Brief summary

This is a phase IIb randomised double-blind, placebo-controlled dose ranging trial to determine the safety and efficacy of vitamin D3 in preventing the risk of MS in patients with a first demyelinating event.

There is a great deal of lay information to suggest that people with MS or at risk of MS should take oral Vitamin D supplementation. However, no adequately powered scientific study has examined the role of Vitamin D3 supplementation as prevention or treatment for MS. There is insufficient commercial interest in conducting such a study, but the need for evidence is great. If effective, oral Vitamin D supplementation could provide a low cost MS therapy with virtually no side effects. Therefore, MS Research Australia is sponsoring this trial with the support of state MS societies, other trusts and foundations and community donations.

The trial will assess the efficacy of oral vitamin D3 in preventing the development of multiple sclerosis in participants at high risk of MS (that is people with a first demyelinating event, FDE).

The trial will compare three doses of oral daily vitamin D3 (1,000IU 5,000IU or 10,000IU/day) to placebo. After agreeing to participate, participants will be randomly and blindly assigned to one of these four study arms and will continue to take study medication for 48 weeks.

The primary end-point measure will be the onset of a second demyelinating event as determined clinically or by MRI scan. The trial will also assess whether there is a dose-response relationship between the study medication and the study end-points and where there is a relationship between serum vitamin D levels achieved and study end-points. The trial will also determine if oral vitamin D3 has an acceptable side effect and safety profile by monitoring any adverse events (including changes in blood calcium levels) over the 12 months.

The trial aims to enrol 240 participants with FDE from 21 Australian and New Zealand MS trial centres over the course of three years.

Inclusion criteria

For inclusion in this study, a participant must meet the following criteria:

• aged between 18 and 65 years old inclusive
• have a first isolated, well-defined, uni- or multi-focal first demyelinating event (FDE)
• be able to receive first dose of study drug within 120 days of FDE symptom onset
• have an MRI brain scan that is supportive of demyelinating disease (Paty A or Paty B criteria)
• an EDSS between 0 – 6.5 (inclusive)
• be able to give informed consent and sign the informed consent form
• be willing to avoid open-label vitamin D supplementation and external serum vitamin D testing for the duration of the study
• be willing to avoid use of sunbeds
• not have received any prior disease modifying treatment for MS other than glucocorticoids
• be willing to avoid treatment with beta-interferon, glatiramer acetate, natalizumab, mitoxantrone, or other chemotherapy agent specifically for demyelinating disease until recurrent disease activity occurs (ie the primary endpoint is met)
• be able and willing to comply with all study procedures including MRI scanning as per protocol
• be willing to use effective contraceptive methods for the duration of the study and at least 6 months following.

For full details of Exclusion criteria please visit the ANZCTR trial listing here.

Anticipated start date

May 2013

Recruitment contacts

Further details

Full details of the trial can be found on www.msra.org.au/prevanz
Or visit the ANZCTR trial listing here.

Region

NSW/SA/VIC/QLD/WA/TAS/NZ

Trial results

Expected in 2019

Last updated

March 2018