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ONT01 for Lupus Nephritis and Systemic Lupus Erythematosus

This study is testing a drug called ONT01 for people with Lupus Nephritis (a kidney condition caused by lupus) or Systemic Lupus Erythematosus (SLE), which is a chronic autoimmune disease. ONT01 is taken by mouth twice a day. The main goals are to see if ONT01 is safe and well-tolerated, and if it can improve lupus symptoms when used with other common lupus treatments like mycophenolate mofetil. You may be able to join if you are at least 18 years old, have been diagnosed with SLE, and have active lupus that affects parts of your body other than your kidneys, and have tried at least one other lupus medication (not including hydroxychloroquine). The study plans to enroll 61 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It aims to enroll 61 participants.
What's involved
You would take ONT01 orally twice daily, approximately 12 hours apart, under fasted conditions (2 hours before and 1 hour after each dose) with water. The study will track your progress from enrollment until 18 weeks.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed from enrollment until 18 weeks after the study begins.

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NCT07107659

Safety and Efficacy of ONT01 in Lupus

Recruiting
EARLY_PHASE1Ages 18+InterventionalTreatment
Hospital for Special Surgery, New York
~61 participants
Updated 2025-11-20 on ClinicalTrials.gov
What's tested:ONT01 is a small molecule CD11b modulator.

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Adverse Events, Study Completion versus Withdrawal
Measured over From Enrollment until 18 weeks

Conditions

Lupus Nephritis (LN)
Lupus Nephritis - WHO Class III
Lupus Nephritis - WHO Class IV
Lupus Nephritis - World Health Organization (WHO) Class III
Lupus
SLE
Systemic Lupus Erythematosus
Systemic Lupus Erythematosus (Disorder)

Explore related trials

Lupus GlomerulonephritisSystemic Lupus Erythematosus

Where it's being run

1 sites across 1 states
New York1

Who to contact

Timothy B Niewold, MD
[email protected]
646 797-8698
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Eligibility criteria

Inclusion

Active non-renal SLE is defined as having a SLEDAI of 6 or greater (with at least 1 non-renal clinical domain) OR Active nephritis defined as having a no or partial response after initial induction and maintenance therapy with mycophenolate mofetil (and other standard of care therapies) for 3 months or more for class III, IV, IV, V (or combination) nephritis.
Active LN is defined as follows: a. kidney biopsy showing Class III, IV, V, III+V, or IV+V, within 1 year from screening, AND b. 24-hour urine protein/creatinine ratio \>=1g/g at screening, AND c. absence of partial renal response (PRR)
Partial renal response (PRR) is defined as a. 24-hour UPCR improved by \>=25% after 3 months from the start of induction standard of care (SOC) therapy (baseline), or \>= 50% after 6 months from the induction therapy (UPCR), AND b. 24-hour UPCR\<2g/g if baseline was \< 3g/g, OR \< 3g/g if baseline at induction was \>= 3g/g. AND d. EGFR\>=60 ml/min/1.73 M2 or no less than 80% of Baseline eGFR (at induction) AND e. No intercurrent rescue therapy, death, or early SOC treatment discontinuation or study withdrawal No response (NR) is defined as a. no achievement of at least a partial renal response, OR b. use of intercurrent rescue therapy, OR c. death 4. Female patients who are women of childbearing potential must agree to use a highly effective form of contraception during the study and for at least 120 days after last exposure to study drug. Male patients with female partners of childbearing potential must use effective barrier contraception (i.e., condoms) during the study and for at least 120 days after last exposure to study drug. Also, patients may not proceed with sperm or egg donation during the study and for at least 120 days after the last exposure to study drug

Exclusion

Aspartate aminotransferase, alanine aminotransferase or alkaline phosphatase level \> 2.5 × upper limit of normal (ULN), or
Total bilirubin \> 1.5 × ULN, or
Hemoglobin \< 5.0 mmol/L \[9 g/dL\], or
White blood cells \< 2.5 × 109/L, or
Absolute neutrophil count \< 1500 /mm3, or
Platelets \< 75 × 109/L 13. Clinically significant chest imaging (e.g. X-ray, computed tomography or magnetic resonance imaging \[MRI\]) abnormalities per Sponsor-Investigator opinion (e.g. interstitial lung disease) or evidence of active TB on chest X-ray. Chest imaging study must have been performed in 3 months prior to the Screening Visit or during the Screening period. 14. Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.

What this trial measures

  • Adverse Events, Study Completion versus WithdrawalFrom Enrollment until 18 weeks

    This pilot phase 1 study will establish the highest safe dose of ONT01 and help generate preliminary evidence in support of future, large-scale, multi-site, controlled Phase 2 efficacy clinical trials with ONT01 in lupus, so that it can be developed as a novel, myeloid directed, oral therapeutic for lupus.