ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
This is an ongoing study, called ELEVATE, for people living with Acute Hepatic Porphyria (AHP). It's a global project collecting information to better understand this rare condition. Researchers want to see how AHP naturally affects people over time and how doctors are treating it in everyday practice. They are especially interested in gathering real-world information on the safety and effectiveness of approved AHP medications, including a newer one called givosiran. By collecting this information, the study hopes to improve our understanding of AHP and potentially lead to better care in the future. It's not testing a new treatment, but rather observing how existing treatments are used and how the condition develops in the real world.
At a glance
What is this study about?
This study, known as ELEVATE, is a bit different from a typical clinical trial. Instead of testing a new medicine, it's a 'registry'. Think of it like a comprehensive patient database specifically designed for people living with Acute Hepatic Porphyria (AHP). The main goal is to build a clearer picture of what it's like to live with AHP today.
Researchers are collecting information from patients over time to understand the 'natural history' of the condition – that means observing how AHP progresses and impacts people's lives without interference. They also want to see how doctors are currently managing AHP in real-world settings, including which treatments are being used and how effective they are. This includes gathering data on new approved treatments for AHP, such as givosiran, to see how they perform in broader use.
By carefully collecting this information from many people across different countries, the study hopes to fill gaps in our understanding of AHP. This knowledge is crucial for doctors and researchers to develop better care strategies, improve diagnoses, and ultimately enhance the quality of life for people with AHP. It helps them understand what works best in practice and where more support or different treatments might be needed.
Key takeaways
- It's a registry, not a trial of a new drug.
- Aims to understand AHP's progression and real-world treatments.
- Includes data on approved medications like givosiran.
- Open to people with AHP aged 12 and older.
- Participation involves sharing medical information, not new procedures.
- Helps improve future AHP care.
Who may be eligible?
To take part in this registry study, you must have a confirmed diagnosis of Acute Hepatic Porphyria (AHP). This diagnosis needs to be made by your doctor. The study is open to both men and women, aged 12 years and older – there's no upper age limit.
You wouldn't be able to join this particular registry if you are currently taking part in another clinical trial where you are receiving an experimental or investigational medicine. This is to ensure the data collected accurately reflects real-world treatment and progression, rather than the effects of a study drug.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do I have a confirmed diagnosis of Acute Hepatic Porphyria (AHP) from my doctor?
- Am I at least 12 years old?
- Am I currently NOT taking part in another clinical trial testing an experimental medicine?
What does participation involve?
If you decide to take part in this registry, it largely involves allowing your medical information related to your AHP to be collected. This typically means that your doctors will provide de-identified health data to the registry over time. There are no new treatments to take, no special appointments to attend for the study itself, and no new medical procedures you’d need to undergo. The study gathers information from your regular medical care. The length of time you participate would depend on the study design, but registries like this usually follow participants for an extended period to observe long-term trends.
Potential risks and benefits
Locations (30)
- Clinical Trial SiteVerified postcodeBirmingham, United States· Recruiting
- Clinical Trial SiteVerified postcodeGilbert, United States· Recruiting
- Clinical Trial SiteVerified postcodeSan Francisco, United States· Recruiting
- Clinical Trial SiteVerified postcodeBaltimore, United States· Recruiting
- Clinical Trial SiteVerified postcodeBoston, United States· Recruiting
- Clinical Trial SiteVerified postcodeMinneapolis, United States· Recruiting
- Clinical Trial SiteVerified postcodeNew York, United States· Recruiting
- Clinical Trial SiteVerified postcodeWinston-Salem, United States· Completed
- Clinical Trial SiteVerified postcodeOklahoma City, United States· Recruiting
- Clinical Trial SiteVerified postcodePhiladelphia, United States· Completed
- Clinical Trial SiteVerified postcodeGalveston, United States· Recruiting
- Clinical Trial SiteVerified postcodeSalt Lake City, United States· Recruiting
Common questions
What is a patient registry?
A patient registry is like a large collection of health information from people with a specific condition. It helps researchers understand the condition better over time.
Do I have to take new medication?
No, this study is not testing a new medication. It's observing how people with AHP are managed with their usual care and existing approved treatments.
Will my personal details be kept private?
Yes, highly confidential measures are in place to protect your privacy. Your personal identifying information will be kept separate from the data collected for the study.
How long will I be involved?
Registries often follow participants for several years to gather long-term information. Your medical team can provide more specific details.
Does taking part affect my regular medical care?
No, participating in this registry will not change your medical care. You will continue to see your own doctors for your AHP treatment as usual.
How to find out more
Alnylam Clinical Trial Information Line
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
Discussion
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