Alport syndrome

At ENYO Pharma, patients with impaired kidney function are in the center of our discussions.

We are committed every day to develop new therapeutics for improving the quality of life of patients with Alport syndrome. Working closely with our patient advocacy partners, as key players as representatives of patients, families and caregivers, support our best strategy for clinical study development in Alport syndrome.

ALPESTRIA-1 clinical trial

We are aiming to preserve kidney function and reverse kidney damage with the investigational drug Vonafexor tested in our Phase 2 clinical study named “ALPESTRIA-1”.

Alpestria-1 clinical study

What is the ALPESTRIA-1 clinical study?

The Alport syndrome Efficacy and Safety Trial-1

  • Objectives: The ALPESTRIA-1 study is a Phase 2 clinical study evaluating the safety, the tolerability and the benefit of three dose levels of the investigational drug Vonafexor on renal function and biomarkers in Alport syndrome patients.
  • Design: The study is an open label and single arm study with a fixed dose-escalation. There is no placebo group (all subjects will receive study drug).
  • Treatment: All participants will receive all three dose levels of Vonafexor as oral tablets once daily for 24 weeks then stop the treatment for 12 weeks in the follow-up period.
  • Assessments: The study offers assessments with a mix of 5 site visits, 4 home visits and 4 phone calls, with urine and/or blood sampling.
  • Enrollment: The study aims at enrolling 20 patients in US and EU (France, Germany and Spain).
Vonafexor

What is Vonafexor?

The investigational drug for ALPESTRIA-1 clinical study

  • Drug: Vonafexor is a small molecule with strong fibrolytic and anti-inflammatory properties in both organs, kidney and liver. Learn more about Vonafexor
  • Regulatory: Vonafexor was granted with Orphan Drug Designation (ODD) by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) in 2023.
Participant's eligibility

Can I participate in the ALPESTRIA-1 study?

I may be eligible if:

  • Age: I am 18–55 years of age in EU or 16-55 years of age in US
  • Sex: I am a woman or a man
  • Diagnosis: I have been diagnosed with Alport syndrome & it was confirmed with a genetic test
  • Kidney function: I have a mild to moderate loss of kidney function
  • Proteinuria/albuminuria: I have an increased proteinuria and/or albuminuria
  • Treatment: I can be on ACEi, ARB or SGLT2i treatment
Status and site locations

Study status and site locations

The study will be conducted in both US and Europe

  • Study status: Recruiting
  • Site locations: The study will be conducted in US, France, Spain and Germany.

All study sites are displayed below. To get information on a specific site, please select on the list or click the pin in the map. Click on the +/- signs or scroll up/down to zoom in/out on the map.

Boise Kidney and Hypertension Institute

Address: 1070 N. Curtis Road Suite 130, Boise,
ID 83706, US

Website: Boise Kidney & Hypertension

Contact: Dr. Arnold Silva ; Send email

Cleveland Clinic Foundation, Kidney Medicine
Address: 9500 Euclid Avenue, Cleveland, OH 44195, US
Website: Cleveland Clinic Foundation
UCLA Health, David Geffen School of Medicine, Department of Nephrology
Address: UCLA Nephrology Office, 700 Tiverton Ave, CHS-Factor Room 7-155, Los Angeles, CA 90095, US
Website: UCLA Health
NANI Clinical Research
Address: 911 N Elm Street, Hinsdale, IL 60521, US
Website: NANI Clinical Research
Columbia University Medical Center, Division of Nephrology
Address: 622 W 168th Street, PH4, Rm 124, New York, NY 10032, US
Website: Columbia University Medical Center
Washington University in St Louis , Division of Nephrology
Address: Campus Box 8126, 600 South Euclid Avenue, St. Louis, MO 63110, US
Website: Washington University in St Louis
Renal Disease Research Institute
Address: 411 N. Washington Avenue, Suite 6000, Dallas, TX 75126
Website: Renal Disease Research Institute
University of Alabama at Birmingham, Division of Nephrology
Address: 1720 2nd Ave S ZRB 604 Birmingham, AL 35294, US
Website: University of Alabama at Birmingham
Hôpital Necker Enfants Malades, Service de Nephrologie – Dialyse Adultes
Address: 149 rue de Sevres, 75015 Paris, France
Website: Hôpital Necker Enfants Malades
Hôpital Lapeyronie, Service de Nephrologie, Dialyse et Transplantation
Address: 371 Av. du Doyen Gaston Giraud, 34090 Montpellier , France
Website: Hôpital Lapeyronie
CHU de Bordeaux, Service de Nephrologie Dialyse Transplantation
Address: Place Amelie Raba Leon, 33076 Bordeaux, France
Website: CHU de Bordeaux
Hôpital de la Conception, Centre de néphrologie et transplantation rénale
Address: 147 Bd Baille, 13385 Marseille, France
Website: Hôpital de la Conception
Hospital Virgen de la Arrixaca, Dept. Nephrology
Address: Cartagena km 7, Murcia 30120, Spain
Website: Hospital Virgen de la Arrixaca
Fundacio Puigvert, Dept. Nephrology
Address: Cartagena 340, 08025 Barcelona, Spain
Website: Fundacio Puigvert
Hospital De Sagunto, Internal Medicine
Address: Av/Ramon y Cajal, SN, 46520/Sagunto/Valencia, Spain
Website: Hospital De Sagunto
Fundacion Jimenez Diaz, Nephrology and Hypertension
Address: Av Reyes Catolicos 2, 28040, Madrid, Spain
Website: Fundacion Jimenez Diaz
Charite Universitatsmedizin Berlin, Dept. Nephrology and Medical Intensive CareAddress: Chariteplatz 1, 10117 Berlin
Website: Charite Universitatsmedizin Berlin
University Medicine Göttingen, Dept. Nephrology and Rheumatology
Address: Robert-Koch Str. 40, 37075 Goettingen
Website: University Medicine Göttingen

Lern more about the study

Learn more about the study

  • If you have any questions about the clinical study, please speak with your physician, visit NCT06425055 or Alport Syndrome Foundation website.
  • To download the “Alpestria-1 clinical study” flyer click below
  • Download ALPESTRIA-1 flyer

    ALPESTRIA-1 clinical trial

    Patient advocacy partnership

    Patient advocacy organizations support patients and families by providing educational resources, building community, raising awareness, providing access to information from research and clinical experts, amplifying the patient voice, creating opportunities for patients to learn from and support each other, and organizing patient participation in research.

    Alport Syndrome Foundation

    Alport Syndrome Foundation

    Alport Syndrome Foundation is a patient-led non-profit organization in the United States dedicated to improving the lives of those affected by Alport Syndrome.

    About Alport syndrome

    • Disease overview: Alport syndrome is a rare kidney disease based on an inherited defect in type IV collagen, a structural material required for kidney, eye, and ear function.
      Learn morePrimary symptom is hematuria (blood in the urine) early in life followed by proteinuria (presence of proteins in the urine), hypertension and oedema. Together, these symptoms lead to a progressive decline of kidney function and ultimately to end-stage renal disease (ESRD). Risk for disease progression depends on many factors including the genetic type and variant of patients. Individuals living with Alport syndrome often develop hearing loss and eye conditions.
    • Mode of inheritance: There are three genetic types of Alport syndrome.
      Learn moreThe most common is X-Linked Alport Syndrome (XLAS) based on COL4A5 collagen IV gene mutation where hematuria history (+/- proteinuria) or renal failure often occur in the family. XLAS represents approximately 74% of identified patient population. The other genetic types are Autosomal Dominant Alport syndrome (ADAS) and Autosomal Recessive Alport syndrome (ARAS) based on COL4A3 or COL4A4 collagen IV gene mutation, respectively. ARAS type represents 17% and ADAS type represents 9% of Alport syndrome patients.
    • Incidence: Alport syndrome is the second most common inherited kidney disease, slightly behind autosomal-dominant polycystic kidney disease (ADPKD).
      Learn moreThe global incidence of Alport syndrome is unknown as individuals are underdiagnosed or misdiagnosed. However, Alport syndromes is estimated to affect 1:5000 – 10,000 live births in the US (XLAS: 1/2000 births; ARAS: 1/10,000 births; ADAS: 1/100 births).
    • Treatment: There is currently no cure for Alport syndrome and no currently available treatment options have demonstrated efficacy in helping patients avoid ESRD.
      Learn moreCurrent standard of care are therapeutics used for Chronic Kidney Disease (CKD), including angiotensin converting enzyme inhibitors (ACEis) or angiotensin receptor blockers (ARBs). These medications have negative side effects, are not tolerated by all patients, can exacerbate hyperkalemia, and do not ultimately lead to patients avoiding dialysis and kidney transplant.

    REFERENCES:

    • ASF, Alport Syndrome Foundation: Alport Syndrome Foundation
    • NORD, National Organization for Rare Disorders (US): NORD
    • Orphanet (EU): Orphanet
    • Global data. Alport Syndrome Marketed and Pipeline Drugs Assessment, Clinical Trials and Competitive Landscape report. May 27, 2022. Global data