Ambroxol update

[CuriousMe12]

I just received this from PD front line:

Dear Mr..In January of this year, Cure Parkinson’s and partners announced funding for the starting up of a Phase 3 clinical trial to evaluate the disease modifying potential of a cough medicine called ambroxol in people with Parkinson’s. Preclinical data in models of Parkinson’s indicated that ambroxol has neuroprotective properties, and a 6-month Phase 2 clinical trial in people with Parkinson’s provided encouraging results that justified further investigation of this agent.

The new Phase 3 study is called “ASPro-PD” and it represents an exciting, but challenging new endeavour for our charity as it is our largest clinical trial to date (involving over 330 people with Parkinson’s), and half of the participants need to be carriers of a specific genetic risk factor. In preparation for this, Cure Parkinson’s took the step of funding the “PD-Frontline” study to help identify individuals who are suitable to take part in the study.

In addition to finding the right participants for the ASPro-PD study, there is also a need to reformulate the drug being tested. In the Phase 2 study, the participants were required to take 21 pills of ambroxol per day (in addition to their PD medication) to achieve the required dose. This was a heroic effort by the participants in the 6-month study, but we knew that there would be a need to reformulate the drug for this larger and longer study to prevent people from pulling out of the study due to medication burden.

These efforts to reformulate ambroxol have resulted in a delay to the start of the ASPro-PD study. We know that this will be frustrating and disappointing for those of you who have put yourself forward to participate in this ground-breaking trial. Sadly such delays are common for clinical trials of such size and complexity. Rest assured that we are doing everything we can to help expedite the process and we hope to have further and better news for you soon. We remain committed to seeing ambroxol tested further in Parkinson’s. If you have any questions, please contact one of the following people:

[park_bear]

This is a UK only trial. Other than that eligibility requirements seem relatively broad:

clinicaltrials.gov/study/NC...

Express interest here:

cureparkinsons.org.uk/resea...

Description here:

cureparkinsons.org.uk/2023/...

[Zella23]

My husband and I were both participants in the PD frontline investigation. They told us they were looking for genetic markers for PD in our DNA. I don’t have PD but my husband has so I was used as a control. We started it at least two years ago. At the time we weren’t told it was a forerunner for the Ambroxol trial. They contacted us quite frequently and sent us a few online questionnaires. He was more interested to see if his PD genetic.

Recently my husband was told he didn’t fit the criteria for the trial as his PD isn’t genetic and his age is also a barrier.

[CuriousMe12 in reply to Zella23]

That's interesting Zelĺa. I guess it's the one time you might want a mutation. I never finalised my original application and i think they only included me in communication as i asked, a while back, why they weren't including general parkinsons people.

[SilentEchoes in reply to Zella23]

Do you know what genetic marker they're looking for?

[Zella23]

Also the amount of pills needed daily would have put him off. Although I think they are trying to address that issue. He’s not on many PD meds so the thought of taking that many would have made him think twice. Our children were pleased though about it not being genetic.

[CuriousMe12 in reply to Zella23]

Yes pros and cons to having the gene mutation .I'd have thought they'd have anticipated and addressed the pill volume at the start of the study.

[amykp]

it’s a GBA mutation.

[MarionP]

Too bad the drug industry won't let the FDA allow it in the U.S., perhaps that is a measure of how valuable it might actually be.

[ShakesMcgee in reply to MarionP]

You can buy it and ship to usa. I have 6 boxes I'm about to try.

[MarionP in reply to ShakesMcgee]

You can purchase it without a prescription or being a prescriber?

[CuriousMe12 in reply to MarionP]

Looks like mucosolvan is a brand with Ambroxol as the active ingredient.

carefromnature.co.uk/mucoso...

This study

ncbi.nlm.nih.gov/pmc/articl... Ambroxol (2-amino-3,5-dibromo-N-[trans-4-hydroxycyclohexyl]benzylamine), an over-the-counter product, is a mucoactive agent and has been used widely to treat both acute and chronic respiratory diseases since 1978. This review aims to provide an overview of the clinical evidence available on the use of ambroxol in children with acute and chronic respiratory diseases.

[MarionP in reply to CuriousMe12]

Okie dokie, very good, thanks!