ISTH - Resous Rekòmande

Yon konpilasyon nan Terapi jèn nan papye syantifik nan emofil, chwazi esè klinik, dokiman regilasyon ak lyen itil

WFH eLearning Center esè klinik

https://elearning.wfh.org/elearning-centres/clinical-trials/

Sant eLearning sa a pibliye pa World Federation of Hemophilia
(WFH) epi yo te lye isit la ak pèmisyon.

© 2022 Federasyon Mondyal Emofili https://elearning.wfh.org/elearning/centres/clinical-trials

Papye syantifik ak dokiman regilasyon

Revizyon Jenoterapi pou Hemofili

1. Abbasi J. Terapi jèn pou emofil montre pwomès. JAMA. 2018; 319 (6): 539. https://www.ncbi.nlm.nih.gov/pubmed/29450507

2. Arruda VR, et al. Nouvo apwòch pou terapi emofili: siksè ak defi. San. 2017; 130: 2251-2256. https://www.ncbi.nlm.nih.gov/pubmed/29018078

3. balansansh P, et al. Nouvo terapi ak aktyèl pwogrè klinik nan hemofili A. Ther Adv Ematol.2018; 9: 49-61. https://www.ncbi.nlm.nih.gov/pubmed/?term=29387330

4. Beck DL. Avans nan terapi jèn pou emofili. Ash Klinik Nouvèl. Oktòb NAN, NAN. https://www.ashclinicalnews.org/features/breakthroughs-gene-therapy-hemophilia/

5. Colella P, et al. Emerging pwoblèm nan AAV-medyatè nan terapi jèn vivo. Mol Ther Meth Clin Dev. 2018; 8: 87-104. https://www.ncbi.nlm.nih.gov/pubmed/29326962

6. DiMichele DM. Navige vit monte desann sou gran wout inovasyon nan terapi emofil. HemaSphere. KOULYE: KOUMAN POU: SEKSYON. https://www.ncbi.nlm.nih.gov/pubmed/30887008

7. Doshi BS, et al. Terapi jèn pou emofili: kisa lavni an kenbe? Ther Adv Ematol. 2018; 9: 273-293. https://www.ncbi.nlm.nih.gov/pubmed/30210756

8. Dunbar CE, et al. Terapi jèn vini nan laj. Syans. 2018; 359: 4672. https://www.ncbi.nlm.nih.gov/pubmed/29326244

9. Evens H, et al. Terapi jèn emofilik: Soti nan pyèj anbale pou gamechanger. Emofili. 2018;24(Suppl. 6):50–59. https://www.ncbi.nlm.nih.gov/pubmed/29878653

10. George LA. Terapi jèn nan emofil vini ki gen laj. Pwogram Edikasyon Hematoloji Am Soc Hematol. 2017; 587-594. https://www.ncbi.nlm.nih.gov/pubmed/29222308

11. Mahlangu J, et al. Terapi kap fè emosyonèl pou pèspektiv global. Emofili. 2018;24(Suppl. 6):15–21. https://www.ncbi.nlm.nih.gov/pubmed/29878661

12. Makris M. Yon laj an lò pou tretman emofili? Emofili. 2018; 24: 175-176. https://www.ncbi.nlm.nih.gov/pubmed/29601683

13. Park CY, et al. Genomic-koreksyon teknoloji pou jèn koreksyon nan emofili. Imèn Jen. 2016; 135: 977-981. https://www.ncbi.nlm.nih.gov/pubmed/27357631

14. Perrin GQ, et al. Mizajou sou terapi jèn pou emofili. San. 2019; 133: 407-414. https://www.ncbi.nlm.nih.gov/pubmed/30559260

15. Pierce GF, et al. Yon multitude nan terapi anba etid pou emofili. Mol Ther. 2017; 25: 2429-2430. https://www.ncbi.nlm.nih.gov/pubmed/29055619

16. Pierce GF, et al. Pase, prezan ak tan kap vini nan terapi jèn emofilik: Soti nan vektè ak transjèn rive rezilta li te ye ak enkoni. Emofili. 2018;24(Suppl. 6):60–67. https://www.ncbi.nlm.nih.gov/pubmed/29878660

17. Pierce GF, et al. Premye WFH jèn terapi tab wonn tab la: Konprann peyizaj la ak defi nan terapi jèn pou emofili atravè mond lan. Emofili. 2019; 1–6. https://www.ncbi.nlm.nih.gov/pubmed/30604914

18. Tiyo SW. Nouvo terapi pou emofili. Pwogram Edikasyon Hematoloji Am Soc Hematol. 2016; (1): 650-656. https://www.ncbi.nlm.nih.gov/pubmed/27913542

19. Tiyo SW. Terapi jèn pou emofili. Pedyat Kansè nan san. KOULYE: KOUMAN POU: SEKSYON. https://www.ncbi.nlm.nih.gov/pubmed/29077262

20. Schutgens R, et al. Terapi jèn nan emofili: Soti nan battage a nan espwa. HemaSphere. 2018;2(2):22:e37. https://journals.lww.com/hemasphere/Fulltext/2018/04000

Prezantasyon ki sot pase yo

EAHAD Gene Therapy Hub ak Spoke Model Webinar
30 mas 2022
https://www.youtube.com

Lyen entent itil

Fondasyon Nasyonal pou Hemofili
https://www.hemophilia.org/

Mondyal Federasyon nan Emofili
https://www.wfh.org

Pwogrè nan klinik nan Terapi Gene pou Emofili
https://www.medscape.org/sites/advances/gene-therapy-hemophilia

Seleksyone esè klinik yo

Hemofilya A

  • NCT02576795. Yon Faz 1/2, Dòz-Eskalasyon, Sekirite Sosyal, Tolerans ak Efikasite Etid sou Rokarovovec Valoctocogene, yon viris ki asosye ak Adenovirus-vektè ki gen medyatè Transfè jeni nan VIII Imèn Faktè nan pasyan ki gen imès grav A. https://clinicaltrials.gov/ct2/show/NCT02576795
  • NCT03520712. Yon Faz 1/2 Sekirite Sosyal, Tolerans, ak efikasite etid sou Rokarovovec Valoctocogene, yon viris ki asosye-Associated vektè-médiésye Gene Transfere nan VIII Imèn Faktè nan pasyan Hemophilia A ki gen rezidyèl FVIII Nivo ≤ 1 IU / dL ak antikò pre-egziste ki kont AAV5 . https://clinicaltrials.gov/ct2/show/NCT03520712
  • NCT03392974. Faz 3 Etid Evalye Efficacité / Sekirite nan Valoctocogene Roxaparvovec yon AAV vektè-medyatè Gene Transfè nan hFVIII nan yon Dòz nan 4E13vg / kg nan Hemophilia A Pasyan ki gen rezidyèl FVIII Nivo ≤1IU / dL ki resevwa profiletik FVIII perfusion. https://clinicaltrials.gov/ct2/show/NCT03392974
  • NCT03370913. Yon Faz 3 Open-Label, Single-bra etidye Evalye efikasite a ak Sekirite nan BMN 270, yon viris ki asosye-Associated vektè ki gen medyatè transfè jeni nan VIII Imèn Faktè nan pasyan Hemophilia A ki gen rezidyèl FVIII Nivo ≤ 1 IU / dL ki resevwa pwofilaktik Perfusion FVIII. https://clinicaltrials.gov/ct2/show/NCT03370913
  • NCT03432520. Yon Evalyasyon milti-sant nan alontèm Sekirite Sosyal la ak efikasite nan SPK-8011 [Adeno-Associated vektè viral Avèk B-Domèn Efase moun jèn VIII Faktè] nan Gason ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03432520
  • NCT03003533. Transfè jèn, Dòz-Jwenn Sekirite, Tolerans, ak efikasite Etid la SPK-8011 [yon recombinant Adeno-Associated vektè viral Avèk Imen faktè Imen VIII] nan moun ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03003533
  • NCT03734588. Etid Dòz-pou jwenn nan Terapi jèm SPK-8016 nan pasyan ki gen emofil A pou sipòte evalyasyon nan moun ki gen inhibiteur FVIII. https://clinicaltrials.gov/ct2/show/NCT03734588
  • NCT03370172. Yon Global, Open-Label, milti-sant, Faz 1/2 etid sou Sekirite Sosyal la ak Dòz la nan BAX 888, yon Adeno-Associated Serotip viris 8 (AAV8) vektè eksprime B-Domèn Efase VIII efase (BDD-FVIII) nan emofil grav. Yon sijè administre yon sèl perfusion vennsèn. https://clinicaltrials.gov/ct2/show/NCT03370172
  • NCT03061201. Yon Faz 1/2, Louvri-etikèt, Adaptatif, etid Dòz-K ap evalye Sekirite Sosyal la ak Tolerabilite nan SB-525 (Recombinant AAV2 / 6 Imèn Faktè Terapi jèn 8 moun) nan granmoun pou Timoun ki gen emofil grav .. https://clinicaltrials.gov/ct2/show/NCT03061201
  • NCT03001830. GO-8: Terapi jèn pou emofil A lè l sèvi avèk yon serotip 8 Kadsid pseudotyped Adeno ki asosye ak vektè Vajen kodaj Faktè VIII-V3. https://clinicaltrials.gov/ct2/show/NCT03001830
  • NCT03217032. Lentiviral FVIII Terapi jèn pou emofili A. https://clinicaltrials.gov/ct2/show/NCT03217032
  • NCT03818763. Faz I etidye Evalye Sekirite ak Posibilite Transfè Gene Twopilatèn ematopyotik ki sible akouchman faktè VIII nan platelèt pou pasyan ki gen emofil A. https://clinicaltrials.gov/ct2/show/NCT03818763

Hemofil B

  • NCT02484092. Terapi jèn, Open-Label, etid Dòz-Eskalasyon nan SPK-9001 [Adeno-Associated vektè viral Avèk Imèn moun faktè IX] Nan sijè ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT02484092
  • NCT03307980. Ranpli Etid Long: Yon Faktè IX (ranje) Transfè Gene, milti-sant Evalyasyon nan tèm long Sekirite Sosyal ak efikasite nan SPK9001 https://clinicaltrials.gov/ct2/show/NCT03307980
  • NCT02396342. Yon Faz I / II, Open-mete etikèt sou, san kontwòl, Single-dòz, Dòz-moute, Multi-sant Jijman Mennen ankèt sou yon Adeno-ki asosye vektè viral ki gen yon Codon-optimize Imèn moun faktè IX (AAV5-hFIX) administre nan pasyan granmoun ki gen Emofili B. ki Grav oswa ki Modere. https://clinicaltrials.gov/ct2/show/NCT02396342
  • NCT03569891. Faz III, Open-mete etikèt sou, Single-dòz, milti-sant, multinasyonal Jijman Mennen ankèt sou yon serotip 5 vektè Adeno-ki asosye viral ki gen Variant a Padua nan yon Codon-optimize Imèn moun faktè IX-(AAV5-hFIXco-Padova, AMT-061) Administre soufè adilt ki gen emofil B. grav oswa modere grav. https://clinicaltrials.gov/ct2/show/NCT03569891
  • NCT03489291. Faz IIb, Open-mete etikèt sou, Single-dòz, Single-bra, milti-sant Jijman Konfime Faktè IX la Aktivite Nivo nan serotip 5 Adeno ki asosye vektè viral ki gen Variant la Padua nan yon Codon-optimize Imèn Faktè Imèn Codon (AAV5 -hFIXco-Padova, AMT-061) administre soufè adilt ki gen emofil gra oswa modere grav B. https://clinicaltrials.gov/ct2/show/NCT03489291
  • NCT01687608. Yon Faz 1/2 Open-Label, Single Single Dòz Jij la nan yon oto-konplemante optimize Adeno ki asosye viris Serotip 8 Faktè IX Terapi jèn (AskBio009) nan granmoun ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT01687608
  • NCT03369444. Yon Faz I / II, Louvri etikèt, Multicentre, Dòz sèl ki monte, Etid sou sekirite nan yon Novè Adeno-Associated vektè viral (FLT180a) nan pasyan ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT03369444
  • NCT03641703. Yon etid Open-etikèt, multikentrik, alontèm pou envestige sou sekirite ak durabilite nan repons apre Dosaj nan yon Novè Adeno ki asosye-vektè viral (FLT180a) nan pasyan ki gen emofil B. https://clinicaltrials.gov/ct2/show/NCT03641703
  • NCT02695160. Yon Etid Faz I, Open-Etikèt, Dòz Moute Pou Evalye Sekirite ak Tolerabilite AAV2 / 6 Faktè IX Terapi Jen Genyen Atravè Zin Nuklea Dwèt (ZFN) Entèmedyè ki se Medyatè Sible SB-FIX nan sijè pou granmoun ki gen emofil grav B. https://clinicaltrials.gov/ct2/show/NCT02695160
  • NCT00979238. Yon etid ouvri sou etikèt dòz Dòz yon vektè Auto-konplemantè ki asosye ak Adeno (scAAV 2/8-LP1-hFIXco) pou transfè jèn nan emofil B. https://clinicaltrials.gov/ct2/show/NCT00979238
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