DDrare: Database of Drug Development for Rare Diseases

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.

Drug = 2011-2012 seasonal flu vaccine Intramuscular; 2011-2012 seasonal flu vaccine Subcutaneous; 4-Aminopyridine; 4-aminopyridine; 4-cholesten-3-one, oxime; 4-cholesten-3-one,oxime; AVXS-101; AVXS-101 (previously known as scAAV9.CB.SMN); Adipose derived mesenchymal stem cell; Air; Amifampridine; Amifampridine Phosphate; Amifampridine Phosphate 10 MG Oral Tablet; Biocarn; Branaplam; Celecoxib; GH; HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY; Hydroxyurea; ISIS 396443; ISIS 396443 (BIIB058); ISIS 396443, BIIB058; Itraconazole; LEVOCARNITINE; LMI070; Leuprolide; Levocarnitine; Mestinon; Midazolam; N.a.; NUSINERSEN; NUSINERSEN SODIUM; Nerve Growth Factor; Norditropin SimpleXx; Norditropin SimpleXx 15 mg/1.5 ml; Nursinersen; Nusinersen; Nusinersen Injectable Product; Nusinersen Treatments; OLESOXIME; ONASEMNOGENE ABEPARVOVEC; Olesoxime; Onasemnogene Abeparvovec-xioi; Onasemnogene abeparvovec; Onasemnogene abeparvovec-xioi; Orfiril Saft; Other: Monthly assessments of functional motor abilities by a trained therapist; Other: Prospective observational registry; PARACETAMOL; Paracetamol; Paracetamol 120Mg/5mL Oral Suspension; Phenylbutyrate; Phosphate; Placebo; Procedure: Air stacking manuever; Procedure: Sham Procedure; Procedure: Sham procedure; Pyridostigmine; Pyridostigmine Bromide; Pyridostigmine bromide; RO6885247; RO6885247/F02; RO6885247/F03; RO7034067; RO7034067 / F12; RO7034067 / F13; RO7034067/F06 + solvente (RO; RO7034067/F06 with solvent (RO7034067/F08); RO7034067/F07 + solvente (RO; RO7034067/F07 with solvent (RO7034067/F09); RO7034067/F12; RO7034067/F13; RO7090919; RVG 03820; Reldesemtiv; Reldesemtiv 150 mg; Reldesemtiv 450 mg; Riluzole; Risdiplam; SESAME OIL, REFINED; SMART; SRK-015; Salbutamol; Sesame oil; Sodium phenylbutyrate; Sodium phenylbutyrate (NaPB); Somatotropin; Somatropin; Spinraza; Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide; TRO19622; Testosterone; VALPROATE SODIUM; VOLMAX*30CPR 4MG R.P.; VPA; Valproate; Valproate, Levocarnitine; Valproic Acid; Valproic Acid (VPA); Valproic Acid and Levocarnitine; Valproic acid; Zolgensma

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2016-000778-40-FR (EUCTR)	21/04/2017	24/10/2016	A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy	A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY	Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;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]	Product Name: RO7034067 Product Code: RO7034067 /F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 /F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	64	Phase 2	France;Belgium;Germany;Italy
2	EUCTR2016-000778-40-DE (EUCTR)	05/01/2017	21/07/2016	A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy	A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY	Type 1 Spinal Muscular Atrophy (SMA) 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]	Product Name: RO7034067 Product Code: RO7034067 /F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 /F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F12 INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 / F13 INN or Proposed INN: n/a Other descriptive name: RO7034067	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	64	Phase 2	Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China
3	EUCTR2016-000778-40-IT (EUCTR)	04/10/2016	16/08/2016	A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy	A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY	Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;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]	Product Name: RO7034067 Product Code: RO7034067 /F06 with solvent (RO7034067/F08) INN or Proposed INN: n/a Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067 /F07 with solvent (RO7034067/F09) INN or Proposed INN: n/a Other descriptive name: RO7034067	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	64	Phase 2	France;Belgium;Germany;Italy
4	EUCTR2016-000750-35-IT (EUCTR)	29/08/2016	28/06/2016	A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients	A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS.	Spinal Muscular Atrophy (SMA) MedDRA version: 19.0;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]	Product Name: RO7034067 Product Code: RO7034067 /F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067 /F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	186	Phase 2	United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden
5	EUCTR2016-000750-35-FR (EUCTR)		26/01/2018	A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients	A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS.	Spinal Muscular Atrophy (SMA) 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]	Product Name: RO7034067 Product Code: RO7034067 /F06 with solvent (RO7034067/F08) INN or Proposed INN: n.a Product Name: RO7034067 Product Code: RO7034067 /F07 with solvent (RO7034067/F09) INN or Proposed INN: n.a	F. Hoffmann-La Roche Ltd	NULL	NA	Female: yes Male: yes	186	Phase 2	United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden

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

EUCTR2016-000778-40-FR
(EUCTR)

21/04/2017

24/10/2016

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy

A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY

Type 1 Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;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]

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

France;Belgium;Germany;Italy

EUCTR2016-000778-40-DE
(EUCTR)

05/01/2017

21/07/2016

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy

A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY

Type 1 Spinal Muscular Atrophy (SMA)
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]

Product Name: RO7034067
Product Code: RO7034067 /F06 with solvent (RO7034067/F08)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067 /F07 with solvent (RO7034067/F09)
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067 / F12
INN or Proposed INN: n/a
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067 / F13
INN or Proposed INN: n/a
Other descriptive name: RO7034067

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China

EUCTR2016-000778-40-IT
(EUCTR)

04/10/2016

16/08/2016

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy

A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

France;Belgium;Germany;Italy

EUCTR2016-000750-35-IT
(EUCTR)

29/08/2016

28/06/2016

A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients

A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS.

Spinal Muscular Atrophy (SMA)
MedDRA version: 19.0;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]

Product Name: RO7034067
Product Code: RO7034067 /F06 with solvent (RO7034067/F08)
INN or Proposed INN: n.a
Product Name: RO7034067
Product Code: RO7034067 /F07 with solvent (RO7034067/F09)
INN or Proposed INN: n.a

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

186

Phase 2

United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden

EUCTR2016-000750-35-FR
(EUCTR)

26/01/2018

A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients

Spinal Muscular Atrophy (SMA)
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]

F. Hoffmann-La Roche Ltd

NULL

Female: yes
Male: yes

186

Phase 2

United States;Spain;Turkey;Switzerland;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden