Gaucher Disease Clinical Trials 2023

Gaucher Disease research studies recruiting patients in 2023 need your help. Receive premium care & cutting edge treatments by enrolling in gaucher disease clinical trials today.

Popular Trials

Mucolytic Agent

N-acetylcysteine for Gaucher Disease

Recruiting1 awardPhase 2

Minneapolis, Minnesota

This trial is testing if a pill called NAC can help people with Type 1 Gaucher disease by reducing harmful processes in their bodies. The study includes both patients with the disease and healthy volunteers to compare results. Researchers hope this will lead to better treatments for the disease.

Popular Filters

Trials for GD Patients

Virus Therapy

FLT201 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Fairfax, Virginia

This trial will follow participants with Gaucher disease type 1 who were previously treated with FLT201 in a different trial. The participants will be monitored for 5 years after the treatment.

Monoclonal Antibodies

LY3884961 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Durham, North Carolina

This trial tests a new drug called LY3884961 in adults with specific symptoms of Gaucher Disease. The study aims to find the safest and most effective dose by observing how patients' bodies react to different amounts of the drug. Researchers will monitor safety, side effects, and changes in disease symptoms over several years.

Gene Therapy

Gene Therapy (FLT201) for Gaucher Disease

Recruiting1 awardPhase 1

Los Angeles, California

This trial will evaluate the safety and effectiveness of a new treatment for Gaucher disease Type 1 in adults.

Substrate Reduction Therapy

Venglustat vs Imiglucerase for Gaucher Disease

Recruiting2 awardsPhase 3

Fairfax, Virginia

This trial compares a pill to injections in patients aged 12-18 and adults with Gaucher disease Type 3. The pill aims to help with brain symptoms, while the injections maintain overall health. The pill has the potential to improve Gaucher disease by rebalancing certain substances in the body.

Enzyme Replacement Therapy

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

Recruiting1 awardPhase 1

San Francisco, California

This trial will test if a baby with a Lysosomal Storage Disease can be safely treated in the womb.

Enzyme Replacement Therapy

PR001 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Oakland, California

This trial is studying a new drug for infants with a rare disease called Gaucher disease. The study will last about 5 years, and patients will be monitored for safety and efficacy.

Trials With No Placebo

Virus Therapy

FLT201 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Fairfax, Virginia

This trial will follow participants with Gaucher disease type 1 who were previously treated with FLT201 in a different trial. The participants will be monitored for 5 years after the treatment.

Monoclonal Antibodies

LY3884961 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Durham, North Carolina

This trial tests a new drug called LY3884961 in adults with specific symptoms of Gaucher Disease. The study aims to find the safest and most effective dose by observing how patients' bodies react to different amounts of the drug. Researchers will monitor safety, side effects, and changes in disease symptoms over several years.

Gene Therapy

Gene Therapy (FLT201) for Gaucher Disease

Recruiting1 awardPhase 1

Los Angeles, California

This trial will evaluate the safety and effectiveness of a new treatment for Gaucher disease Type 1 in adults.

Substrate Reduction Therapy

Venglustat vs Imiglucerase for Gaucher Disease

Recruiting2 awardsPhase 3

Fairfax, Virginia

This trial compares a pill to injections in patients aged 12-18 and adults with Gaucher disease Type 3. The pill aims to help with brain symptoms, while the injections maintain overall health. The pill has the potential to improve Gaucher disease by rebalancing certain substances in the body.

Enzyme Replacement Therapy

In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

Recruiting1 awardPhase 1

San Francisco, California

This trial will test if a baby with a Lysosomal Storage Disease can be safely treated in the womb.

Enzyme Replacement Therapy

PR001 for Gaucher Disease

Recruiting1 awardPhase 1 & 2

Oakland, California

This trial is studying a new drug for infants with a rare disease called Gaucher disease. The study will last about 5 years, and patients will be monitored for safety and efficacy.

Frequently Asked Questions

Do I need insurance to participate in a trial?

Almost all clinical trials will cover the cost of the 'trial drug' — so no insurance is required for this. For trials where this trial drug is given alongside an already-approved medication, there may be a cost (which your insurance would normally cover).

Is there any support for travel costs?

Many of the teams running clinical trials will cover the cost of transportation to-and-from their care center.

Will I know what medication I am taking?

This depends on the specific study. If you're worried about receiving a placebo, you can actively filter out these trials using our search.

How long do clinical trials last?

Some trials will only require a single visit, while others will continue until your disease returns. It's fairly common for a trial to last somewhere between 1 and 6 months.

Do you verify all the trials on your website?

All of the trials listed on Power have been formally registered with the US Food and Drug Administration. Beyond this, some trials on Power have been formally 'verified' if the team behind the trial has completed an additional level of verification with our team.

How quickly will I hear back from a clinical trial?

Sadly, this response time can take anywhere from 6 hours to 2 weeks. We're working hard to speed up how quickly you hear back — in general, verified trials respond to patients within a few days.