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	EUCTR2015-001589-25-FR (EUCTR)	01/12/2015	07/12/2015	A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy	Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA	Spinal Muscular Atrophy MedDRA version: 18.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL , REFINED Other descriptive name: SESAME OIL , REFINED	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	173	Phase 2;Phase 3	France;Belgium;Netherlands;Germany;Italy;United Kingdom
2	EUCTR2015-001589-25-BE (EUCTR)	20/11/2015	25/09/2015	A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy	Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS	Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL , REFINED Other descriptive name: SESAME OIL , REFINED	F. Hoffmann-La Roche Ltd	NULL	Not Recruiting	Female: yes Male: yes	173	Phase 2;Phase 3	France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
3	EUCTR2015-001589-25-GB (EUCTR)	19/11/2015	04/08/2015	A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy	Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS	Spinal Muscular Atrophy MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL , REFINED Other descriptive name: SESAME OIL , REFINED	F. Hoffmann-La Roche Ltd	NULL	Not Recruiting	Female: yes Male: yes	173	Phase 2;Phase 3	France;Poland;Belgium;Netherlands;Germany;Italy;United Kingdom
4	EUCTR2015-001589-25-IT (EUCTR)	05/10/2015	11/08/2015	A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy	Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA	Spinal Muscular Atrophy MedDRA version: 18.0;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: Olesoxime Product Code: RO7090919 INN or Proposed INN: OLESOXIME INN or Proposed INN: SESAME OIL , REFINED Other descriptive name: SESAME OIL , REFINED	F. Hoffmann-La Roche Ltd	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	173		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

EUCTR2015-001589-25-FR
(EUCTR)

01/12/2015

07/12/2015

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Spinal Muscular Atrophy
MedDRA version: 18.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL , REFINED
Other descriptive name: SESAME OIL , REFINED

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

173

Phase 2;Phase 3

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

EUCTR2015-001589-25-BE
(EUCTR)

20/11/2015

25/09/2015

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy

Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS

Spinal Muscular Atrophy
MedDRA version: 20.1;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL , REFINED
Other descriptive name: SESAME OIL , REFINED

F. Hoffmann-La Roche Ltd

NULL

Not Recruiting

Female: yes
Male: yes

173

Phase 2;Phase 3

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

EUCTR2015-001589-25-GB
(EUCTR)

19/11/2015

04/08/2015

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy

Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS

Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL , REFINED
Other descriptive name: SESAME OIL , REFINED

F. Hoffmann-La Roche Ltd

NULL

Not Recruiting

Female: yes
Male: yes

173

Phase 2;Phase 3

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

EUCTR2015-001589-25-IT
(EUCTR)

05/10/2015

11/08/2015

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy

Multicenter, open-label, single arm study to evaluate long-term safety, tolerability, and effectiveness of 10mg/kg olesoxime in patients with SMA

Spinal Muscular Atrophy
MedDRA version: 18.0;Level: LLT;Classification code 10051203;Term: Spinal muscular atrophy congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: Olesoxime
Product Code: RO7090919
INN or Proposed INN: OLESOXIME
INN or Proposed INN: SESAME OIL , REFINED
Other descriptive name: SESAME OIL , REFINED

F. Hoffmann-La Roche Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

173

Italy