3. 脊髄性筋萎縮症 Spinal muscular atrophy Clinical trials / Disease details
臨床試験数 : 217 / 薬物数 : 149 - (DrugBank : 33) / 標的遺伝子数 : 54 - 標的パスウェイ数 : 80
Showing 1 to 10 of 14 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2017-000266-29-NL (EUCTR) | 25/03/2019 | 05/06/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 ... | 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 Infusion Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients wi ... | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area ... | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | France;Belgium;Spain;Germany;Netherlands;Italy;United Kingdom;Sweden | ||
| 2 | EUCTR2017-004087-35-NL (EUCTR) | 28/02/2019 | 09/07/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-s ... | A 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 SMN2 - SPR1NT A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnose ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada ... | ||
| 3 | EUCTR2017-004087-35-BE (EUCTR) | 31/01/2019 | 16/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-s ... | A 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 SMN2 - SPR1NT A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnose ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada ... | ||
| 4 | EUCTR2017-000266-29-BE (EUCTR) | 31/01/2019 | 23/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 ... | 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 Infusion Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients wi ... | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area ... | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Spain;Belgium;Netherlands;United Kingdom;Italy;Sweden | ||
| 5 | EUCTR2017-004087-35-IT (EUCTR) | 24/12/2018 | 23/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-s ... | A 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 SMN2 - SPR1NT A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnose ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada;Belgium;Australia;Germany;Netherlands;Japan United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;Italy;United Kingdom;Canada ... | ||
| 6 | EUCTR2017-004087-35-ES (EUCTR) | 21/12/2018 | 20/04/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-s ... | A 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 SMN2 - SPR1NT A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnose ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 44 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ... | Australia;Germany;Netherlands;Japan;Belgium;Canada;Italy;United Kingdom;Israel;Korea, Democratic People's Republic of;Spain;Taiwan;United States Australia;Germany;Netherlands;Japan;Belgium;Canada;Italy;United Kingdom;Israel;Korea, Democratic Peo ... | ||
| 7 | EUCTR2017-000266-29-ES (EUCTR) | 18/12/2018 | 28/05/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 ... | 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 Infusion Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients wi ... | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area ... | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 8 | EUCTR2017-000266-29-DE (EUCTR) | 05/11/2018 | 05/03/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 ... | 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 Infusion Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients wi ... | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 MedDRA version: ... | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 30 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom;Sweden | ||
| 9 | EUCTR2017-004087-35-GB (EUCTR) | 24/08/2018 | 08/05/2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-s ... | A 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 SMN2 - SPR1NT A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnose ... | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | Novartis Gene Therapies, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 29 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada ... | ||
| 10 | EUCTR2017-000266-29-GB (EUCTR) | 12/06/2018 | 12/02/2018 | Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 ... | 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 Infusion Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients wi ... | Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1;Therapeutic area ... | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: onasemnogene abeparvovec Product Name: AVXS-101(previously known as scAAV9.CB.SMN) Product Code: AVXS-101 INN or Proposed INN: ... | AveXis, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 33 | Phase 3 | France;Spain;Belgium;Netherlands;Italy;United Kingdom;Sweden |