Immune Thrombocytopenia
Conditions
Keywords
Adult ITP
Brief summary
The goal of this clinical trial is to demonstrate GL-2045 safety and efficacy proof of concept by demonstrating intravenous immunoglobulin (IVig)-like platelet responses in adult patients with Immune Thrombocytopenia (ITP).
Detailed description
This is a Phase 1b, open-label, multiple subcutaneous (SC) injection dose study in participants with ITP. The specific aims of this study are to determine: * The safety of the study drug when given to participants with ITP * The effect of the study drug on safety blood tests and blood platelet counts * How the study drug affects certain responses in the body such as severity of bleeding, levels of fatigue and health-related quality of life * How much of the study drug gets into the bloodstream * The body's immune response to the study drug This information, together with pharmacokinetic (PK) data, will help to establish doses and dosing regimens suitable for use in future studies. The effects of GL-2045 on multiple biomarkers will also be investigated.
Interventions
DRUGGL-2045
Administrative route: SC injection
Sponsors
Gliknik Inc.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Male or female between 18 and 80 years of age with ITP or certain limited ITP conditions. 2. Females must not be pregnant or lactating. Males and females of childbearing potential must agree to use contraception. 3. A platelet count of 10 to 49 × 10\^9/L at baseline. 4. Study subjects must have prior response to corticosteroids or to intravenous immunoglobin (IVIg) documented by a consultant hematologist. Prior response to thrombopoietin receptor agonists is insufficient for eligibility. 5. The subject may be on a stable dose of corticosteroids for the 3 months prior to study entry, limited to a daily dose of prednisone 10 mg or equivalent. No reductions or increases of steroids are allowed during the study. Key
Exclusion criteria
1. Any serious adverse event (SAE) with prior IVIg dosing. 2. Prior splenectomy within 1 year of randomization or planned splenectomy during the study period. 3. Participants with grade 2 bleeding by World Health Organization (WHO) bleeding criteria in the 8 weeks prior to treatment. 4. Abnormal organ function including liver and kidney. 5. Any previous or current treatments prohibited by the protocol. 6. Treatment within the last 4 weeks or intention to treat during the study with a thrombopoietin receptor agonist including romiplostim (Nplate®), eltrombopag (Revolade®), or avatrombopag (Doptelet®).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, severity and type of adverse events and laboratory abnormalities | Screening (Days -28 to -2) to Follow-up (Day 55) |
| Multiple measurements of change from baseline in platelet count | Day -1 to Follow-up (Day 55) |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in Tumor Necrosis Factor-Alpha (TNF-α) Levels | Day 1 to Follow-up (Day 55) |
| Change from Baseline in Classical Complement Pathway (CCP) Inhibition | Day 1 to Follow-up (Day 55) |
| Changes in bleeding scale | Day -1 to Follow-up (Day 55) |
| Changes from baseline in Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue) Questionnaire | Day -1 to Follow-up (Day 55) |
| Changes from baseline in FACIT-Thrombocytopenia 6 Questionnaire | Day -1 to Follow-up (Day 55) |
| Area under the plasma concentration-time curve from time 0 to the time of last quantifiable concentration (AUCO-tlast) | Day 1 to Follow-up (Day 55) |
| Maximum observed concentration (Cmax) | Day 1 to Follow-up (Day 55) |
| Time of the maximum observed concentration (tmax) | Day 1 to Follow-up (Day 55) |
| Incidence of immune response to drug | Day 1 to Day 48 |
Countries
United Kingdom
Contacts
CONTACTGliknik Clinical Trials Group
PRINCIPAL_INVESTIGATORJim Bush, MBChB, PhD
Fortrea Clinical Research Unit Ltd.
Outcome results
None listed