Safety and Efficacy Study of PF-06835375 in Primary Immune Thrombocytopenia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: PF-06835375.
- Who it may be relevant to
- Registry conditions: Primary Immune Thrombocytopenia. Basic parameters: 18 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, Czechia, Hungary +2
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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 received 1 subcutaneous injection of dose 1 every month for 3 months during the 12-week treatment period. Each participant in cohorts 2, 3, or 4 (will) receive(d) subcutaneous injection(s) of dose 2, 3, or 4 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.
Interventions
- Biological PF-06835375
CXCR5 inhibitor
Primary outcome measures
- Proportion of participants with change from baseline of platelet counts [Time frame: baseline through 12 and 16 weeks]
Secondary outcome measures (6)
- proportion of participants with modified overall response (mOR) [Time frame: baseline through 12 and 16 weeks]
- proportion of participants with complete response (CR) [Time frame: baseline through 12 and 16 weeks]
- Number of Participants With Treatment Emergent Treatment-Related Adverse Events (AEs) [Time frame: baseline through end of study (Week 20 for cohort 1 and Week 24 for cohorts 2 and 3)]
- Proportion of participants with change from baseline of platelet counts [Time frame: baseline to Week 20 and Week 24]
- Proportion of participants with change from baseline of circulating B cells [Time frame: baseline to Week 20 and Week 24]
- Proportion of participants with change from baseline of circulating cTfh cells [Time frame: baseline to Week 20 and Week 24]
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
Australia · 8 centers
- Liverpool Hospital — Liverpool
- South West Radiology — Liverpool
- Slade Pharmacy — Mount Kuring-Gai
- Calvary Mater Newcastle — Waratah
- Flinders Medical Centre — Bedford Park
- ICON Cancer Centre - Kurralta Park — Kurralta Park
- The Alfred Hospital — Melbourne
- Royal Perth Hospital — Perth
Hungary · 6 centers
- Pécsi Tudományegyetem Klinikai Központ — Pécs
- Somogy Megyei Kaposi Mór Oktató Kórház — Kaposvár
- Semmelweis University — Budapest
- Petz Aladár Egyetemi Oktató Kórház — Győr
- Tolna Varmegyei Balassa Janos Korhaz — Szekszárd
- Komárom-Esztergom Vármegyei Szent Borbála Kórház — Tatabánya
Poland · 6 centers
- AIDPORT Sp. z o.o. — Skórzewo
- InterHem — Bialystok
- Klinika Hematologii i Transplantologii Uniwersyteckie Centrum Kliniczne — Gdansk
- Pratia Onkologia Katowice — Katowice
- Uniwersytecki Szpital Kliniczny w Poznaniu — Poznan
- Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wroclawiu — Wroclaw
Czechia · 4 centers
- Fakultni nemocnice Hradec Kralove — Hradec Králové
- Fakultni nemocnice Ostrava — Ostrava - Poruba
- Fakultni nemocnice Kralovske Vinohrady — Prague
- Vseobecna fakultni nemocnice v Praze — Prague
United Kingdom · 4 centers
- Addenbrooke's Hospital — Cambridge
- Derriford Hospital — Plymouth
- The Royal Cornwall Hospital — Truro
- Hammersmith Hospital — London
Canada · 2 centers
- Unity Health Toronto, St. Michael's Hospital — Toronto
- McGill University Health Centre — Montreal
United States · 1 center
- East Carolina University — Greenville
Identifiers
NCT: NCT05070845 · C1131003 · 2023-509338-21-00