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_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2010-020386-24-GB (EUCTR)	31/01/2011	08/10/2010	Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patients	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 14.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]		TROPHOS SA	NULL	Not Recruiting			Female: yes Male: yes	150	Phase 2	France;Belgium;Germany;Netherlands;Italy;United Kingdom
2	EUCTR2010-020386-24-DE (EUCTR)	29/12/2010	28/07/2010	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy	Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime	TROPHOS SA	NULL	Not Recruiting			Female: yes Male: yes	150	Phase 2	Belgium;Netherlands;Germany;United Kingdom;Italy
3	EUCTR2010-020386-24-NL (EUCTR)	18/11/2010	28/06/2010	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy	Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime	TROPHOS SA	NULL	Not Recruiting			Female: yes Male: yes	150	Phase 2	Belgium;Germany;Netherlands;United Kingdom;Italy
4	NCT01302600 (ClinicalTrials.gov)	November 2010	18/2/2011	Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.	Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.	Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III Non Ambulant	Drug: Olesoxime;Drug: Placebo	Hoffmann-La Roche	Association Française contre les Myopathies (AFM), Paris	Completed	3 Years	25 Years	Both	165	Phase 2	Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom
5	EUCTR2010-020386-24-BE (EUCTR)	29/10/2010	27/07/2010	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years). MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy	Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime	TROPHOS SA	NULL	Not Recruiting			Female: yes Male: yes	150	Phase 2	Belgium;Germany;Netherlands;United Kingdom;Italy
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
6	EUCTR2010-020386-24-IT (EUCTR)	14/09/2010	01/09/2010	Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients	Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients	Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years MedDRA version: 9.1;Level: LLT;Classification code 10032950	Product Name: olesoxime Product Code: TRO19622 INN or Proposed INN: olesoxime	TROPHOS	NULL	Not Recruiting			Female: yes Male: yes	150	Phase 2	Belgium;Germany;Netherlands;United Kingdom;Italy
7	EUCTR2006-006845-14-FR (EUCTR)	20/03/2007	15/02/2007	Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.	Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (SMA) MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy	Product Code: TRO19622 Other descriptive name: 4-cholesten-3-one,oxime	TROPHOS	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes		Phase 1b	France
8	EUCTR2010-020386-24-FR (EUCTR)		01/06/2010	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.	Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years) MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy	Product Name: OLESOXIME Product Code: TRO19622 INN or Proposed INN: olesoxime Other descriptive name: 4-cholesten-3-one, oxime	TROPHOS SA	NULL	NA			Female: yes Male: yes		Phase 2	France;Belgium;Germany;Netherlands;United Kingdom;Italy

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

EUCTR2010-020386-24-GB
(EUCTR)

31/01/2011

08/10/2010

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 yrs SMA patients

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 14.0;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

TROPHOS SA

NULL

Not Recruiting

Female: yes
Male: yes

150

Phase 2

France;Belgium;Germany;Netherlands;Italy;United Kingdom

EUCTR2010-020386-24-DE
(EUCTR)

29/12/2010

28/07/2010

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy

Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime

TROPHOS SA

NULL

Not Recruiting

Female: yes
Male: yes

150

Phase 2

Belgium;Netherlands;Germany;United Kingdom;Italy

EUCTR2010-020386-24-NL
(EUCTR)

18/11/2010

28/06/2010

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy

Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime

TROPHOS SA

NULL

Not Recruiting

Female: yes
Male: yes

150

Phase 2

Belgium;Germany;Netherlands;United Kingdom;Italy

NCT01302600
(ClinicalTrials.gov)

November 2010

18/2/2011

Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.

Phase II, Multicenter, Randomized, Adaptive, Double-blind, Placebo Controlled Study to Assess Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Year Old Spinal Muscular Atrophy (SMA) Patients.

Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III Non Ambulant

Drug: Olesoxime;Drug: Placebo

Hoffmann-La Roche

Association Française contre les Myopathies (AFM), Paris

Completed

3 Years

25 Years

Both

165

Phase 2

Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom

EUCTR2010-020386-24-BE
(EUCTR)

29/10/2010

27/07/2010

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years).
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy

Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime

TROPHOS SA

NULL

Not Recruiting

Female: yes
Male: yes

150

Phase 2

Belgium;Germany;Netherlands;United Kingdom;Italy

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

EUCTR2010-020386-24-IT
(EUCTR)

14/09/2010

01/09/2010

Phase II,multicenter,randomized,adaptive,double-blind,placebo controlled study to assess safety and efficacy of olexosime (TRO19622)in 3-25 year old Spinal Muscular Atrophy (SMA) patients

Spinal Muscular Atrophy - type II or III in non ambulant patients aged 3-25 years
MedDRA version: 9.1;Level: LLT;Classification code 10032950

Product Name: olesoxime
Product Code: TRO19622
INN or Proposed INN: olesoxime

TROPHOS

NULL

Not Recruiting

Female: yes
Male: yes

150

Phase 2

Belgium;Germany;Netherlands;United Kingdom;Italy

EUCTR2006-006845-14-FR
(EUCTR)

20/03/2007

15/02/2007

Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (SMA)
MedDRA version: 9.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy

Product Code: TRO19622
Other descriptive name: 4-cholesten-3-one,oxime

TROPHOS

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 1b

France

EUCTR2010-020386-24-FR
(EUCTR)

01/06/2010

Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients.

Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years)
MedDRA version: 12.1;Level: LLT;Classification code 10041582;Term: Spinal muscular atrophy

Product Name: OLESOXIME
Product Code: TRO19622
INN or Proposed INN: olesoxime
Other descriptive name: 4-cholesten-3-one, oxime

TROPHOS SA

NULL

Female: yes
Male: yes

Phase 2

France;Belgium;Germany;Netherlands;United Kingdom;Italy