3. Spinal muscular atrophy
179 clinical trials,   102 drugs   (DrugBank: 26 drugs),   52 drug target genes,   78 drug target pathways

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
7 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04042025
(ClinicalTrials.gov)
February 10, 202031/7/2019Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioiA Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III;SMABiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLEnrolling by invitationN/AN/AAll308Phase 4United States;Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;Korea, Republic of
2NCT03837184
(ClinicalTrials.gov)
May 15, 20198/2/2019Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 CopiesPhase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSpinal Muscular Atrophy Type IBiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsPRA Health SciencesActive, not recruitingN/A6 MonthsAll2Phase 3Japan;Korea, Republic of;Taiwan
3NCT03461289
(ClinicalTrials.gov)
August 29, 20184/3/2018Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1European, Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSMABiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.NULLCompletedN/A6 MonthsAll33Phase 3Belgium;France;Italy;United Kingdom;Germany;Netherlands;Spain;Sweden
4NCT03505099
(ClinicalTrials.gov)
April 10, 201813/4/2018Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2Spinal Muscular AtrophyBiological: onasemnogene abeparvovec-xioiNovartis PharmaceuticalsPRA Health SciencesActive, not recruitingN/A42 DaysAll30Phase 3United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom
5NCT03381729
(ClinicalTrials.gov)
December 14, 201713/12/2017Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular AtrophyPhase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular AtrophySpinal Muscular AtrophyBiological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLSuspended6 Months60 MonthsAll51Phase 1United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03306277
(ClinicalTrials.gov)
October 24, 20172/10/2017Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous InfusionSMA - Spinal Muscular Atrophy;Gene TherapyBiological: Onasemnogene Abeparvovec-xioiAveXis, Inc.NULLCompletedN/A180 DaysAll22Phase 3United States
7NCT03421977
(ClinicalTrials.gov)
September 21, 201730/1/2018Long-Term Follow-up Study for Patients From AVXS-101-CL-101A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101Spinal Muscular Atrophy 1Biological: Onasemnogene Abeparvovec-xioiNovartis PharmaceuticalsNULLActive, not recruitingN/AN/AAll13United States