Clinical trials for PNH

Overview
Depending on several factors, you may be eligible to participate in clinical trials studying new treatments for paroxysmal nocturnal hemoglobinuria (PNH). Clinical trials are the only way that new treatments for PNH can be tested. Eligibility factors may include your age, your overall health condition, and which treatments you’ve already tried.

Clinical trials may test new drugs, new dosages, or new combinations of existing drugs, new delivery methods of drugs, or the effect of lifestyle interventions such as diet, exercise, and supplements. Trials may also pair standard treatments with complementary practices such as yoga or acupuncture. Quality-of-life or supportive care trials may be evaluating new palliative approaches to better control symptoms.

What all clinical trials have in common is that every participant receives at least the current standard of treatment, and the knowledge gained from the trial may benefit other people with PNH.

What does it involve?
Before you enroll in a clinical study, read the informed consent form very closely. Ask your doctor or the doctor in charge of the study to explain any portions you have trouble understanding. Pay close attention to descriptions of the potential risks and benefits associated with participation. Work with your medical team to decide whether a clinical trial is right for you.

You can withdraw from a clinical trial at any time.

Intended outcomes
The goal of participating in a clinical trial is to receive the highest possible standard of treatment for PNH and further research to better help others living with the condition.

Constraints
Although the new treatment may be more effective than the standard treatment against PNH, it may not be effective in your specific case.

If the new treatment proves effective for you, you may lose access to it after the trial is completed.

The new treatment may cause unforeseen side effects. Although the doctors leading the clinical trial will educate you about known risks, it’s impossible to know what might happen with a novel treatment.

Some insurance companies may not cover all costs associated with being in clinical trials. However, any remaining costs are often covered by the organization sponsoring the clinical trial. Be sure to ask about costs before enrolling. Find out which medical expenses will be paid by the trial, whether the sponsor will cover any care resulting from injury or damage you may receive as part of the trial, and whether long-term aftercare is included.

Some trials may be located far from your home and require travel.

For more details about this treatment, visit:

ClinicalTrials.gov
https://clinicaltrials.gov/