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- busulfan / Generic mfg., OTL-200 / Orchard Therap
[VIRTUAL] LENTIVIRAL HEMATOPOIETIC STEM AND PROGENITOR CELL GENE THERAPY (HSPC-GT) FOR METACHROMATIC LEUKODYSTROPHY (MLD): CLINICAL OUTCOMES FROM 33 PATIENTS (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4988;
P1/2, P2
Clinical Trial Registry: Trial 1, OTL-200-f: Gene Therapy for Metachromatic Leukodystrophy (MLD). NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987
- busulfan / Generic mfg., OTL-200 / Orchard Therap
[VIRTUAL] LENTIVIRAL HEMATOPOIETIC STEM AND PROGENITOR CELL GENE THERAPY (HSPC-GT) FOR METACHROMATIC LEUKODYSTROPHY (MLD): CLINICAL OUTCOMES FROM 33 PATIENTS (ePoster Area) - Jul 3, 2020 - Abstract #EBMT2020EBMT_4987;
P1/2, P2
Clinical Trial Registry: Trial 1, OTL-200-f: Gene Therapy for Metachromatic Leukodystrophy (MLD). NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Enrollment closed, Viral vector: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - May 3, 2020
P2, N=10, Active, not recruiting, Sponsor: Orchard Therapeutics
NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987 Recruiting --> Active, not recruiting
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Trial initiation date: OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - May 3, 2020
P3, N=6, Recruiting, Sponsor: Orchard Therapeutics
Recruiting --> Active, not recruiting Initiation date: Mar 2020 --> Jun 2020
- OTL-200 / Orchard Therap
[VIRTUAL] Effects of lentiviral (LV) hematopoietic stem and progenitor cell gene therapy (HSPC-GT) on central and peripheral demyelination and brain atrophy in late- infantile metachromatic leukodystrophy (LI-MLD) (Room Copenhagen) - Apr 9, 2020 - Abstract #EAN2020EAN_2452;
Here, we present OTL-200 treatment effects on central and peripheral demyelination and brain atrophy in these 16 LI-MLD patients using brain MRI and nerve conduction velocity (NCV)... Collectively, these results suggest that OTL- 200 may prevent, stabilize, or delay the hallmark progressive CNS and PNS damage of LI-MLD, consistent with treatment effects observed on motor function and cognition.
- | Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
New P3 trial: OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Feb 25, 2020
P3, N=6, Recruiting, Sponsor: Orchard Therapeutics
- busulfan / Generic mfg., OTL-200 / Orchard Therap
[VIRTUAL] LENTIVIRAL HEMATOPOIETIC STEM AND PROGENITOR CELL GENE THERAPY (HSPC-GT) FOR METACHROMATIC LEUKODYSTROPHY (MLD): CLINICAL OUTCOMES FROM 33 PATIENTS (ePoster Area) - Feb 8, 2020 - Abstract #EBMT2020EBMT_1998;
P1/2, P2
Clinical Trial Registry: Trial 1, OTL-200-f: Gene Therapy for Metachromatic Leukodystrophy (MLD). NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987
- | OTL-200 / Orchard Therap
$ORTX Announces the Filing and Validation of MAA by EMA for OTL-200 for the Treatment of Metachromatic Leukodystrophy https://t.co/wCONN4s36l (Twitter) - Dec 2, 2019
- ||| Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Phase classification, Enrollment change, Trial primary completion date, Gene therapy: TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Dec 20, 2018
P1/2, N=20, Active, not recruiting, Sponsor: Orchard Therapeutics Limited
NCT01560182, https://clinicaltrials.gov/ct2/show/NCT01560182; EudraCT # 2009-017349-77 Trial 2: OTL-200-c: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD); NCT03392987, https://clinicaltrials.gov/ct2/show/NCT03392987 Phase classification: P2 --> P1/2 | N=14 --> 20 | Trial primary completion date: Apr 2023 --> Apr 2018
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Phase classification, Viral vector: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Dec 19, 2018
P2, N=10, Recruiting, Sponsor: Orchard Therapeutics Limited
Phase classification: P2 --> P1/2 | N=14 --> 20 | Trial primary completion date: Apr 2023 --> Apr 2018 Phase classification: P3 --> P2
- | Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
New P3 trial, Viral vector: A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Jan 8, 2018
P3, N=10, Recruiting, Sponsor: GlaxoSmithKline
- | Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Phase classification, Enrollment change, Trial primary completion date, Gene therapy: TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Mar 24, 2017
P2, N=14, Active, not recruiting, Sponsor: GlaxoSmithKline
Phase classification: P3 --> P2 Phase classification: P1/2 --> P2 | N=20 --> 14 | Trial primary completion date: Apr 2018 --> Apr 2023
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
New P1/2 trial, Gene therapy: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy (clinicaltrials.gov) - Sep 24, 2015
P1/2, N=50, Recruiting, Sponsor: Shenzhen Second People's Hospital
- | Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Enrollment closed, Trial primary completion date, Gene therapy: TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Apr 21, 2015
P1/2, N=20, Active, not recruiting, Sponsor: IRCCS San Raffaele
Phase classification: P1/2 --> P2 | N=20 --> 14 | Trial primary completion date: Apr 2018 --> Apr 2023 Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Oct 2017 --> Apr 2018
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
Enrollment status, Trial primary completion date, Gene therapy: TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Dec 9, 2014
P1/2, N=20, Enrolling by invitation, Sponsor: IRCCS San Raffaele
Enrolling by invitation --> Active, not recruiting | Trial primary completion date: Oct 2017 --> Apr 2018 Recruiting --> Enrolling by invitation | Trial primary completion date: Dec 2015 --> Oct 2017
- Lenmeldy (atidarsagene autotemcel) / Kyowa Kirin
New P1/2 trial, Gene therapy: TIGET-MLD: Gene Therapy for Metachromatic Leukodystrophy (MLD) (clinicaltrials.gov) - Mar 15, 2012
P1/2, N=8, Recruiting, Sponsor: IRCCS San Raffaele