Adrenoleukodystrophy Clinical Trials 2022

Browse 8 Adrenoleukodystrophy Medical Studies Across 10 Cities
2 Phase 3 Trial · 16 Adrenoleukodystrophy Clinics

Condition

Location

Distance

Age

Clinical Trials Adrenoleukodystrophy Clinical Trials 2022

Reviewed by Michael Gill, B. Sc.

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Phase-Based Progress Estimates

SBT101for AMN

No Cost For Treatment

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 of the human adenosine triphosphate (ATP)-binding cassette transporter subfamily D member 1 (ABCD1; hABCD1) gene, in adult patients with adrenomyeloneuropathy (AMN) aged 18-65 years. Patients will receive a single dose of SBT101 via IT route (or an imitation procedure) and will be followed for safety and efficacy for 2 years. Patients receiving SBT101 will be followed for an additional 3 years (5 total) for Safety. Patients receiving an imitation procedure will be offered the opportunity to receive SBT101 after 2 years, as data indicate.

No Cost For Treatment

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Phase-Based Progress Estimates

Pramipexolefor Adrenoleukodystrophy

Waitlist Available

FDA Approved Drug

This trial will study whether a medication called pramipexole can help reduce symptoms of restless leg syndrome (RLS) in women with a rare disorder called X-linked adrenoleukodystrophy (ALD). Pramipexole is approved by the FDA to treat Adrenoleukodystrophy, a condition that affects the nervous system. Pramipexole has previously been approved by the FDA to treat a different condition.

Waitlist Available

FDA Approved Drug

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Phase-Based Progress Estimates

VK0214for Adrenoleukodystrophy

No Cost For Treatment

This trial is testing a new drug, VK0214, given in multiple doses to see what the safest dose is. Up to 36 people will be enrolled, with some given the drug and some given a placebo. VK0214 is used to treat Adrenoleukodystrophy. VK0214 was previously approved by the FDA for a different condition. This treatment is free.

No Cost For Treatment

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Phase-Based Progress Estimates

Lenti-Dfor Adrenoleukodystrophy

Minneapolis, MN

Waitlist Available

This trial will enroll males with CALD who are less than or equal to 17 years old. The trial will be open-label, meaning that both the participants and the study staff will know which participants are receiving the Lenti-D Drug Product. The trial will be conducted at multiple sites internationally. Approximate 35 participants will be infused with Lenti-D Drug Product. Lenti-D is an FDA-approved drug used to treat patients with Adrenoleukodystrophy. This is a rare condition that affects the nervous system. There is no placebo group in this clinical trial.

Waitlist Available

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Phase-Based Progress Estimates

Blood And Marrow Transplantfor Thalassemia

Minneapolis, MN

This trial is for people with high-risk non-malignant diseases who don't have a suitable HLA-matched sibling donor, using a reduced intensity conditioning T-cell replete haploidentical donor. Blood and Marrow Transplant is used to treat Thalassemia. This treatment has previously been approved by the FDA for a different condition. No patients in this trial will receive a placebo.

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Phase-Based Progress Estimates

Activefor Adrenoleukodystrophy

Waitlist Available

This trial will test if MIN-102 is an effective and safe treatment for male patients with AMN phenotype of X-linked adrenoleukodystrophy. The primary treatment for adrenoleukodystrophy is active treatment.

Waitlist Available

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Phase-Based Progress Estimates

Stem Cell Transplantationfor Hunter Syndrome

Minneapolis, MN

This trial tests a new way to do bone marrow transplants that may be safer for people with certain disorders. The primary treatment being studied is IMD Preparative Regimen, and it is being studied for its potential to treat Hunter Syndrome. There will be no placebo group in this trial, as all patients will receive the active treatment. This treatment has previously been approved by the FDA for a different condition.

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Phase-Based Progress Estimates

Hematopoietic Stem Cell Infusionfor Sphingomyelin Lipidosis

Waitlist Available

This trial is testing a new way to give stem cell transplants that may help prevent GVHD while still being effective. The purpose of this trial is to test the safety and effectiveness of hematopoietic stem cell infusion in treating Sphingomyelin Lipidosis. All patients in this trial will receive the treatment, and there will be no placebo group. The treatment is free for all participants. This type of treatment has previously been approved by the FDA for a different condition.

Waitlist Available

8 Adrenoleukodystrophy Clinical Trials Near Me

Worcester, MA

Boston, MA

Palo Alto, CA

Baltimore, MD

More Trial Locations

An interactive map to display trial locations for University of Massachusetts Chan Medical School, Massachusetts General Hospital, Viking Clinical Site 203, Viking Clinical Site 201, and 11 more locations.

Top Hospitals for Adrenoleukodystrophy Clinical Trials

Location

Active Adrenoleukodystrophy Clinical Trials

All Time Trials for Adrenoleukodystrophy

First Recorded Adrenoleukodystrophy Trial

Trials

Massachusetts General Hospital, Boston, Massachusetts

2

3

2017

Pramipexole for Adrenoleukodystrophy

, Active for Adrenoleukodystrophy

University of Massachusetts Chan Medical School, Worcester, Massachusetts

1

1

2022

Viking Clinical Site 203, Palo Alto, California

1

1

2021

VK0214 for Adrenoleukodystrophy

Viking Clinical Site 201, Baltimore, Maryland

1

1

2021

VK0214 for Adrenoleukodystrophy

Viking Clinical Site 207, Gainesville, Florida

1

1

2021

VK0214 for Adrenoleukodystrophy

Top Treatments for Adrenoleukodystrophy Clinical Trials

Treatment Name

Active Adrenoleukodystrophy Clinical Trials

All Time Trials for Adrenoleukodystrophy

First Recorded Adrenoleukodystrophy Trial

Trials

SBT101

1

1

2022

Pramipexole

1

1

2021

Pramipexole for Adrenoleukodystrophy

VK0214

1

1

2021

VK0214 for Adrenoleukodystrophy

Lenti-D

1

2

2016

Lenti-D for Adrenoleukodystrophy

Blood and Marrow Transplant

1

1

2018

Blood and Marrow Transplant for Thalassemia

Top Cities for Adrenoleukodystrophy Clinical Trials

City

Active Adrenoleukodystrophy Clinical Trials

Top Active Site for Adrenoleukodystrophy Clinical Trials

Trial List

Boston, Massachusetts

3

Massachusetts General Hospital

Pramipexole for Adrenoleukodystrophy

, Lenti-D for Adrenoleukodystrophy

, Active for Adrenoleukodystrophy

Minneapolis, Minnesota

3

University of Minnesota

Lenti-D for Adrenoleukodystrophy

, Blood and Marrow Transplant for Thalassemia

, Stem Cell Transplantation for Hunter Syndrome

Palo Alto, California

2

Viking Clinical Site 203

VK0214 for Adrenoleukodystrophy

, Lenti-D for Adrenoleukodystrophy

Baltimore, Maryland

2

Viking Clinical Site 201

VK0214 for Adrenoleukodystrophy

, Active for Adrenoleukodystrophy

Worcester, Massachusetts

1

University of Massachusetts Chan Medical School

Which phases are most popular for adrenoleukodystrophy clinical trials?

Phase

Active Adrenoleukodystrophy Clinical Trials

Number of Unique Treatments

Number of Active Locations

Trial List

Phase 1

1

1

5

VK0214 for Adrenoleukodystrophy

Phase 1 & 2

2

2

1

, hematopoietic stem cell infusion for Sphingomyelin Lipidosis

Phase 2

2

2

2

Blood and Marrow Transplant for Thalassemia

, Stem Cell Transplantation for Hunter Syndrome

Phase 2 & 3

1

1

0

Active for Adrenoleukodystrophy

Phase 3

1

1

0

Lenti-D for Adrenoleukodystrophy

Phase 4

1

1

0

Pramipexole for Adrenoleukodystrophy

How many adrenoleukodystrophy clinical trials are open to youth and / or seniors?

Age Group

Active Adrenoleukodystrophy Clinical Trials

Trial List

< 18

1

Lenti-D for Adrenoleukodystrophy

18+

4

, Pramipexole for Adrenoleukodystrophy

, VK0214 for Adrenoleukodystrophy ... View More

< 65

2

Blood and Marrow Transplant for Thalassemia

, Stem Cell Transplantation for Hunter Syndrome

Any Age

1

hematopoietic stem cell infusion for Sphingomyelin Lipidosis

Most Recent Adrenoleukodystrophy Clinical Trials

Clinical Trial

Began Recruiting Date

Phase

6/30/2022

Phase 1 & 2

Pramipexole for Adrenoleukodystrophy

9/1/2021

Phase 4

VK0214 for Adrenoleukodystrophy

6/22/2021

Phase 1

Lenti-D for Adrenoleukodystrophy

1/24/2019

Phase 3

Blood and Marrow Transplant for Thalassemia

7/2/2018

Phase 2

Phase 3 Adrenoleukodystrophy Clinical Trials

Clinical Trial

Began Recruiting Date

Phase

Lenti-D for Adrenoleukodystrophy

1/24/2019

Phase 3

Active for Adrenoleukodystrophy

12/8/2017

Phase 2 & 3

Pediatric Adrenoleukodystrophy Clinical Trials

Clinical Trial

Began Recruiting Date

Phase

Lenti-D for Adrenoleukodystrophy

1/24/2019

Phase 3

Blood and Marrow Transplant for Thalassemia

7/2/2018

Phase 2

Stem Cell Transplantation for Hunter Syndrome

7/10/2014

Phase 2

hematopoietic stem cell infusion for Sphingomyelin Lipidosis

4/1/2011

Phase 1 & 2

Senior Friendly Adrenoleukodystrophy Clinical Trials

Clinical Trial

Began Recruiting Date

Phase

6/30/2022

Phase 1 & 2

Pramipexole for Adrenoleukodystrophy

9/1/2021

Phase 4

VK0214 for Adrenoleukodystrophy

6/22/2021

Phase 1

Active for Adrenoleukodystrophy

12/8/2017

Phase 2 & 3

hematopoietic stem cell infusion for Sphingomyelin Lipidosis

4/1/2011

Phase 1 & 2

About The Author

Michael Gill - B. Sc.

First Published: October 8th, 2021

Last Reviewed: August 20th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

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