Current ALD Clinical Trials

There are a growing number of opportunities for people with ALD/AMN to participate in clinical research, both interventional and observational. View a listing of some of the current clinical trials to determine if you may be a candidate for ongoing research. You can also visit Clinicaltrials.gov to find other ALD/AMN trials not listed below. The following image shows both the current, standard therapies and the current or scheduled clinical trials separated by each ALD phenotype down the left-hand side.

ALD-102 (Lenti-D Gene Therapy)

(currently recruiting, limited)
bluebird bio, Boston, MA
Open label, multi-center, single arm, global study
Initial results from an ongoing phase 2/3 clinical trial:

More information on this trial can be found here and at the adrenoleukodystrophy database.

OP-101

(Planned recruitment in 2018)
Orpheris, Inc., Redwood City, CA
Orpheris is the first company focusing on advanced cerebral disease. Dendrimer conjugated N-acetylcysteine – an approved drug – targets microglial cells in the CNS that drive neuroinflammation. Microglial cells play an important role in inflammation in CALD, and this compound should be able to stop the inflammation and stabilize neurological function. Opheris recently conducted a phase 1 study of safety pharmacokinetics and now are planning their first phase 1/2/3 study.

Eligibility Criteria:

Exclusion Criteria:

MIN-102

(currently recruiting in Europe; US recruitments expected in 2018)
Minoryx Therapeutics, Barcelona, Spain
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked ALD to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of ALD.

A 2 year randomized placebo-controlled study – after the 2 years option to enter into the extension phase with MIN-102.
Learn more about the study here.

Vitamin D

(currently recruiting)
Stanford University, Palo Alto, CA
A pilot study of vitamin D in boys with cerebral ALD.
Prior research suggests that higher vitamin D levels in the blood are associated with reduced brain inflammation among individuals with multiple sclerosis, a disease that is similar to the cerebral demyelinating form of ALD. However, serious side effects (e.g. hypercalcemia, kidney stones) can occur if vitamin D levels get too high.
The current study is designed to establish a safe dose of vitamin D for boys with ALD. Although the doses chosen for this study are expected to be safe, the investigators will monitor participants for early signs of vitamin D-related toxicity. The investigators will also examine whether or not vitamin D supplementation affects markers of oxidative stress and inflammation in the blood and brains of ALD boys.
Single-arm, dose-escalation starting at 2,000 IU of vitamin D3 daily for a 6 month period, followed by a 4,000 IU daily for at least 6 months thereafter. No placebo group.
For more information, click here.

NV-1205

(Currently enrolling non-US sites)
Neurovia, Inc., San Francisco, CA
This study is to evaluate the safety, pharmacokinetics, and efficacy of NV1205 in pediatric patients (boys) diagnosed with cerebral ALD.
The study consists of:

In Part 1, subjects will have an initial 4-week treatment period at the assigned dose and, if no safety concerns are noted, subjects continue for another 8 weeks of extended safety assessment.
There will be several cohorts of subjects enrolled. After each Cohort has completed the 4-week initial safety assessment, the safety data will be reviewed by an independent Data Safety Monitoring Board (DSMB) and, subject to DSMB recommendation, Cohort 2 will be enrolled and receive the next dose level. After Cohort 2 has completed 4 weeks of treatment, the DSMB will review all available safety data (Cohorts 1 and 2) and, subject to DSMB recommendation, Cohort 3 will be enrolled and receive the next dose level. Additional Cohorts may be enrolled at the recommendation of the DSMB with an incremental dose increase.
In Part 2, subjects will continue to receive treatment in the LTT period of the study.
More information can be found here.