Clinical trial NCT05070845: Safety and Efficacy Study of PF-06835375 in Primary Immune Thrombocytopenia
Official title: AN INTERVENTIONAL PHASE 2, OPEN-LABEL, MULTI-CENTER STUDY TO EVALUATE SAFETY AND EFFICACY OF PF-06835375 IN ADULT PARTICIPANTS WITH MODERATE TO SEVERE PRIMARY IMMUNE THROMBOCYTOPENIA
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Primary Immune Thrombocytopenia
Keywords: ITP
platelets
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2021-10-07
Enrollment: 61
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase 2, open-label, multicenter, multiple subcutaneous injection, safety and efficacy study of PF-06835375 in adult participants with primary immune thrombocytopenia (ITP). This study will focus on participants with persistent (\>3 months and ≤12 months), or chronic (\>12 months) ITP

Detailed description: This study is designed to elucidate the effects of PF-06835375 on platelet counts in participants with moderate to severe primary ITP. Based on the experience with other B-cells depleting agents, it is expected that the platelet counts will increase following a standard treatment. Each participant in cohort 1 will receive 1 subcutaneous injection of dose 1 every month for 3 months during the 12-week treatment period. And each participant in cohorts 2 and 3 will receive 1 subcutaneous injection of dose 2 or 3 every month for 4 months during the 16-week treatment period. This should provide sufficient levels of exposure and depletion of CXCR5 positive cells to sustain the effects of PF-06835375 during the treatment period. Additional depletion of Tfh cells may provide sustained increase in platelet count following the last treatment.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of Primary ITP. Ongoing ITP (platelet counts \<50 x 109/L) \[No severe bleeding within 1 month or during screening\] AND Persistent ITP (3 to 12 months) or Chronic ITP \>12 months

Exclusion Criteria:

* Bleeding event according to the WHO grading scale ≥2 occurring ≤4 weeks prior to screen OR a current bleeding event that, in the opinion of the investigator, requires treatment with standard of care therapy OR require blood or blood products during screening
* Splenectomy within 3 months of randomization or planned during the study duration.

Primary outcomes:
measure: Proportion of participants with change from baseline of platelet counts
timeFrame: baseline through 12 and 16 weeks
Secondary outcomes:
measure: proportion of participants with modified overall response (mOR)
timeFrame: baseline through 12 and 16 weeks
measure: proportion of participants with complete response (CR)
timeFrame: baseline through 12 and 16 weeks
measure: Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs)
timeFrame: baseline through end of study (Week 20 for cohort 1 and Week 24 for cohorts 2 and 3)
measure: Proportion of participants with change from baseline of platelet counts
timeFrame: baseline to Week 20 and Week 24
measure: Proportion of participants with change from baseline of circulating B cells
timeFrame: baseline to Week 20 and Week 24
measure: Proportion of participants with change from baseline of circulating cTfh cells
timeFrame: baseline to Week 20 and Week 24
Other outcomes:
Locations:
city: Greenville
country: United States
facility: East Carolina University
state: North Carolina
city: Kogarah
country: Australia
facility: Clinical Research Unit Haematology, Division of Cancer Services, St. George Hospital
state: New South Wales
city: Liverpool
country: Australia
facility: Liverpool Hospital
state: New South Wales
city: Liverpool
country: Australia
facility: South West Radiology
state: New South Wales
city: Mount Kuring-Gai
country: Australia
facility: Slade Pharmacy
state: New South Wales
city: Waratah
country: Australia
facility: Calvary Mater Newcastle
state: New South Wales
city: Bedford Park
country: Australia
facility: Flinders Medical Centre
state: South Australia
city: Kurralta Park
country: Australia
facility: ICON Cancer Centre - Kurralta Park
state: South Australia
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Toronto
country: Canada
facility: Unity Health Toronto, St. Michael's Hospital
state: Ontario
city: Montreal
country: Canada
facility: McGill University Health Centre
state: Quebec
city: Hradec Kralove
country: Czechia
facility: Fakultni nemocnice Hradec Kralove
city: Praha 2
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze
city: Pécs
country: Hungary
facility: Pécsi Tudományegyetem Klinikai Központ
state: Baranya
city: Kaposvár
country: Hungary
facility: Somogy Megyei Kaposi Mór Oktató Kórház
state: Somogy
city: Budapest
country: Hungary
facility: Semmelweis University
city: Györ
country: Hungary
facility: Petz Aladár Egyetemi Oktató Kórház
city: Skórzewo
country: Poland
facility: AIDPORT Sp. z o.o.
state: Wielkopolskie
city: Bialystok
country: Poland
facility: InterHem
city: Gdansk
country: Poland
facility: Klinika Hematologii i Transplantologii Uniwersyteckie Centrum Kliniczne
city: Katowice
country: Poland
facility: Pratia Onkologia Katowice
city: Poznan
country: Poland
facility: Uniwersytecki Szpital Kliniczny w Poznaniu
city: Wroclaw
country: Poland
facility: Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu
city: Plymouth
country: United Kingdom
facility: Derriford Hospital
state: Devon
city: Truro
country: United Kingdom
facility: The Royal Cornwall Hospital
state: England
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: