Active not recruitingPHASE2INTERVENTIONAL

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

This research study, called AG348-C-010, is testing a new drug named AG-348 in adults who have a specific type of thalassaemia, known as non-transfusion-dependent thalassaemia (NTDT). The main goals are to see how well the medicine works, if it's safe, and how the body handles it. Twenty participants were involved, starting with a 50mg dose twice daily, which could be increased to 100mg based on their safety and blood test results. The study has a main treatment period of up to 24 weeks, followed by a longer extension period that could last up to 10 years for those who are suitable. This longer follow-up helps researchers understand the drug's effects over a much longer time.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Agios Pharmaceuticals, Inc.

Enrolment target

Start

20 Mar 2019

Estimated completion

30 Sep 2030

Thalassemia

Results

Results from this study

Posted November 2021

Results have been published for this study.

Primary outcome

Percentage of Participants Achieving a Hemoglobin Response (HR)

HR was defined as a ≥1.0 gram per deciliter (g/dL) increase in Hb concentration from Baseline at 1 or more assessments between Week 4 and Week 12 (inclusive). A participant's Baseline Hb concentration was defined as the average of all the participant's available Hb concentrations during the screening period up to the first dose of study drug.

Full results on the registry

What is this study about?

This study is investigating a new medication called AG-348 for adults living with a condition called thalassaemia. Thalassaemia is a genetic blood disorder where the body doesn't make enough healthy red blood cells. Red blood cells carry oxygen around your body, so not having enough can make you feel tired and weak, among other health problems. This particular study focuses on people with "non-transfusion-dependent thalassaemia" (NTDT), which means they generally don't need regular blood transfusions to manage their condition, unlike other forms of thalassaemia.

The main purpose of this research is to figure out if AG-348 can help improve the health of people with NTDT. Scientists want to understand how effective it is, meaning whether it makes a positive difference to symptoms or blood test results, and importantly, if it is safe to use. They also want to learn how the body absorbs, distributes, breaks down, and gets rid of the medicine.

Twenty adult participants took part in this study. They started with a dose of AG-348 taken twice a day, and this dose could be adjusted after six weeks depending on how they were feeling and their blood test results. The study was designed in two parts: an initial treatment period lasting up to 24 weeks, and then a much longer follow-up period of up to 10 years for those who continued to do well and met certain criteria. This long-term follow-up helps researchers gather detailed information on the drug's effects over many years.

Key takeaways

The study tests a new drug, AG-348, for adults with specific thalassaemia.
It aims to see if the drug is safe and helps improve health.
Participants take medication twice daily, with potential dose changes.
The study includes a short treatment period and a long-term follow-up.
Eligibility is for adults whose thalassaemia doesn't require regular blood transfusions.

Who may be eligible?

To join this study, participants needed to be adults aged 18 or older with a confirmed diagnosis of thalassaemia. Specifically, they must have had a type of thalassaemia where they didn't need regular blood transfusions. Their haemoglobin (a measure of red blood cells) levels also needed to be 10.0 grams per decilitre or lower, based on several blood tests.

Another key requirement was that they should not have received more than five units of blood transfusions in the 24 weeks before starting the study, and no transfusions at all in the eight weeks directly before starting. Participants also needed to be willing to follow all study instructions and have healthy organ function. Women able to become pregnant and men whose partners could become pregnant needed to use effective birth control during and for a period after the study.

People were not able to join if they had certain other types of thalassaemia (like Hb S or Hb C), had other serious medical conditions that might make the study risky or hard to understand, or had recently had or were scheduled to have surgery to remove their spleen. They also couldn't be taking part in another clinical study at the same time.

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.

Are you an adult (18 years or older)?
Do you have a type of thalassaemia that rarely or never needs blood transfusions?
Has it been at least 8 weeks since your last blood transfusion (if any)?
Are you willing to take study medication and attend regular appointments?
Are you using or willing to use effective birth control if applicable?

Answer every question to see your result.

What does participation involve?

If you took part in this study, you would have an initial treatment period lasting up to 24 weeks. During this time, you would take the study medication, AG-348, twice a day. The dose might start at 50mg twice daily and could be increased to 100mg twice daily after six weeks, depending on how your body reacted and based on blood test results. You would attend regular visits for health checks, blood tests to measure your *haemoglobin* levels and see how the drug is working, and to discuss any side effects you might be experiencing. After this initial period, if you met certain criteria and wished to continue, you could enter an extension period that could last up to 10 years. This would involve ongoing medication and regular follow-up visits to monitor your health and the long-term effects of the drug. You would be in the study for the entire duration of your participation, and you could choose to leave the study at any time.

Potential risks and benefits

Participating in research studies like this can offer potential benefits, such as access to a new medication not yet widely available, and close monitoring of your health by a medical team. However, there are also potential risks; the new drug AG-348 might cause side effects, some of which could be serious, and it might not work for everyone. The study team would explain all known risks before you decide to join. It's very important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UCSF Benioff Children's Hospital Oakland
Verified postcode
Oakland, United States
Massachusetts General Hospital
Verified postcode
Boston, United States
University Health Network (Toronto General Hospital)
Verified postcode
Toronto, Canada
Imperial College Healthcare NHS Trust (Hammersmith Hospital)
Verified postcode
London, United Kingdom

Common questions

What is thalassaemia?

Thalassaemia is a blood condition where your body doesn't produce enough healthy red blood cells, which carry oxygen around your body.

What does 'non-transfusion-dependent' mean?

It means you have a type of thalassaemia where you typically don't need regular blood transfusions to live, unlike some other forms of the condition.

What is AG-348?

AG-348 is a new medication being tested in this study to see if it can help improve the health of adults with non-transfusion-dependent thalassaemia.

How long does the study last?

The main treatment part of the study is up to 24 weeks, and then there's an optional longer follow-up period that could last up to 10 years.

Can I leave the study if I want to?

Yes, you are free to leave the study at any time, for any reason, and your medical care will not be affected.