University of Auckland
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 26 Trials 
28 Trials

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Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Roxburgh, Richard
STRIDE, NCT04535609 / 2020-002855-40: An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients

Completed
2
213
Europe, Canada, US, RoW
Mavodelpar, REN001, Placebo
Reneo Pharma Ltd
Primary Mitochondrial Myopathy
09/23
10/23
ACHIEVE, NCT05481879 / 2022-000889-18: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Recruiting
1/2
104
Europe, RoW
DYNE-101, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Myotonic Dystrophy Type 1 (DM1)
07/26
07/26
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
McKeage, Mark
NCT03906331: Expanded Access for the Treatment of Cancers With Rearranged During Transfection (RET) Activation

Approved for marketing
N/A
Europe, Japan, US, RoW
Selpercatinib, LOXO-292, LY3527723
Eli Lilly and Company, Eli Lilly and Company
Non Small Cell Lung Cancer, Medullary Thyroid Cancer, Colon Cancer, Breast Cancer, Pancreatic Cancer, Papillary Thyroid Cancer, Other Solid Tumors With Evidence of Activating RET Alteration
 
 
Statland, Jeffrey
COURAGE-ALS, NCT04944784 / 2020-004040-29: A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)

Terminated
3
489
Europe, Canada, US, RoW
Reldesemtiv, Placebo
Cytokinetics
Amyotrophic Lateral Sclerosis
07/23
07/23
COURAGE OLE, NCT05442775 / 2021-004727-33: A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)

Terminated
3
71
Europe, Canada, US, RoW
Reldesemtiv
Cytokinetics, Cytokinetics Inc
Amyotrophic Lateral Sclerosis
06/23
06/23
Fortify, NCT05775848: Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)

Active, not recruiting
3
81
Europe, US, RoW
BBP-418 (ribitol), Placebo
ML Bio Solutions, Inc.
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
07/27
07/27
NCT04886518: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Hourglass Jul 2022 - Dec 2022 : Efficacy and safety data for treatment of EDS in patients with Myotonic Dystrophy Type 1
Active, not recruiting
2
30
Canada, US
Pitolisant Oral Tablet, Placebo oral tablet
Harmony Biosciences, LLC
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
10/23
10/24
MOMENTUM, NCT04004065 / 2019-000601-77: Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment

Checkmark From MOMENTUM trial for DMD
May 2021 - May 2021: From MOMENTUM trial for DMD
Checkmark Interim data from the MOMENTUM study
Dec 2020 - Dec 2020: Interim data from the MOMENTUM study
Checkmark Data from MOMENTUM trial for Duchenne muscular dystrophy
More
Active, not recruiting
2
62
Europe, Canada, US
Vesleteplirsen, SRP-5051
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
10/23
01/29
LION-CS101, NCT05230459: A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)

Recruiting
1/2
10
US
AB-1003 dose level 1, AB-1003 dose level 2, Placebo
Asklepios Biopharmaceutical, Inc.
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2, LGMD2I, Muscular Dystrophy, LGMD2, LGMD, FKRP, FKRP Mutation, Fukutin Related Protein
12/28
12/28
MOVE FSHD, NCT04635891: Motor Outcomes to Validate Evaluations in FSHD

Recruiting
N/A
450
Europe, Canada, US
University of Kansas Medical Center, FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche
FSHD
12/26
01/27
ENDURE, NCT06270719: An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

Recruiting
N/A
500
US
Delandistrogene Moxeparvovec, SRP-9001, delandistrogene moxeparvovec-rokl, ELEVIDYS, Standard of Care, Chronic glucocorticoid treatment
Sarepta Therapeutics, Inc.
Duchenne Muscular Dystrophy
12/29
12/38
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
NCT04174157: Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Recruiting
N/A
700
Europe, Japan, US, RoW
Prospective observational registry, Zolgensma
Novartis Pharmaceuticals, United BioSource, LLC
Spinal Muscular Atrophy (SMA)
06/38
06/38
Loaiza-Bonilla, Arturo
SUNRAY-01, NCT06119581: A Study of First-Line Olomorasib (LY3537982) and Pembrolizumab With or Without Chemotherapy in Patients With Advanced KRAS G12C-Mutant Non-small Cell Lung Cancer

Recruiting
3
1016
Europe, Canada, Japan, US, RoW
LY3537982, Olomorasib, Pembrolizumab, Placebo, Cisplatin, Carboplatin, Pemetrexed
Eli Lilly and Company, Loxo Oncology, Inc.
Carcinoma, Non-Small-Cell Lung, Neoplasm Metastasis
10/26
10/29
NCT05800015: A Trial to Learn How the Combination of Fianlimab With Cemiplimab and Chemotherapy Works Compared With Cemiplimab and Chemotherapy for Treating Adult Patients With Advanced Non-small Cell Lung Cancer

Recruiting
2/3
950
Canada, US, RoW
fianlimab, REGN3767, cemiplimab, REGN2810, Libtayo, Pemetrexed, Alimta, Paclitaxel, Carboplatin, Paraplatin, Cisplatin, Platinol, Placebo
Regeneron Pharmaceuticals
Non-small Cell Lung Cancer
01/30
12/31
R3767-ONC-2235, NCT05785767: A Study to Learn if a Combination of Fianlimab and Cemiplimab Versus Cemiplimab Alone is More Effective for Adult Participants With Advanced Non-Small Cell Lung Cancer (NSCLC)

Hourglass Nov 2024 - Dec 2024 : Initial data from trial in combination with Libtayo for advanced NSCLC
Recruiting
2/3
850
Canada, US, RoW
fianlimab, REGN3767, cemiplimab, REGN2810, Libtayo, Placebo
Regeneron Pharmaceuticals
Advanced Non-Small Cell Lung Cancer
03/30
02/32
Actuate 1801, NCT03678883 / 2018-003739-32: 9-ING-41 in Patients with Advanced Cancers

Active, not recruiting
2
350
Europe, Canada, US
9-ING-41, Gemcitabine - 21 day cycle, Gemzar, Doxorubicin., Doxil, Adriamycin, Lomustine, CCNU, Gleostine, Carboplatin., Paraplatin, Nab paclitaxel., Abraxane, Protein-bound paclitaxel, Nanoparticle albumin-bound paclitaxel, Paclitaxel., Taxol, Gemcitabine - 28 day cycle, Irinotecan, Camptosar
Actuate Therapeutics Inc., Actuate Therapeutics Inc
Cancer, Pancreatic Cancer, Sarcoma, Renal Cancer, Refractory Cancer, Refractory Neoplasm, Refractory Non-Hodgkin Lymphoma, Pancreatic Adenocarcinoma, Resistant Cancer, Neoplasm Metastasis, Neoplasm of Bone, Neoplasm, Breast, Neoplasm of Lung, Neoplasms,Colorectal, Neoplasms Pancreatic, Malignant Glioma, Malignancies, Malignancies Multiple, Bone Metastases, Bone Neoplasm, Bone Cancer, Pancreas Cancer, Pancreatic Neoplasms, Breast Neoplasms, Acute T Cell Leukemia Lymphoma
01/25
01/26
NCT03906331: Expanded Access for the Treatment of Cancers With Rearranged During Transfection (RET) Activation

Approved for marketing
N/A
Europe, Japan, US, RoW
Selpercatinib, LOXO-292, LY3527723
Eli Lilly and Company, Eli Lilly and Company
Non Small Cell Lung Cancer, Medullary Thyroid Cancer, Colon Cancer, Breast Cancer, Pancreatic Cancer, Papillary Thyroid Cancer, Other Solid Tumors With Evidence of Activating RET Alteration
 
 
Walker, Michaela
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
Rodrigues, Miriam
ACHIEVE, NCT05481879 / 2022-000889-18: Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Recruiting
1/2
104
Europe, RoW
DYNE-101, Placebo
Dyne Therapeutics, Dyne Therapeutics, Inc
Myotonic Dystrophy Type 1 (DM1)
07/26
07/26
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
Whited, Kaylene
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Active, not recruiting
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
10/24
12/24
ONYX, NCT05626855 / 2022-001771-14: Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab-

Active, not recruiting
3
260
Europe, US
Apitegromab
Scholar Rock, Inc., Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
11/26
01/27
NCT04886518: Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Hourglass Jul 2022 - Dec 2022 : Efficacy and safety data for treatment of EDS in patients with Myotonic Dystrophy Type 1
Active, not recruiting
2
30
Canada, US
Pitolisant Oral Tablet, Placebo oral tablet
Harmony Biosciences, LLC
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
10/23
10/24
FREEDOM-DM1, NCT06204809: Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants with Myotonic Dystrophy Type 1

Recruiting
1
32
Europe, Canada, US
PGN-EDODM1 for infusion, Placebo
PepGen Inc
Myotonic Dystrophy 1
04/25
04/25
GRASP-01-002, NCT05257473: Defining Endpoints in Becker Muscular Dystrophy

Recruiting
N/A
80
Europe, US, RoW
Virginia Commonwealth University, Edgewise Therapeutics, Inc.
Becker Muscular Dystrophy, Muscular Dystrophies, Muscular Dystrophy in Children, Muscular Dystrophy, Becker
05/25
05/26
Clark, Alys
NCT06738589: The Inflate Study; Defining the Soft Palate in the Upper Airway Flow Pathway

Not yet recruiting
N/A
50
RoW
non-invasive ventilation
Fisher and Paykel Healthcare, The University of Auckland
Focus of the Study is the Anatomy of the Soft Palate, Non-invasive Ventilation
01/26
08/27

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