Research Faces of MLD

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Clinical Trials

Click here if you are looking for information on current MLD clinical trials.

The following is an outline of the general requirements and issues associated with the four phases of Clinical Trials necessary to move a therapy or drug from idea to approval. Each of these phases can take years to complete and might include an iteration back to an earlier step if new insights are found or if the results are not as expected.

Also note that it is quite common in rare diseases, such as MLD, for trial design and number of partcipants to vary sgnificantly from the guidelines below.

Goal: Establish safety and dosage limits in a small group of people (20-80)
Participants are often terminally ill individuals who have not responded to other treatment.

Is the treatment safe?

At which doses is the treatment safe?

What are the adverse side effects, if any?

How will you address these side effects?

Will the benefits of the treatment outweigh the risks?

Publish results in a peer-reviewed journal

Goal: Test the efficacy and safety in a larger group of people (100-300)

Can you deliver the gene effectively to target cells?

Do target cells express the delivered gene?

Do you observe health improvements in study participants? Short-term? Long-term?

Continue to measure side effects

Publish results in a peer-reviewed journal

Goal: Test the therapy in a large group of people (1,000-3,000)

Give treatment in a "double-blind" scenario. Neither the treating physicians nor the patient knows whether the treatment is authentic or a "placebo" (control). This ensures the validity of results.

Publish results in a peer-reviewed journal

Get FDA approval for general clinical use

Write proposals, fill out paperwork, and answer questions

Wait for approval

If approved, use your therapy to treat patients and proceed to Phase 4

Goal: Further test the efficacy and optimal use of the treatment in general use

Publish results in a peer-reviewed journal


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