Evaluation of Safety, Tolerability, and Efficacy of Intravenous NVG-2089 and Human Normal Immunoglobulin in Patients with Immune Thrombocytopenia
- Trial ID
- 2024-520359-26-00
- Protocol
- NVG-2089-200
- Sponsor
- Nuvig Therapeutics Inc.
Trial statistics
Diseases & Conditions
Objectives
The primary objective of this study is to evaluate the **safety** and **tolerability** of sequential single ascending doses of NVG-2089 in participants with **Immune Thrombocytopenia (ITP)**. This is clinically relevant as it aims to determine the potential adverse effects and the maximum tolerated dose of NVG-2089, which is crucial for ensuring patient safety and guiding future therapeutic dosing strategies.
Secondary objectives include:
- To evaluate the effects of sequential single ascending doses of NVG-2089 in participants with ITP.
- To evaluate the population pharmacokinetics (PK) of sequential single ascending doses of NVG-2089 in participants with ITP.
Participants
The clinical trial involves a total of **2 participants** diagnosed with **Immune Thrombocytopenia (ITP)**. The study population includes both male and female participants aged between 18 to 80 years. Participants were selected based on their diagnosis of persistent or chronic primary ITP, with a history of response to at least one previous therapy. The trial includes individuals who are asymptomatic or exhibit minor mucocutaneous bleeding, with a platelet count ranging from 20,000 to less than 50,000 cells/mm³. Both genders are represented, and the trial considers lifestyle factors such as the use of contraception for participants of reproductive potential. The study population includes a vulnerable group, ensuring that participants or their legally authorized representatives are capable of providing informed consent.
Plans and Procedures
The clinical trial is designed to evaluate the **safety** and tolerability of sequential single ascending doses of NVG-2089 in participants diagnosed with **immune thrombocytopenia** (ITP). This is a Phase 2, open-label, intra-participant dose escalation study. The trial will involve a series of study visits, beginning with a screening visit to confirm eligibility based on specific inclusion criteria, such as age, diagnosis, and platelet count. Participants will be required to have a history of response to at least one previous therapy for ITP and meet other health and safety criteria. The trial is expected to commence recruitment on September 9, 2025, and conclude by December 9, 2026.
Participants will be involved in the study for a maximum treatment period of 9999 days, with the primary endpoint being the incidence, nature, and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). Secondary endpoints include the percentage of participants achieving a response, duration of response, changes in platelet count, and pharmacokinetic parameters of NVG-2089. The study will be conducted under controlled conditions, with participants receiving the investigational product via intravenous infusion.
The sequence of study visits includes an initial screening visit to assess eligibility, followed by dosing visits where participants will receive NVG-2089. Follow-up visits will be scheduled to monitor safety and efficacy outcomes, with the end-of-study visit marking the conclusion of participant involvement. Participants may be withdrawn from the study early if they experience significant adverse events, fail to comply with study procedures, or withdraw consent. The trial is not categorized as low intervention, and it is essential that participants adhere to the study protocol to ensure the integrity of the data collected.
Treatment
The clinical trial involves the administration of two treatments, one of which is **NVG-2089**, an investigational drug. NVG-2089 is provided as a **solution for infusion** and is administered via **intravenous infusion**. The maximum daily dose is set at 9999 mg/kg, with the same limit for the total dose. The treatment period can extend up to 9999 days. NVG-2089 is a protein-based substance, categorized under "Protein - Other," and is developed by NUVIG THERAPEUTICS INC. The primary objective of the trial is to evaluate the safety and tolerability of sequential single ascending doses of NVG-2089 in participants with **immune thrombocytopenia**.
The second treatment used in the trial is **human normal immunoglobulin (IV)**, which serves as an auxiliary treatment. This medication is also administered as an **intravenous infusion**. The pharmaceutical form is denoted as PHF00230MIG. The maximum daily and total dose for this treatment is 1000 mg/kg, with a treatment period limited to 1 day. Human normal immunoglobulin (IV) is a structurally diverse substance derived from blood. It is classified under the ATC code J06BA02, which corresponds to normal human immunoglobulins for intravascular administration. This treatment has been re-packed and re-labelled for the purposes of the trial.
Efficacy
The efficacy of the investigational product NVG-2089 in the clinical trial will be assessed through several secondary endpoints. These include the percentage of participants achieving a response, defined by specific criteria based on baseline platelet counts. For participants with a dose-specific baseline platelet count of less than 30,000 cells/mm3, a response is defined as either a doubling of the baseline platelet count or achieving a platelet count of at least 50,000 cells/mm3. For those with a baseline platelet count of 30,000 cells/mm3 or more, a response is defined as an increase in platelet count by more than 20,000 cells/mm3 from the baseline or achieving a platelet count of at least 80,000 cells/mm3.
Additional efficacy parameters include the duration of response and both absolute and percentage changes from the dose-specific baseline in platelet count. The dose-specific baseline for response analysis is defined as the pre-dose platelet count on each dosing day. Pharmacokinetic (PK) parameters of NVG-2089 will also be evaluated as part of the efficacy assessment. These endpoints will be measured and analyzed at specified timepoints throughout the trial to determine the efficacy of NVG-2089 in participants with **Immune Thrombocytopenia**.
Inclusion and Exclusion Criteria
Inclusion Criteria
- Male and female participants, age 18 to 80 years at time of screening.
- 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).
- Asymptomatic or with minor mucocutaneous bleeding AND platelet count <50,000 cells/mm3, measured on 2 occasions at least 5 days apart during the screeninig period.
- 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.
- Female participants of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 1.
- 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.
- 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 informed consent form (ICF).
- 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 informed consent form (ICF).
Exclusion Criteria
- Secondary forms of ITP (e.g., ITP secondary to infection, autoimmune diseases, lymphoproliferative diseases and medications)
- 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"
- Transfusion of blood, blood products (including immune globulin), or plasmapheresis within 4 weeks prior to screening.
- 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."
- Receipt of dexamethasone within 4 weeks prior to screening.
- Receipt of rituximab or an anti-CD20 agent within 6 months prior to screening.
- Receipt of an neonatal Fc receptor (FcRn) inhibitor within 12 weeks prior to screening.
- Receipt of IVIg within 4 weeks prior to screening.
- Receipt of anticoagulants within 4 weeks prior to screening
- Receipt of another investigational drug within 4-weeks or 5 half-lives (whichever is longer) prior to screening.
- Concurrent treatment with other monoclonal antibody and/or Fc therapies.
- History of splenectomy.
- Current or past history (within 12 months of screening) of alcohol, drug, or medication abuse. Positive urine drug screen at screening visit unless due to medication prescribed by a treating physician for pre-existing ongoing medical condition.
- 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.
- Poor venous access.
- A known allergy to study drug (NVG-2089) and/or any of its components.
- History of malignancy within 2 years of screeninig, unless the participant received treatment with curative intent that was completed prior to study screening. Participants with fully excised non-melanoma skin cancer or cervical cancer are allowed.
- History of solid organ transplant or hematopoietic stem cell transplantation.
- Planned or anticipated medical or surgical procedure, including dental procedure, during the timeframe of study conduct.
- Clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including active viral infection at screening.
- 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.
- ECG findings of QTcF > 450 msec (males) or > 470 msec (females), poorly controlled atrial fibrillation or other clinically significant abnormalities."
- 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 and/or alanine aminotransferase ≥ 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
- Prior treatment with study drug (NVG-2089)
Trial Status by Country
| Country | Status | Start of Recruitment | Planned Patients |
|---|---|---|---|
 Greece | Recruiting | 09 Sept 2025 | 20 |
 Poland | Recruiting | 09 Sept 2025 | 20 |
 Spain | Recruiting | 09 Sept 2025 | 20 |
Sites & Investigators
Investigational Products
Details about the medicinal products being studied in this clinical trial.
| Product Name | Role in Trial | Formulation | Administration | Max Daily Dose | Treatment Duration | EU MP Number |
|---|---|---|---|---|---|---|
NVG-2089 | Test | SOLUTION FOR INFUSION | INTRAVENIOUS INFUSION | 9999 | 9999 | PRD11625759 |
IMMUNOGLOBULINS, NORMAL HUMAN, FOR INTRAVASCULAR ADM. | Other | PHF00230MIG | INTRAVENIOUS INFUSION | 1000 | 1 | SCP11430138 |