Bitopertin for Erythropoietic Protoporphyria

(APOLLO Trial)

Recruiting at 15 trial locations

Overseen By Disc Medicine Clinical Trials

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Disc Medicine, Inc

Must not be taking: Opioids, Antipsychotics, CYP3A4 inhibitors

Disqualifiers: Surgery, Anemia, Liver transplant, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

150 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if the drug bitopertin can help individuals with Erythropoietic Protoporphyria (EPP) or X-Linked Protoporphyria (XLP) spend more time in sunlight without pain. Researchers will also examine changes in levels of a specific blood compound after six months of taking the drug. Participants will receive either bitopertin or a placebo (a harmless pill with no active drug) and will need to complete daily questionnaires. Individuals diagnosed with EPP or XLP who struggle with sun exposure might be suitable for this trial. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment nearing widespread availability.

Will I have to stop taking my current medications?

The trial requires a washout period (time without taking certain medications) of at least 2 months for afamelanotide and dersimelagon before screening. You also cannot use opioids for more than 7 days in the 2 months before screening or during the study, and you must avoid certain drugs or herbal remedies that affect liver enzymes for 28 days before starting the trial and throughout the study.

Is there any evidence suggesting that bitopertin is likely to be safe for humans?

Research has shown that bitopertin is generally well-tolerated. A study involving over 4,000 patients found that the safety of bitopertin matched previous results. Although specific side effects were not listed, the large number of participants provides insight into how the drug works in the body. This information offers reassurance about its safety for treating conditions like Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP). ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for EPP?

Unlike the standard treatments for Erythropoietic Protoporphyria, which generally focus on managing symptoms with things like beta-carotene or Afamelanotide, Bitopertin acts on a different level. Bitopertin is unique because it targets the glycine transporter, which is a new approach for this condition. This mechanism potentially reduces the production of toxic compounds responsible for the painful light sensitivity experienced by patients. Researchers are excited about Bitopertin as it offers a novel way to address the root cause of symptoms, rather than just alleviating them.

What evidence suggests that bitopertin might be an effective treatment for EPP or XLP?

Research shows that bitopertin might help treat EPP (erythropoietic protoporphyria) by lowering levels of protoporphyrin IX (PPIX) in the blood. In earlier studies, patients who took bitopertin experienced a significant and lasting drop in PPIX levels compared to those on a placebo. Another study found that bitopertin increased the percentage of people who could be in sunlight without symptoms from 7% to 55%. In animal studies, bitopertin reduced PPIX levels by more than 40%. These findings suggest that bitopertin can help people with EPP tolerate sunlight better by reducing PPIX in their blood. Participants in this trial will receive either a placebo or an oral dose of DISC-1459, which is under study for its potential effects on EPP. ^{1 3 6 7 8}

Who Is on the Research Team?

Will Savage, MD, PhD

Principal Investigator

Disc Medicine

Are You a Good Fit for This Trial?

This trial is for individuals aged 12 or older with a diagnosis of EPP or XLP, confirmed by genetic testing or porphyrin analysis. They must have liver function within certain limits and agree to use effective birth control. Participants need to weigh at least 32 kg if under 18, or have a BMI over 18.5 if adult.

Inclusion Criteria

I have been diagnosed with EPP or XLP through genetic testing or porphyrin analysis.

My liver tests are within the required range.

I have kept a sun exposure diary with at least 85% accuracy for the last 14 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either bitopertin or a placebo for 6 months to evaluate efficacy, safety, and tolerability

6 months

Regular study visits for assessments

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Bitopertin

Trial Overview The study tests bitopertin against a placebo in people with EPP/XLP to see if it allows them more pain-free time in sunlight after six months and how it affects PPIX levels. It involves daily questionnaires and regular visits for assessments.

How Is the Trial Designed?

Group I: DISC-1459 oral dose Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Disc Medicine, Inc

Lead Sponsor

Trials

6

Recruited

580+

Citations

1. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0006497124038369

Results from the Aurora Study: A Phase 2, Randomized, ... Treatment with bitopertin resulted in significant and sustained reductions in PPIX levels compared to PPIX levels for treatment with placebo at both the 20-mg ...

2. ashpublications.org ashpublications.org/blood/article/144/Supplement%201/1093/529163/Results-from-the-Beacon-Trial-A-Phase-2-Randomized

Results from the Beacon Trial: A Phase 2, Randomized, Open ... Bitopertin improved other measures of life tolerance; the proportion of prodrome-free sunlight challenges increased from 7% at screening to 55% ...

3. ir.discmedicine.com ir.discmedicine.com/news-releases/news-release-details/disc-reports-topline-results-phase-2-aurora-study-bitopertin

Disc Reports Topline Results from Phase 2 AURORA ... Treatment with bitopertin resulted in statistically significant reductions in PPIX, the primary endpoint, and significant improvements in the rate of ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10429836/

P1498: INITIAL DATA FROM THE BEACON TRIAL: A ... In murine models of EPP and XLP, treatment with bitopertin lowered blood PPIX levels by >40% compared to controls. Bitopertin treatment in EPP mice also lowered ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06910358

Study of Bitopertin in Participants With EPP or XLP (APOLLO) The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05308472

NCT05308472 | Study of Bitopertin to Evaluate the Safety, ... This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX ...

7. discmedicine.com discmedicine.com/news/disc-medicine-initiates-beacon-a-phase-2-clinical-study-of-bitopertin-in-patients-with-erythropoietic-protoporphyria-epp-and-x-linked-protoporphyria-xlp/

Disc Medicine Initiates BEACON, a Phase 2 Clinical Study ... BEACON study will evaluate bitopertin as a potential disease-modifying treatment for patients with Erythropoietic Protoporphyria (EPP) or X-linked ...

8. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT06910358

Study of Bitopertin in Participants With EPP or XLP (APOLLO) The goal of this clinical trial is to learn if bitopertin works and is safe to treat EPP or XLP in participants 12 years or older.