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:
- Initial cohort of 17 boys completed 24 months of follow-up (US only). Cohort recently expanded to treat an additional 8 patients (in US and EU). 17 boys have completed 24 months of follow-up; 15/17 (88%) remain free of major functional disabilities.
- No treatment-related graft vs. host disease (GVHD) reported to date; adverse events (AE) profile consistent with myeloablative conditioning with busulfan and cyclophosphamide.
- Three AEs considered related or possibly related to drug product: BK-mediated viral cystitis (SAE, grade 3), tachycardia (grade 1), vomiting (grade 1).
- 4 additional boys treated since January 2017
- First boy treated in Europe
More information on this trial can be found here and at the adrenoleukodystrophy database.
(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.
- Male, 2-10 years old
- Diagnosis of cerebral X-linked ALD as documented by:
- Elevated plasma levels of very long chain fatty acids (VLCFA)
- MRI Loes score > 10 and < 20 and gadolinium enhancement
- NFS = 1 to 5
- History of bone marrow transplant
- Continued use of Lorenzo’s Oil, statins, or dietary regimens to lower VLCFA
- Unstable adrenal function
(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.
- Counteracts cellular dysfunction preventing neurodegeneration
- Neuroprotection against VLCFA (protects nerve cells)
- Recovery of motor function
- Reduced neuroinflammation
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.
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.
(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:
- Screening period: within 30 days of first dose
- Main treatment period of 12 weeks (Part 1 – from Screening to Week 12)
- Long Term Treatment (LTT) period (Part 2 – Week 13 through Week 96)
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.