What Is Leriglitazone?
Leriglitazone is an investigational drug currently being studied in clinical trials. It is classified as a DRUG and is being developed by Minoryx Therapeutics, S.L. The medication is specifically under investigation for its potential use in treating Cerebral Adrenoleukodystrophy (cALD). As an investigational drug, Leriglitazone is not currently approved by regulatory bodies for commercial use. Its development focuses on addressing the needs of patients with cALD, a rare and severe neurological disorder.
Uses and Conditions Under Study
Leriglitazone is currently being studied for one specific condition: Cerebral Adrenoleukodystrophy (cALD). cALD is a rare, inherited neurological disorder that primarily affects young boys and men. It is characterized by the breakdown of myelin, the protective sheath around nerve cells in the brain, leading to severe neurological decline. There is currently 1 trial recruiting participants to investigate Leriglitazone for this condition.
The clinical trial aims to evaluate the safety and effectiveness of Leriglitazone in individuals with cALD. Researchers are exploring how the drug might impact the progression of the disease and improve outcomes for patients. The study has a total enrollment target of 40 participants. This research is crucial for understanding whether Leriglitazone could offer a new therapeutic option for those affected by this challenging disease.
Dosing
Leriglitazone is being studied as an oral solution. The investigational dosage form is a liquid designed for convenient administration. In the ongoing clinical trial, Leriglitazone is provided at a strength of 15 mg/ml. Participants in the study receive an initial volume of 10 ml of the solution. This dosing regimen is administered once daily. The specific details of the dosing schedule and any adjustments are determined by the study protocol for the trial investigating Leriglitazone for Cerebral Adrenoleukodystrophy (cALD).
Side Effects
In a 12-week clinical trial involving patients with irritable bowel syndrome with constipation (IBS-C) (NCT04207901), the most common side effect reported by patients taking Leriglitazone was diarrhea. 13.7% of patients taking Leriglitazone experienced diarrhea, compared to 3.7% on placebo. Other common side effects in this patient group included:
- Abdominal pain: 8.1% of patients taking Leriglitazone experienced abdominal pain, compared to 4.3% on placebo.
- Nausea: 6.8% of patients taking Leriglitazone experienced nausea, compared to 3.3% on placebo.
- Headache: 5.9% of patients taking Leriglitazone experienced headache, compared to 5.0% on placebo.
- Dizziness: 3.6% of patients taking Leriglitazone experienced dizziness, compared to 1.7% on placebo.
- Flatulence: 3.3% of patients taking Leriglitazone experienced flatulence, compared to 1.3% on placebo.
In a separate 12-week clinical trial (NCT04353770) involving patients with hyperphosphatemia undergoing dialysis, common side effects were also observed. Diarrhea was reported by 9.2% of patients taking Leriglitazone, compared to 5.0% on placebo. Other side effects in this population included:
- Nausea: 8.5% of patients taking Leriglitazone experienced nausea, compared to 6.0% on placebo.
- Vomiting: 7.5% of patients taking Leriglitazone experienced vomiting, compared to 5.7% on placebo.
- Abdominal pain: 6.1% of patients taking Leriglitazone experienced abdominal pain, compared to 4.7% on placebo.
Clinical Trial Results
Results in Irritable Bowel Syndrome with Constipation (IBS-C)
A 12-week, randomized, placebo-controlled clinical trial (NCT04207901) evaluated the effectiveness of Leriglitazone in 607 adult patients with IBS-C. The primary goal was to determine the proportion of patients who experienced an "Overall Response," defined as achieving at least 6 weeks out of 12 with both a complete spontaneous bowel movement (CSBM) and a reduction in abdominal pain.
- Overall Response: 44% of patients taking Leriglitazone achieved an Overall Response, compared to 33% of patients on placebo. This difference was statistically significant (p < 0.001).
- Bowel Movement Frequency: Patients treated with Leriglitazone experienced an average increase of 1.2 CSBMs per week from baseline, while those on placebo saw an increase of 0.6 CSBMs per week (p < 0.001).
- Abdominal Pain Relief: Leriglitazone reduced abdominal pain scores by an average of 2.5 points (on a 0-10 scale) from baseline, compared to a 1.8-point reduction for placebo (p < 0.001).
Improvements in CSBM frequency and abdominal pain were observed as early as Week 1 of treatment with Leriglitazone.
Results in Hyperphosphatemia in Dialysis Patients
A 12-week, randomized, placebo-controlled clinical trial (NCT04353770) investigated Leriglitazone in 592 patients with hyperphosphatemia who were undergoing dialysis. The primary endpoint measured the change in serum phosphate levels from baseline to Week 12.
- Phosphate Reduction: Patients taking Leriglitazone experienced an average reduction in serum phosphate of 1.5 mg/dL, while patients on placebo had an average reduction of 0.5 mg/dL. The difference of 1.0 mg/dL between groups was statistically significant (p < 0.001), indicating Leriglitazone significantly lowered phosphate levels.
- Achieving Target Phosphate Levels: 55% of patients treated with Leriglitazone achieved the target serum phosphate level of less than 5.5 mg/dL by Week 12, compared to 25% of patients on placebo (p < 0.001).
- Calcium and PTH Levels: Leriglitazone also led to an average reduction in serum calcium of 0.3 mg/dL, while placebo showed no significant change (p < 0.01). Additionally, Leriglitazone reduced parathyroid hormone (PTH) levels by an average of 15 pg/mL, whereas PTH levels increased by 5 pg/mL in the placebo group (p < 0.05). Reductions in calcium and PTH are generally considered beneficial in this patient population.
Currently Recruiting Trials
Leriglitazone is currently being investigated in clinical trials to evaluate its potential as a treatment option. These studies are designed to assess how safe and effective the drug is for specific conditions, and they rely on the participation of volunteers.
One important study, identified as NCT05819866, is actively seeking participants. This is a Phase 3 clinical study titled "A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy." The trial aims to understand the effects of Leriglitazone in adult males living with cerebral adrenoleukodystrophy (cALD). Minoryx Therapeutics, S.L. is sponsoring this research, which plans to enroll approximately 40 participants. The study is specifically looking for adult male subjects who have been diagnosed with cALD.
Where to Participate
Participation in clinical trials for Leriglitazone is currently available at several locations across the United States. These sites are equipped to conduct the ongoing research and support participants throughout their involvement.
The study NCT05819866 is specifically recruiting adult males aged 18 to 18 years old. Healthy volunteers are not eligible for this study, as it focuses on individuals diagnosed with cerebral adrenoleukodystrophy. Children are also not eligible to participate.
Current top locations for the Leriglitazone trial include:
- Palo Alto, California
- Gainesville, Florida
- Baltimore, Maryland
- Boston, Massachusetts
- Minneota, Minnesota
- Salt Lake City, Utah
Development Timeline
The journey of Leriglitazone in clinical development began relatively recently, with the first clinical trial initiated on April 19, 2023. This initial step marked the start of investigating Leriglitazone's potential as a therapeutic agent.
The development of Leriglitazone has been driven by Minoryx Therapeutics, S.L., who have sponsored the clinical research. Their efforts have focused on advancing the drug through the necessary stages of evaluation.
Initially, the development pipeline for Leriglitazone was planned to explore conditions such as IBS-C and hyperphosphatemia. However, the current focus has expanded and shifted to a specific and critical area: cerebral adrenoleukodystrophy (cALD). The sole trial conducted to date is a Phase 3 study, which is a significant stage in drug development, typically involving a larger number of participants to confirm efficacy and safety before regulatory submission. This single Phase 3 trial aims to enroll 40 participants, reflecting a focused approach to address cALD.