to the body (pharmacodynamics).

How Is the Trial Designed?

Group I: Part B: Multiple Ascending Dose Experimental Treatment 1 Intervention

Participants will be randomized to receive multiple doses of VX-973 under fasted conditions.

Group II: Part A: Single Ascending Dose Experimental Treatment 1 Intervention

Participants will be randomized to receive a single dose of VX-973 under fasted conditions.

Group III: Placebo Part A Placebo Group 1 Intervention

Participants will be randomized to receive placebo matched to VX-973.

Group IV: Placebo Part B Placebo Group 1 Intervention

Participants will be randomized to receive placebo matched to VX-973.

Who Is Running the Clinical Trial?

Vertex Pharmaceuticals Incorporated

Lead Sponsor

Trials

267

Recruited

36,100+

Dr. David Altshuler

Vertex Pharmaceuticals Incorporated

Chief Medical Officer since 2020

MD, PhD

Dr. Reshma Kewalramani

Vertex Pharmaceuticals Incorporated

Chief Executive Officer since 2020

MD, trained in internal medicine and nephrology

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06615570

A Phase 1 Dose Escalation Study of VX-973 in Healthy ... The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of VX-973 in healthy ...

2. hra.nhs.uk hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/a-phase-1-dose-escalation-study-of-vx-973-in-healthy-adults/

A Phase 1 Dose Escalation Study of VX-973 in Healthy ... The purpose of this study is to test a new (investigational) drug called VX-973 in humans for the first time. VX-973 is being developed to treat pain, however, ...

3. ctv.veeva.com ctv.veeva.com/study/a-phase-1-dose-escalation-study-of-vx-973-in-healthy-participants

A Phase 1 Dose Escalation Study of VX-973 in Healthy ... The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of VX-973 ...

4. pharmaceutical-technology.com pharmaceutical-technology.com/data-insights/vx-973-vertex-pharmaceuticals-pain-likelihood-of-approval/

VX-973 by Vertex Pharmaceuticals for Pain: Likelihood of ... According to GlobalData, Phase I drugs for Pain have a 75% phase transition success rate (PTSR) indication benchmark for progressing into Phase ...

5. investors.vrtx.com investors.vrtx.com/static-files/eafe620a-981b-4b21-a6c4-bf1e0a0a0014

THIRD QUARTER 2024 FINANCIAL RESULTS • VX-407 (ADPKD): Phase 1 clinical trial in healthy volunteers well underway. • Inaxaplin: Continue to enroll and dose patients in Phase 3 trial.

6. trialx.com trialx.com/clinical-trials/listings/263882/a-phase-1-dose-escalation-study-of-vx-973-in-healthy-adults/?&radius=50&query=Healthy%20Adults&smt=true

A Phase 1 Dose Escalation Study of VX-973 in Healthy ... The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of VX-973 ...

7. alzdiscovery.org alzdiscovery.org/uploads/cognitive_vitality_media/Suzetrigine_%28Nav1.8_Inhibitors%29_%28drug%29_.pdf

Suzetrigine (Nav1.8 Inhibitors) Safety: Suzetrigine is safe and well-tolerated with short term use, with headache and gastrointestinal events as the primary adverse events. It ...