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Lupus Nephritis (LN)Early Phase 1NCT07107659

Safety and Efficacy of ONT01 in Lupus

A Early Phase 1 study testing ONT01 is a small molecule CD11b modulator. in people with Lupus Nephritis (LN) and Lupus Nephritis - WHO Class III, run by Hospital for Special Surgery, New York at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

ONT01 is a drug that is being studied for the treatment of Lupus Nephritis (LN) and Systemic Lupus Erythematosus (SLE) and is not approved by the FDA. The purpose of this study is to better determine whether ONT01 is safe and tolerated by people with lupus nephritis or SLE. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
ONT01 is a small molecule CD11b modulator.
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
61 participants
Where
1 siteUnited States
Study dates
4 Sep 2026to May 2030
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥ 18 years old and able to provide informed consent to participate.
  • Diagnosis of SLE and have fulfilled the ACR classification criteria for SLE during the course of their disease.
  • Active non-renal SLE, with one active non-renal clinical manifestation, who have failed at least 1 disease modifying anti-rheumatic drug (DMARD) therapy (not including hydroxychloroquine and corticosteroids)
  • 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
  • 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

Who cannot join

  • Any condition, including any uncontrolled disease (eg, asthma, interstitial lung disease, pulmonary arterial hypertension, morbid obesity), that in the Sponsor-Investigator's opinion constitutes an inappropriate risk or a contraindication for participation in the study or that could interfere with the study objectives, conduct or evaluation.
  • Active central nervous system SLE associated with significant cognitive impairment leading to inability to provide informed consent and/or comply with the protocol.
  • Comorbidities requiring systemic corticosteroid (CS) therapy, such as asthma or inflammatory bowel disease. Systemic is defined as oral, rectal or any injectable route of administration (thus stable dosing by other routes is allowed, including inhaled, topical, ophthalmic, otic, and intranasal).
  • Active clinically significant viral, bacterial or fungal infection, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 4 weeks of or during the Screening Visit, or completion of oral anti-infectives within 2 weeks before or during the Screening Visit.
  • History of positive human immunodeficiency virus (HIV), hepatitis C antibody and/or polymerase chain reaction, hepatitis B surface antigen (HBsAg) (+), and/or hepatitis B core IgG and/or IgM antibody (+) at the Screening Visit.
  • History, or current diagnosis, of active tuberculosis (TB), or untreated latent TB infection (LTBI), determined by a positive QuantiFERON test at the Screening Visit
Show all 21Show fewer
  • History of malignancy (hematologic or solid tumor) within 10 years prior to Screening Visit, except adequately treated basal cell or squamous cell carcinomas of the skin (no more than 3 lesions requiring treatment in lifetime) or adequately treated carcinoma in situ/cervical intraepithelial neoplasia of the uterine cervix.
  • Immunization with live or live-attenuated vaccines within 1 month before or during the Screening period.
  • Initiation of, or change in, dosing of an angiotensin-converting enzyme inhibitor or angiotensin receptor blocker within 2 weeks before the Screening Visit or during the Screening period.
  • Treatment with Voclosporin or Cyclophosphamide at time of screening.
  • Treatment with other investigational agents within the last 3 months or 5 half-lives, or as per washout requirement from the previous protocol, whichever is longest, prior to the Screening Visit.
  • Clinically significant abnormalities in laboratory tests, unless attributable to active SLE at the Screening Visit
  • 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
  • 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.
  • Pregnant and/or breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of study entry.
  • Patients are unable or unwilling to adhere to the contraception requirements outlined in inclusion criteria 4.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

United States1 site, 1 recruiting

  • Hospital for Special Surgery

    New York, New York, 10021

    Timothy B Niewold, MD, 646 797-8698, felixc@hss.edu

    Christele Felix, 646 714-6196, felixc@hss.edu

    Recruiting

Other trials in United States

About this study

Official title: Safety and Efficacy of ONTO1 in Lupus

ONT01 is a drug that is being studied for the treatment of Lupus Nephritis (LN) and Systemic Lupus Erythematosus (SLE) and is not approved by the FDA. The purpose of this study is to better determine whether ONT01 is safe and tolerated by people with lupus nephritis or SLE. The study also looks at how the administration of ONT01 in combination with widely used treatments given for lupus, including the medication mycophenolate mofetil and others, can improve symptoms of lupus. A total of 61 participants will be enrolled in this study.

Being tested
ONT01 is a small molecule CD11b modulator.
Keywords
Lupus Nephritis, Lupus, SLE
Medical subject headings
Lupus Nephritis, Lupus Erythematosus, Systemic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ONT01 is a small molecule CD11b modulator.Drug

    ONT01 will be administered orally twice daily (in the morning and in the evening at approximately 12 hours apart and approximately the same time each day) and should be taken as whole tablets (not crushed) under fasted conditions (2 hours before and 1 hour after each dose) with 8 oz/240 mL of water.

Study groups in NCT07107659
Study groupRoleWhat they receive
This study will treat patients with Lupus Nephritis (LN) or Systemic Lupus Erythematosus (SLE).ExperimentalLupus Nephritis
Receives: Drug: ONT01 is a small molecule CD11b modulator.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse Events, Study Completion versus Withdrawal

    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.

    Measured: From Enrollment until 18 weeks

Secondary outcomes (1)

  1. Effect on SLE and Lupus Nephritis Biomarkers at Day 28

    Proportion of patients with statistically significant reduction in

    • suPAR (ng/mL)
    • Proteinuria (protein to creatinine ratio (mg/g)
    • C5a (ng/mL)
    • IL-16 (ng/mL)
    • Anti-dsDNA antibodies (IU/mL)
    • Active SLE symptoms (presence or absence of active symptoms as defined by the SLEDAI criteria)

    Lupus Nephritis secondary outcomes: 25 participants will be enrolled. which will allow us to detect a 1.24-fold difference in a biomarker with 80% power, assuming a 30% standard deviation, with an alpha of 0.01.

    Active non-renal lupus secondary outcomes: 36 participants will be enrolled, which will allow us to detect a 1.24-fold difference in a biomarker with 80% power, assuming 30% standard deviation, with an alpha of 0.01.

    Measured: From baseline until Day 28

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Aug 2025
  2. Study start4 Sep 2026 (estimated)
  3. Primary completionMay 2030 (estimated)
  4. Study completionMay 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
61 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07107659
Sponsor's protocol ID
2024-0364
Status last verified
Nov 2025
First submitted
8 Jul 2025
Last updated
20 Nov 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing ONT01 is a small molecule CD11b modulator. in people with Lupus Nephritis (LN) and Lupus Nephritis - WHO Class III, run by Hospital for Special Surgery, New York at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events, Study Completion versus Withdrawal (time frame: From Enrollment until 18 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07107659.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2025.

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