EUCTR2021-000608-39-NO进行中（未招募）1 期
An open-label, multi-center, first in human, phase 1/2a trial to evaluate the safety and preliminary efficacy of autologous tolerogenic dendritic cells ex vivo loaded with recombinant Factor VIII (FVIII) in adults with congenital Hemophilia A (HA) with neutralizing antibodies to FVIII and having failed Immune Tolerance Induction (ITI)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- Idogen AB
- 入组人数
- 9
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Men, 18-60 years of age, diagnosed with congenital severe Hemophilia A (defined as <1% of normal activity of FVIII).
- •2. Positive history of neutralizing anti-FVIII antibodies with a historic peak titer of BU>5, BU>1 at inclusion, confirmed loss of effect of FVIII and by the investigator judged not being able to be treated with FVIII to stop a bleed.
- •3.Confirmed failure with conventional immune tolerance induction (ITI) protocols (i.e. regular, longtime, daily/several times weekly administration of FVIII at high or low dose with or without concomitant immunosuppression) such as the Malmö protocol (Freiburghaus 1999), the high-dose Bonn-protocol (Brackmann 1996) or the low-dose Dutch protocol (Mauser-Bunschoten 1995, Ter Avest 2010).
- •a) Definition of ITI failure: Inhibitor titer decline less than 20% over any 6-month period after the first 3 months of ITI or failure to achieve success or partial response after 33 months of ITI (DiMichele 2007). The subject should have failed at least 1 ITI.
- •4. By the investigator been judged not eligible for, or not possible to treat, with conventional immune tolerance induction (ITI) protocols (i.e. not available at site; patient fulfilling established bad risk criteria” for ITI (Osooli & Berntorp, 2015) and thus assessed as not suitable for ITI; other non-immunological contraindications to ITI such as problematic venous access and/or not willing to undergo the burden of frequent injections)
- •5. Peripheral venous access suitable for leukapheresis.
- •6. Appropriate contraceptive measures for preventing pregnancy to occur during the trial in the subjects’ spouses (as per Clinical Trial facilitation Group [CTFG] guidance, 2014.) (Clinical Trial Facilitation Group (CTFG) 2014).
- •7. Written informed consent is obtained. The subject signing the research consent form is, in the investigator's judgement, deemed to have adequate decision-making capacity to do so.
- •8. Investigator’s judgement that the subject has the physical and mental capacity to participate and complete the trial.
- •9. Vaccinated against Covid-19.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Treatment with Feiba throughout the trial. (Since Feiba contains small amount of FVIII, it will challenge the subject and interfere with the design of the trial, which includes a FVIII challenge with 50 IU/Kg Kovaltry, octocog alfa at Week 20.) However, if a serious bleed not responding to treatment with NovoSeven occurs, Feiba may be considered.
- •2. Treatment with other experimental treatment within 6 months prior to trial start.
- •3. Active infection requiring antibiotics or anti-viral treatment at screening.
- •4. Laboratory test results that deviate more than ±3 standard deviations (SD) from the local site laboratory's normal reference range at screening
- •5. History of clinically significant allergy (anaphylactic shock or anaphylactoid symptoms).
- •6. Previous or concomitant autoimmunity.
- •7. Previous or concomitant malignancy.
- •8. All immunocompromised subjects as determined by the investigator including subjects on systemic immunosuppressant drugs within 12 weeks of enrolment, or planned use during the trial period.
- •9. Any other disease, metabolic dysfunction, physical examination or laboratory finding giving reasonable suspicion that it may impact the safety or efficacy evaluation or render the subject at risk. The absence of such concomitant disease is ensured by a thorough history and physical examination prior to inclusion. If necessary, relevant medical records are requested.
- •10. Systemic inflammation.
- •11. Planned surgery including tooth extraction during the trial period.
- •12. Vaccination within 4 weeks prior to enrollment or planning to be vaccinated during the trial period.
- •13. Contraindications for undergoing leukapheresis:
- •Abnormal vital parameters, abnormal blood pressure or pulse
- •Infectious laboratory parameters (human immunodeficiency virus [HIV] and syphilis). Regarding hepatitis, patients with antibodies against HBs or HBc can be accepted if they are HBsAg negative, and patients with antibodies against HCV (Hepatitis C-Virus) can be accepted if they are HCV RNA negative. Regarding HIV, patients treated with anti-viral treatment with a PCR level of <50 HIV-1 RNA copies/mL and CD34+ >200 cells/µL (validated cell count method) can be accepted.
- •Angiotensin-Converting Enzyme (ACE) inhibitor
研究者
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