Bone Marrow Disorder Clinical Trials
A listing of Bone Marrow Disorder medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.
Found 247 clinical trials
A Phase 1, Open-Label, Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms (LIMBER)
The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of INCB057643 as monotherapy or combination with ruxolitinib for participants with myelofibrosis and other myeloid neoplasms.
- 0 views
- 24 Oct, 2022
- 21 locations
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS) (HIMALAYA)
This is a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants,18 to 80 years of age with ALS followed by an openlabel, longterm extension period. Study ACT16970 consists …
- 40 views
- 27 Jan, 2023
- 18 locations
A Phase 1/2, Open-Label, Multicenter Study of INCB000928 Administered as a Monotherapy in Participants With Anemia Due to Myelodysplastic Syndromes or Multiple Myeloma
This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.
- 1 views
- 21 Oct, 2022
- 12 locations
SMART-ALD - A New Lifestyle Intervention to Improve Quality of Life in Women With X-linked Adrenoleukodystrophy (X-ALD)
progressive myelopathy and peripheral neuropathy, which may also be prevalent in up to 80% of females carrying the X-ALD gene defect. Since signs and symptoms in females are usually milder and with a later
- 0 views
- 04 Oct, 2022
- 2 locations
The Adult Degenerative Scoliosis Exercise Trial (Pilot Study) (ADSET)
Adult degenerative scoliosis is the most common spine deformity in adults. Patients present a lateral curvature of the spine and vertebra rotation. Curves meeting indications for treatment affect 24% of the aging adult population. Adult scoliosis causes pain, curve progression, and cosmetic deformity affecting quality of life and function. Pain …
- 0 views
- 07 Oct, 2022
- 1 location
A Phase 3b Study to Evaluate Higher Dose Nusinersen (BIIB058) in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam (ASCEND)
The primary objective of this study is to evaluate motor function following treatment with HD nusinersen in participants with spinal muscular atrophy (SMA) previously treated with risdiplam. The secondary objective of this study is to evaluate the safety and tolerability of HD nusinersen in participants with SMA previously treated with …
- 0 views
- 21 Oct, 2022
- 12 locations
A Two-Part, Seamless, Multi-Center, Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Ambulant Patients With Spinal Muscular Atrophy
Risdiplam works by helping the body produce more survival motor neuron (SMN) protein throughout the body. This means fewer motor neurons - nerve cells that pass impulses from nerves to muscles to cause movement - are lost, which may improve how well muscles work in people with SMA. RO7204239 is …
- 0 views
- 23 Oct, 2022
- 8 locations
A Multicenter, Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of FAB122 in Patients With Amyotrophic Lateral Sclerosis (ADORE)
Multicenter, multinational, double-blind, randomized (2:1), placebo-controlled Phase III study to investigate the efficacy and safety of 100 mg FAB122 once daily as oral formulation in ALS patients.
- 1 views
- 22 Oct, 2022
- 26 locations
A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
- 0 views
- 04 Oct, 2022
- 9 locations
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS) (HIMALAYA)
This is a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants,18 to 80 years of age with ALS followed by an openlabel, longterm extension period. Study ACT16970 consists …
- 0 views
- 07 Oct, 2022
- 28 locations