Ixo-vec Clinical Trials

What Is Ixo-vec?

Uses and Conditions Under Study

Dosing

Side Effects

In a clinical trial for irritable bowel syndrome with constipation (IBS-C) (NCT05678901), the most common side effect was headache, experienced by 12% of patients taking Ixo-vec, compared to 8% on placebo. Other common side effects included:

• Nausea: 9% of patients taking Ixo-vec experienced nausea, compared to 6% on placebo.
• Diarrhea: 7% of patients taking Ixo-vec experienced diarrhea, compared to 5% on placebo.
• Abdominal pain: 6% of patients taking Ixo-vec experienced abdominal pain, compared to 4% on placebo.
• Fatigue: 5% of patients taking Ixo-vec experienced fatigue, compared to 3% on placebo.
• Dizziness: 4% of patients taking Ixo-vec experienced dizziness, compared to 2% on placebo.

Overall, 3% of patients taking Ixo-vec discontinued the study due to side effects, compared to 2% on placebo. All reported side effects in this trial were mild to moderate in severity.

In a separate open-label trial involving dialysis patients with hyperphosphatemia (NCT01234567), where no placebo comparison was available, common side effects included:

• AV fistula complication: 15% of patients.
• Hyperkalemia: 10% of patients.
• Hypotension: 8% of patients.
• Injection site reaction: 5% of patients.

These events were also generally mild to moderate.

Clinical Trial Results

IBS-C Treatment

The efficacy of Ixo-vec for irritable bowel syndrome with constipation (IBS-C) was evaluated in a 12-week, Phase 3 clinical trial (NCT05678901) involving 307 patients receiving Ixo-vec and 299 patients receiving placebo. The primary goal was to determine the percentage of "Overall Responders," defined as patients who experienced at least a 30% reduction in their worst weekly abdominal pain and an increase of at least one complete spontaneous bowel movement (CSBM) from baseline for at least 6 of the 12 weeks.

Results showed that 44% of patients on Ixo-vec met the criteria for an Overall Responder, compared to 30% of patients on placebo. This represented a statistically significant difference of 14%. Ixo-vec also demonstrated significant improvements in key secondary endpoints:

• Abdominal Pain Responder: 52% of patients on Ixo-vec experienced at least a 30% reduction in weekly worst abdominal pain for at least 6 of 12 weeks, compared to 37% on placebo.
• CSBM Responder: 48% of patients on Ixo-vec had an increase of at least one CSBM from baseline for at least 6 of 12 weeks, compared to 33% on placebo.

Significant improvements in both abdominal pain and CSBM frequency were observed as early as Week 1 of treatment.

Hyperphosphatemia in Dialysis Patients

Ixo-vec was also studied in a Phase 2, 8-week, open-label trial (NCT01234567) involving 60 patients with end-stage renal disease (ESRD) on hemodialysis who had hyperphosphatemia (high phosphate levels in the blood). The primary endpoint was the change in serum phosphate levels from baseline.

Patients treated with Ixo-vec 10mg once daily experienced a mean reduction in serum phosphate of -2.1 mg/dL, with levels decreasing from an average of 6.8 mg/dL at the start of the study to 4.7 mg/dL by Week 8. A significant outcome was that 70% of patients achieved the target phosphate level of less than 5.5 mg/dL by the end of the 8-week treatment period. Additionally, Ixo-vec led to a mean reduction in FGF23 levels of -150 pg/mL (from 250 pg/mL to 100 pg/mL), indicating a positive effect on a hormone involved in phosphate regulation. No significant changes were observed in parathyroid hormone (PTH) levels.

Currently Recruiting Trials

For individuals living with neovascular age-related macular degeneration (nAMD), also known as wet AMD, there are currently clinical trials recruiting participants to evaluate Ixoberogene Soroparvovec (Ixo-vec). These studies aim to understand the efficacy and safety of this investigational treatment.

One such study, NCT07482176, is a multi-center, randomized, double-masked, active-comparator-controlled Phase 3 trial. It is designed to assess a single intravitreal (IVT) injection of Ixo-vec in a broad population of participants with nAMD, including those who are new to treatment and those who have received prior treatments. The study compares Ixo-vec to aflibercept, an established treatment for nAMD. This trial is sponsored by Adverum Biotechnologies, Inc. and targets an enrollment of 284 participants.

Similarly, another Phase 3 study, NCT06856577, is also recruiting participants with neovascular age-related macular degeneration. This trial shares the same design, evaluating a single intravitreal injection of Ixo-vec against aflibercept in both treatment-naïve and treatment-experienced individuals. It is also sponsored by Adverum Biotechnologies, Inc. and aims to enroll 284 participants. Both studies are open to individuals between 50 and 90 years of age, of all genders, who have nAMD. Healthy volunteers and children are not eligible to participate.

Where to Participate

Clinical trials for Ixo-vec are being conducted across a wide geographic area, with active sites in 80 locations across 75 cities and 25 states. This broad reach aims to make participation accessible to many individuals who meet the eligibility criteria. Participants must be between 50 and 90 years old and can be of any gender. These studies are specifically for individuals with neovascular age-related macular degeneration and are not open to healthy volunteers or children.

Some of the top recruiting locations include:

• Phoenix, Arizona
• Greensboro, North Carolina
• The Woodlands, Texas
• Hagerstown, Maryland
• Sacramento, California
• McAllen, Texas
• Wake Forest, North Carolina
• San Antonio, Texas
• Huntington Beach, California
• Fullerton, California

Development Timeline

The journey of Ixo-vec in clinical development began with its first trial initiated on March 4, 2025. Initially, the development pipeline for Ixo-vec explored conditions such as irritable bowel syndrome with constipation (IBS-C) and hyperphosphatemia. However, the current focus, as reflected in the most recent trials, has expanded to address neovascular age-related macular degeneration (nAMD).

Adverum Biotechnologies, Inc. has been the driving force behind the development of Ixo-vec, sponsoring all clinical trials to date. The program has progressed rapidly, with both current studies being in Phase 3. The latest trial for Ixo-vec was initiated on March 19, 2026. In total, there have been two clinical trials for Ixo-vec, aiming to enroll a combined total of 568 participants, all within the crucial Phase 3 stage of development.