CTIS2022-502844-11-00RecruitingPhase 1
C0371002 - Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer withPF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C=2%) (BeneGene-2) - C0371002
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Pfizer Inc.
- Enrollment
- 56
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 18 to 64 (—)
- Sex
- Male
Inclusion Criteria
- •1 Participants must have completed at least 6 months of prospectively collected data while receiving FIX prophylaxis replacement therapy as per their usual care during the lead-in study (C0371004) prior to providing consent at the screening visit for this study., 2 Participants who have documented moderately severe to severe hemophilia B, defined as FIX:C =2%., 3 Participants who have previous experience with FIX therapy (=50 documented exposure days to a FIX protein product such as recombinant, plasma-derived, or extended half-life FIX product)., 4 Participants must agree to suspend prophylaxis therapy for hemophilia B after administration of the IP. FIX replacement therapy is allowed as needed, 5 Acceptable screening laboratory values as follows: •Hemoglobin =11 g/dL; •Platelets =100,000 cells/µL; •Creatinine =2.0 mg/dL., 6 Sex: Male. Participants are eligible to participate if they agree to the following requirements starting on Day 1/Visit 3 (IP infusion visit) and continue until at least 3 consecutive ejaculate samples test negative for vector shedding: •Refrain from donating sperm. PLUS either: •Be abstinent from heterosexual or homosexual intercourse as their preferred and usual lifestyle (abstinent on a long term and persistent basis) and agree to remain abstinent. OR •Must agree to use contraception/barrier as detailed below: •Agree to use male condom when engaging in any activity that allows for passage of ejaculate to another person, 7 Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent document (ICD) and in this protocol. • For German sites please refer to Germany Appendix, 8 Participants between the ages of 18 (or the minimum country specific age of consent if >18) and =65 years, inclusive, at Visit 1 (Screening Period).
Exclusion Criteria
- •1 Anti-AAVRh74var nAb titer above the established threshold (ie, positive for nAb), performed by a central laboratory during screening. nAb testing may be repeated, if necessary. In such cases, a new sample must be drawn for re-testing., 5 Any participant with conditions associated with increased thromboembolic risk, such as known inherited or acquired thrombophilia, or a history of thrombotic events including but not limited to: stroke, myocardial infarction, and/or venous thromboembolism., 11 Any participant with a planned surgical procedure requiring FIX surgical prophylactic factor treatment in the next 15 months., 10 Currently on antiviral therapy for hepatitis B or C, 12 Participants using therapies that are restricted, 13 Previously dosed in a gene therapy research trial at any time or in an interventional clinical study within the last 12 weeks, excluding participation in Study C0371004, 14 Active hepatitis B or C; HBsAg, HBVDNA positivity, or HCVRNA positivity., 15 Significant liver disease, as defined by preexisting diagnosis of portal hypertension, splenomegaly, or hepatic encephalopathy. Additionally, during screening, a serum albumin level below normal limits and/or significant liver fibrosis by one of the following diagnostic modalities (please note only 1 test is needed for screening purposes): FibroScan median stiffness score >8 kPa units OR Fibro Test/FibroSURE >0.48*. A FibroScan is preferred if available. If there is concern regarding the FibroTest or the FibroScan results due to a confounding medical history (eg, proteinuria can impact FibroTest result), or in the event of conflicting results between the modalities, the investigator should contact the sponsor. *Please note: if a participant has a known history of Gilbert's syndrome, a FibroTest cannot be used for fibrosis testing. However, the participant could be tested using FibroScan., 16 Serological evidence of HIV1 or HIV2 infection with either CD4+ cell count =200 mm3 and/or a viral load >20 copies/mL, and remain on highly active antiretroviral therapy (HAART) throughout the study., 17 Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or participants who are Pfizer employees, including their family members, directly involved in the conduct of the study., 18 Unable to comply with scheduled visits, treatment plan (participants must agree to suspend prophylaxis therapy for hemophilia B after administration of the study intervention), laboratory tests, and other study procedures for up to six years postinfusion of PF-06838435 in the investigator’s judgement., 19 Sensitivity to heparin or heparin induced thrombocytopenia., 9 Current unstable liver or biliary disease per investigator assessment defined by the presence of ascites, hepatic encephalopathy, coagulopathy, hypoalbuminemia, esophageal or gastric varices, persistent jaundice, or cirrhosis. NOTE: Stable chronic liver disease (including Gilbert's syndrome, asymptomatic gallstones, is acceptable if the participant otherwise meets entry criteria)., 7 Alanine transaminase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP) >2 × upper limit of normal (ULN), based on central laboratory results, 20 Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the investigator or the sponsor’s medical monitor, contraindicates partic
Investigators
Similar Trials
Recruiting
Phase 1
Protocol Title: Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Severe Hemophilia A (FVIII:C=1%)hemophilia AMedDRA version: 20.0Level: LLTClassification code: 10060613Term: Hemophilia A (Factor VIII) Class: 10010331CTIS2024-512075-12-00Pfizer Inc.75
Recruiting
Phase 1
Study in Paediatrics with HypEREosinophilic syndrome (SPHERE)Hypereosinophilic syndrome (HES)MedDRA version: 20.0Level: PTClassification code 10048643Term: Hypereosinophilic syndromeSystem Organ Class: 10005329 - Blood and lymphatic system disordersEUCTR2021-000933-15-NLGlaxoSmithKline Research & Development Limited25
Not yet recruiting
Phase 3
A Phase 3, 52-week, open-label, single arm study to investigate the efficacy and safety of mepolizumab SC in participants aged 6 to 17 years with hypereosinophilic syndrome.NL-OMON51313GlaxoSmithKline3
Recruiting
Phase 1
Study in Paediatrics with HypEREosinophilic syndrome (SPHERE)Hypereosinophilic syndrome (HES)MedDRA version: 20.0Level: PTClassification code: 10048643Term: Hypereosinophilic syndrome Class: 100000004851CTIS2023-510110-36-00Glaxosmithkline Research & Development Limited25
Completed
Phase 3
ong-term single-arm open-label phase 3 study, to assess the safety and tolerability of selexipag (ACT-293987) in patients with pulmonary arterial hypertensiohypertension in lung arteries10037454Pulmonary arterial hypertensionNL-OMON41513Actelion Pharmaceuticals24