Safety, Tolerability, and Efficacy of NVG-2089 in Participants with Immune Thrombocytopenia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Nuvig Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

20 Aug 2025 → ongoing

Decision date (initial)

2025-08-14

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Nuvig Therapeutics, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Efficacy, Pharmacokinetic, Safety

To evaluate the safety and tolerability of sequential single ascending doses of NVG-2089 in participants with ITP

Secondary objectives 2

1. 1. To evaluate the effects of sequential single ascending doses of NVG-2089 in participants with ITP
2. 2. To evaluate the population PK of sequential single ascending doses of NVG-2089 in participants with ITP

Conditions and MedDRA coding

Immune Thrombocytopenia (ITP)

Study design 3 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Male and female participants, age 18 to 80 years at time of screening.
2. 2. Diagnosis of persistent (>3 months and ≤12 months), or chronic (>12 months) primary ITP. If the participant has received prior treatment for ITP, they must have a history of response to at least one previous therapy (defined as increase in platelet count to ≥50,000 cells/mm3 with an increase of ≥ 20,000 cells/mm3 relative to platelet count prior to treatment).
3. 3. Asymptomatic or with minor mucocutaneous bleeding AND platelet count of ≥20,000 to <50,000 cells/mm3, measured on 2 occasions. At least one measurement should be obtained during screening. Documented historical platelet count obtained within 4 weeks prior to screening will also be acceptable for one of the two readings.
4. 5. If participant has received prior IVIg therapy participant must have shown a sufficient platelet response (doubling of platelet count within 7 days of IVIg infusion) and must not have lost response to IVIg therapy while on treatment.
5. 6. Female participants of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 1.
6. 7. Female participants who are sexually active with a male partner of reproductive potential must use double contraception (including a barrier contraceptive and another method) from at least 28 days prior to Screening and for 90 days after last dose of study drug; female participants must also refrain from oocyte donation for the purpose of reproduction during this period. Exceptions are made for surgically sterile participants, or post-menopausal females (defined as 12 months of spontaneous amenorrhea or 6 months of spontaneous amenorrhea with serum follicle-stimulating hormone levels >40 mIU/mL or 6 weeks postsurgical bilateral oophorectomy with or without hysterectomy). Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the participant.
7. 8. Male participants with female partners who are of reproductive potential must agree to the use of highly effective, barrier contraception for the duration of the study, and for 90 days after the last dose of study drug.
8. 9. Participant is capable or has a legally authorized representative(s) (LAR[s]) capable of providing a signed informed consent which includes compliance with the requirements and restrictions listed in the ICF.

Exclusion criteria 23

1. 1. Secondary forms of ITP (e.g., ITP secondary to infection, autoimmune diseases, lymphoproliferative diseases and medications)
2. 2. History of splenectomy.
3. 3. History of malignancy, unless the participant received treatment with curative intent. Participants with fully excised non-melanoma skin cancer or cervical cancer are allowed.
4. 4. History of solid organ transplant or hematopoietic stem cell transplantation.
5. 5. Planned or anticipated medical or surgical procedure, including dental procedure, during the timeframe of study conduct.
6. 6. Clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including active viral infection at screening.
7. 7. Any medical condition that, in the opinion of the investigator, would interfere with study evaluations or procedures, and/or put the participant at increased risk.
8. 8. ECG findings of QTcF > 450 msec (males) or > 470 msec (females), poorly controlled atrial fibrillation or other clinically significant abnormalities."
9. 9. Other significant organ dysfunction, including but not limited to, hematologic, renal, or hepatic dysfunction, as evidenced by: a. Absolute neutrophil count ≤ 1.5 x 10^9/L b. Hemoglobin (Hgb) < 9 g/dL c. Aspartate aminotransaminase (AST) and/or alanine aminotransferase (ALT) ≥ 2 x the upper limit of normal (ULN), d. Albumin ≤ 3 g/dL e. Total bilirubin ≥ 1.5 x ULN f. Estimated glomerular filtration rate < 50 mL/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) method"
10. 10. Any of the following at screening: a. Active Hepatitis B Virus (HBV): Hepatitis surface antigen (HBsAg) positive b. Active Hepatitis C Virus (HCV): serology positive for HCV-antibody c. Human Immunodeficiency Virus (HIV) positive serology"
11. 11. Transfusion of blood, blood products (including immune globulin), or plasmapheresis within 4 weeks prior to screening.
12. 12. Change in current ITP therapy (e.g., prednisone, methylprednisone, mycophenolate, dapsone, danazol, azathioprine, or TPO receptor agonist) or dose within 4 weeks prior to screening."
13. 13. Receipt of dexamethasone within 4 weeks prior to screening.
14. 14. Receipt of rituximab or an anti-CD20 agent within 6 months prior to screening.
15. 15. Receipt of an neonatal Fc receptor (FcRn) inhibitor within 12 weeks prior to screening.
16. 16. Receipt of IVIg within 4 weeks prior to screening.
17. 17. Receipt of anticoagulants within 4 weeks prior to screening
18. 18. Receipt of another investigational drug within 4-weeks or 5 half-lives (whichever is longer) prior to screening.
19. 19. Concurrent treatment with other monoclonal antibody and/or Fc therapies.
20. 20. Current or past history (within 12 months of screening) of alcohol, drug, or medication abuse. Positive urine drug screen at screening visit.
21. 21. Pregnant or lactating women and those intending to become pregnant during the study or are unwilling to apply an effective birth control method (such as implants, injectables, combined oral contraceptives, intrauterine devices [IUDs], sexual abstinence, or vasectomized partner) up to 90 days after last study drug administration.
22. 22. Poor venous access.
23. 23. A known allergy to study drug and/or any of its components.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence, nature, and severity of TEAEs, and serious adverse events (SAEs)

Secondary endpoints 4

1. 1. Percent of participants achieving a response. − For participants with a dose-specific baseline platelet count <30,000 cells/mm3, a response is defined as a doubling of the dose-specific baseline platelet count or a platelet count of ≥50,000 cells/mm3; − For participants with a dose-specific baseline platelet count ≥30,000 cells/mm3, a response is defined as an increase in the platelet count to >20,000 cells/ mm3 from dose-specific baseline or a platelet count of ≥80,000 cells/mm3
2. 2. Duration of response
3. 3. Absolute and percent change from the dosespecific baseline in platelet count Note: Dose-specific baseline for response analysis is defined as predose platelet count on each dosing day.
4. 4. PK parameters of NVG-2089

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Nuvig Therapeutics Inc.

Sponsor organisation

Nuvig Therapeutics Inc.

Address

4200 Bohannon Drive Suite 250

City

Menlo Park

Postcode

94025-1021

Country

United States

Scientific contact point

Organisation

Nuvig Therapeutics Inc.

Contact name

Alan Glicklich

Public contact point

Organisation

Nuvig Therapeutics Inc.

Contact name

Yvonne Coffey

Third parties 6

Locations

3 EU/EEA countries · 11 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 24 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications