Improvements were seen within 4 weeks

 

Lower levels of toxins were achieved in participants taking CTEXLI vs those who took placebo

Patients taking CTEXLI saw lower toxin levels in the blood and urine after 4 weeks on treatment compared with those on placebo.

 

With ongoing CTEXLI treatment, the reduction in CTX toxins lasts over time

Toxin levels in the blood and urine stayed consistently low with continued CTEXLI use.

 

When participants stopped taking CTEXLI, CTX toxin levels increased

When patients switched from CTEXLI to placebo, CTX toxins in the blood and urine increased within 2 weeks.

How was CTEXLI studied?

CTEXLI was evaluated in a 24-week clinical study

The purpose of the study was to see if CTEXLI helps reduce the production of 2 key CTX toxins.

13* people aged 16 or older with CTX participated in the study. It was a randomized, double-blind study. This means neither the participants nor the doctors knew who received CTEXLI or placebo. Placebo looked like a "real" medicine, but did not have any active medicine.

CTEXLI study design and timeline
  • All 13 participants received CTEXLI for the first 8 weeks
  • Participants were divided into 2 groups for the next 4 weeks:
  • One group received CTEXLI
  • The other group received a placebo
  • Both groups received CTEXLI again for 8 weeks
  • In the final 4 weeks, the groups switched treatments:
  • The group that had CTEXLI received placebo
  • The group that had placebo received CTEXLI

*14 participants signed up for the study, but only 13 were ultimately included.

Since stopping CTEXLI may cause toxins in the blood and urine to go back up, it's important to keep taking CTEXLI as prescribed by your health care provider.

Learn About Taking CTEXLI

CTEXLI has an established FDA-approved safety profile

The safety of CTEXLI was carefully studied in a clinical trial. The most common side effects of CTEXLI are diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory infections.

Hepatotoxicity is a potential serious side effect of CTEXLI. You will need to undergo laboratory (blood) testing before starting and periodically while on treatment with CTEXLI to assess liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury.

People with preexisting liver disease or bile acid abnormalities may be at a higher risk for liver injury during treatment.

Stop taking CTEXLI immediately and tell your health care provider right away if you get any signs or symptoms of liver problems, including:

  • Stomach (abdominal) pain
  • Bruising
  • Dark-colored urine
  • Feeling tired
  • Bleeding
  • Yellowing of the skin and eyes
  • Nausea
  • Itching

Your doctor may change your dose or temporarily or permanently stop treatment with CTEXLI if you have certain side effects. These are not all the possible side effects of CTEXLI. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Mirum Pharmaceuticals at 1-855-MRM-4YOU (1-855-676-4968).

Talk to a health care provider to learn more about CTEXLI.