Clinical trials: ALD
Cerebral Adrenoleukodystrophy
A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)
An Open-Label, multicenter study in male pediatric patients with cerebral x-linked adrenoleukodystrophy (cald) to assess the effects of MIN-102 treatment on disease progression prior to human stem cell transplant (HSCT)
- Trial name
- NEXUS (NCT04528706)
- Testing
- MIN-102
- Where
- 5 sites in Argentina, France and 2 more
- Volunteers
- 13 people
- Sponsor
- Minoryx Therapeutics, S.L.
ALD (Adrenoleukodystrophy)
Study to Assess PXL065 in Subjects With Adrenomyeloneuropathy (AMN) Form of X-linked Adrenoleukodystrophy (X-ALD or ALD)
A randomized open-label Phase 2a study to assess the pharmacokinetics and pharmacodynamic parameters of PXL065 after 12 weeks of treatment in male subjects with adrenomyeloneuropathy (AMN).
- Trial name
- NCT05200104
- Testing
- PXL065
- Sponsor
- Poxel SA
Alcohol-associated Liver Disease
RESTORE TRIAL: Study of the Efficacy and Safety of Pemvidutide in the Treatment of Alcohol-Associated Liver Disease (ALD)
This is a Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pemvidutide in subjects with ALD.
- Trial name
- NCT07009860
- Testing
- Pemvidutide
- Where
- 37 sites in United States, Puerto Rico
- Volunteers
- 121 people
- Sponsor
- Altimmune, Inc.
X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD)
Observational Study to Evaluate Allogeneic HSCT Outcomes for Cerebral Adrenoleukodystrophy (CALD)
Study ALD-103 will be a multi-site, global, prospective and retrospective data collection study that is designed to evaluate outcomes of allo-HSCT in male subjects with CALD ≤17 years of age.
- Conditions
- X-Linked Adrenoleukodystrophy (X-ALD), Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
- Trial name
- NCT02204904
- Testing
- Allo-HSCT
- Where
- 13 sites in United States, Argentina and 5 more
- Volunteers
- 59 people
- Sponsor
- Genetix Biotherapeutics Inc.
Liver Diseases, Alcoholic
A Study to Investigate the Safety and Efficacy of Efimosfermin Alfa Compared With Placebo in Adult Participants With Alcohol-related Liver Disease (ALD)
This is a dose-ranging study to evaluate safety of efimosfermin alfa and to establish proof-of-concept that efimosfermin alfa therapy provides benefit in participants with ALD.
- Trial name
- ALLSTAR (NCT07791043)
- Testing
- Efimosfermin alfa
- Where
- 4 sites in United States, Argentina
- Volunteers
- 274 people
- Sponsor
- GlaxoSmithKline
X-linked Adrenoleukodystrophy
Disease Progression in Women With X-linked Adrenoleukodystrophy
Observational, single-site prospective and minimally interventional study in women with X-linked adrenoleukodystrophy (ALD), conducted in France.
- Trial name
- NCT06178120
- Where
- France
- Volunteers
- 40 people
- Sponsor
- Minoryx Therapeutics, S.L.
AMN, AMN Gene Mutation
A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)
This is a Phase 1/2 randomized, blinded, dose-escalation study to evaluate the safety and efficacy of intrathecal (IT) administration of SBT101, a recombinant adeno-associated virus serotype 9 (AAV9) containing a functional copy…
- Conditions
- AMN, AMN Gene Mutation, X-ALD
- Trial name
- PROPEL (NCT05394064)
- Testing
- SBT101 Treatment
- Where
- 2 sites in United States, Netherlands
- Volunteers
- 9 people
- Sponsor
- SwanBio Therapeutics, Inc.
Infantile Refsum's Disease, Zellweger Syndrome
Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid
OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II.
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Adrenoleukodystrophy, Peroxisomal Disorders
- Trial name
- NCT00007020
- Testing
- Cholic Acids
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD)
Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study (Study ALD-102 or Study…
- Conditions
- Cerebral Adrenoleukodystrophy (CALD), Adrenoleukodystrophy (ALD), X-Linked Adrenoleukodystrophy (X-ALD)
- Trial name
- NCT02698579
- Testing
- No interventional drug product utilized in this follow-up study
- Where
- 13 sites in United States, Argentina and 7 more
- Volunteers
- 64 people
- Sponsor
- Genetix Biotherapeutics Inc.
Cerebral Adrenoleukodystrophy (CALD)
A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel
The main aim of this study is to assess and describe the safety outcomes, including newly diagnosed malignancies, of patients with CALD treated with eli-cel in the post-marketing setting (tradename Skysona) and to describe major…
- Trial name
- Stargazer (NCT06224413)
- Testing
- No Intervention
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Genetix Biotherapeutics Inc.
X-Linked Adrenoleukodystrophy
A Study to Prospectively Assess Disease Progression in Male Children With X-ALD
This is a non-interventional, multi-center study that follows general principles of periodic assessment of X-ALD patients in routine practice.
- Trial name
- NCT03278899
- Where
- 3 sites in United States
- Volunteers
- 100 people
- Sponsor
- NeuroVia, Inc.
Adrenoleukodystrophy
A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the adrenomyeloneuropathy (AMN) phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the…
- Trial name
- Advance (NCT03231878)
- Testing
- Leriglitazone
- Where
- 10 sites in United States, France and 6 more
- Volunteers
- 116 people
- Sponsor
- Minoryx Therapeutics, S.L.
Healthy
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of OP-101 After Intravenous Administration in Healthy Volunteers
A clinical study to measure the effect of OP-101 after being administered intravenously in healthy volunteers.
- Trial name
- NCT03500627
- Testing
- OP-101
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Orpheris, Inc.
Inherited Metabolic Disorders (IMD)
A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456
A follow-up study to evaluate the safety and clinical outcomes of patients with inherited metabolic disorders (IMD) who have undergone hematopoietic stem cell transplantation (HSCT) with MGTA-456
- Trial name
- IMD-002 (NCT04008849)
- Testing
- Safety and efficacy assessments
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Magenta Therapeutics, Inc.
Cerebral Adrenoleukodystrophy (CALD)
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the…
- Trial name
- NCT01896102
- Testing
- Lenti-D Drug Product (eli-cel)
- Where
- 8 sites in United States, Argentina and 4 more
- Volunteers
- 32 people
- Sponsor
- Genetix Biotherapeutics Inc.
Cerebral Adrenoleukodystrophy (CALD)
A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and…
- Trial name
- NCT03852498
- Testing
- Lenti-D
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 35 people
- Sponsor
- Genetix Biotherapeutics Inc.
Adrenomyeloneuropathy, Adrenoleukodystrophy
MD1003-AMN MD1003 in Adrenomyeloneuropathy
The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
- Conditions
- Adrenomyeloneuropathy, Adrenoleukodystrophy, AMN
- Trial name
- NCT02961803
- Testing
- MD1003 100 mg capsule
- Where
- 4 sites in France, Germany and 1 more
- Volunteers
- 67 people
- Sponsor
- MedDay Pharmaceuticals SA
Liver Diseases, Alcoholic
A Study to Investigate the Safety and Efficacy of GSK4532990 Compared With Placebo in Adult Participants Aged 18 to 70 Years With Alcohol-related Liver Disease
The goal of this study is to assess the safety and efficacy of GSK4532990 in participants with alcohol-related liver disease.
- Trial name
- STARLIGHT (NCT06613698)
- Testing
- GSK4532990
- Where
- 140 sites in United States, Argentina and 16 more
- Volunteers
- 393 people
- Sponsor
- GlaxoSmithKline
Hepatitis
Study to Evaluate the Safety and Efficacy of Larsucosterol in Participants With Alcohol-associated Hepatitis (AH)
The primary purpose of this study is to evaluate the safety and efficacy of larsucosterol, as determined by transplant-free survival through Day 90 in participants with severe alcohol-associated hepatitis (AH) with pre-treatment…
- Trial name
- NCT07428538
- Testing
- Larsucosterol
- Where
- 92 sites in United States
- Volunteers
- 350 people
- Sponsor
- Bausch Health Americas, Inc.