ALN-6400 for Healthy Volunteers

Recruiting at 1 trial location

Overseen By Alnylam Clinical Trial Information Line

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Alnylam Pharmaceuticals

Disqualifiers: HIV, Hepatitis B, Hepatitis C, others

Compensation: Varies

Number of Visits: Unknown

Duration: 1 day

120 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is researching ALN-6400, a new treatment tested in two parts. The first part involves healthy adults to assess safety and how the body processes a single dose. The second part focuses on adults with Hereditary Hemorrhagic Telangiectasia (HHT), a condition causing frequent nosebleeds and other bleeding issues, to evaluate multiple doses for effectiveness and safety. Healthy individuals who feel well and those diagnosed with HHT who regularly experience symptoms might be suitable candidates. As a Phase 1/Phase 2 trial, participants have the opportunity to be among the first to receive this new treatment and help determine its effectiveness and safety.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that ALN-6400 is likely to be safe for humans?

Research shows that ALN-6400 is still undergoing testing to determine its safety in humans. For single doses, no specific side effects have been reported yet. Since this study is in an early stage, any side effects that occur are likely to be mild and temporary, which is common in such trials.

For multiple doses, detailed information on side effects is not yet available. Early-stage trials like this one primarily focus on ensuring a treatment's safety before advancing to more complex studies. Serious issues are unlikely, but minor side effects might still occur.

Overall, researchers are closely monitoring ALN-6400 to ensure participant safety. The trial is designed to detect any problems early, prioritizing the safety of participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care, ALN-6400 is a cutting-edge treatment that uses gene silencing technology to target specific disease pathways at the genetic level. Most current treatments for similar conditions focus on symptom management or general pathways rather than directly addressing the root cause at the genetic level. Researchers are excited about ALN-6400 because it has the potential for greater precision and efficiency in treatment, promising not only better outcomes but also fewer side effects. This novel approach could revolutionize how we treat conditions traditionally managed by less targeted therapies.

What evidence suggests that ALN-6400 might be an effective treatment for HHT?

Research shows that ALN-6400 may help treat Hereditary Hemorrhagic Telangiectasia (HHT). This treatment targets specific processes in the body that cause symptoms like bleeding. Early results suggest that ALN-6400 can improve these symptoms in a controlled manner. Although information from human studies is still being gathered, the treatment's mechanism offers hope for enhancing the quality of life for people with HHT. More research is needed to confirm its effectiveness, but initial signs are promising. Participants in this trial will receive either a single or multiple doses of ALN-6400, or a placebo, to further evaluate its potential benefits. ^{1 2 3 4 6}

Who Is on the Research Team?

Medical Director

Principal Investigator

Alnylam Pharmaceuticals

Are You a Good Fit for This Trial?

This trial is for healthy adult volunteers who want to help test a new medication. There are specific requirements and exclusions that aren't listed here, but generally, participants should be in good health without any significant medical conditions.

Inclusion Criteria

I am a healthy adult.

Exclusion Criteria

Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN)

I have HIV or chronic hepatitis B or C.

Is not willing to comply with the contraceptive requirements during the study period

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of ALN-6400 or placebo

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ALN-6400

Trial Overview The study is testing ALN-6400, which is a new drug. Participants will receive either the actual drug or a placebo (a substance with no therapeutic effect) to compare outcomes. The focus is on how safe it is and how the body responds to different doses.

How Is the Trial Designed?

Group I: Part B: ALN-6400 Experimental Treatment 1 Intervention

Participants will be administered multiple doses of ALN-6400.

Group II: Part A: ALN-6400 Experimental Treatment 1 Intervention

Participants will be administered a single dose of ALN-6400.

Group III: Part A: Placebo Placebo Group 1 Intervention

Participants will be administered a single dose of placebo.

Group IV: Part B: Placebo Placebo Group 1 Intervention

Participants will be administered multiple doses of placebo.

Who Is Running the Clinical Trial?

Alnylam Pharmaceuticals

Lead Sponsor

Trials

81

Recruited

16,100+

Dr. Yvonne Greenstreet

Alnylam Pharmaceuticals

Chief Executive Officer since 2021

MD from the University of Leeds, MBA from INSEAD

Dr. Pushkal Garg

Alnylam Pharmaceuticals

Chief Medical Officer since 2016

MD from Columbia University

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06659640

NCT06659640 | A Study to Evaluate ALN-6400 in Healthy ... The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in ...

2. clinicaltrials.alnylam.com clinicaltrials.alnylam.com/trials/6659640

ALN-6400-001 - Alnylam Clinical Trials The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in ...

3. trialx.com trialx.com/clinical-trials/listings/294075/a-study-to-evaluate-aln-6400-in-adult-healthy-volunteers/

A Study to Evaluate ALN-6400 in Adult Healthy Volunteers The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ...

4. withpower.com withpower.com/trial/phase-1-healthy-subjects-hs-10-2024-a7dd4

ALN-6400 for Healthy Volunteers The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending doses of ALN-6400 in ...

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT06659640/a-study-to-evaluate-aln-6400-in-adult-healthy-volunteers

A Study to Evaluate ALN-6400 in Healthy Volunteers and ... The purpose of this study is to: evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single ascending ...

6. clinicaltrial.be clinicaltrial.be/en/details/33060?per_page=20&only_recruiting=0&only_eligible=0&only_active=0

A Study to Evaluate the Safety and Tolerability of ALN-AP... The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of a single dose and multiple ...