An Exploratory Study of Golidocitinib in Adult Patients With ITP

Peking University

Status and phase: Not yet enrolling; Phase 2; Phase 1

Conditions: Immune Thrombocytopenia (ITP)

Treatments: Drug: Placebo; Drug: Golidocitinib

Study type: Interventional

Funder types: Other

Identifiers

NCT07196163

DZ2024J0003

Details and patient eligibility

About

This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.

Enrollment: 138 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and female participants between 18 and 80 years old.
• Primary ITP for >3 months.
• An average of two platelet counts (⩾ 7 days apart) of < 30 × 10⁹/L.
• Have relapsed or treatment-resistant to previous ITP therapies, including corticosteroids and at least one other ITP therapy.
• Have history of response to previous treatments.
• Adequate hematologic, hepatic, and renal fuction.
• Participants willing to comply with contraceptive restrictions.

Exclusion criteria

• Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia.
• Previous or current therapy and comedications meet exclusion criteria.
• Patients with major caridiovascular disease, active infetion, maligancy or uncontrolled systemic disease.
• Women who are breast feeding.
• History of hypersensitivity to sudy drug with a similar chemical structure or class.
• Previously received JAK inhibitors.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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138 participants in 3 patient groups, including a placebo group

Golidocitinib Part B

Experimental group

Description:

Golidocitinib at recommended phase 2 doses, administered orally, once daily.

Treatment:

Drug: Golidocitinib

Placebo Part B

Placebo Comparator group

Description:

Placebo at recommended phase 2 doses, administered orally, once daily.

Treatment:

Drug: Placebo

Golidocitinib Part A

Experimental group

Description:

Golidocitinib dose escalation, administered orally, once daily.

Treatment:

Drug: Golidocitinib