Intermediate Expanded Access Protocol for ALS
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT04081714|
Expanded Access Status : Available
First Posted : September 9, 2019
Last Update Posted : November 3, 2020
|Condition or disease||Intervention/treatment|
|Amyotrophic Lateral Sclerosis||Drug: CNM-Au8|
This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to thirty (30) participants diagnosed with ALS.
Safety, pharmacokinetics, and pharmacodynamics of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at MGH, remotely over telephone, or via tele-visit. Collection of samples for pharmacokinetic and pharmacodynamic analysis may not be feasible for visits completed remotely. Participants will be screened over up to a 4-week period prior to treatment initiation. Participants who meet the inclusion criteria and none of the exclusionary criteria may be enrolled into the EAP. Participants may initiate treatment on the same day as the Screening visit, provided all inclusion and exclusion criteria have been fulfilled prior to treatment initiation.
There will be four study periods:
- A screening period over up to four (4) weeks (Screening Period);
- Initial treatment period of twenty-four (24) weeks (Treatment Period 1);
Up to three consecutive optional follow-on treatment periods of twenty-four (24) week duration:
- Treatment Period 2,
- Treatment Period 3,
- Treatment Period 4;
- A four (4) week safety follow-up period (End-of-Study [EOS] Assessment).
Per protocol all participants will receive open-label oral treatment daily up to 24-weeks during Treatment Period 1. Participants may optionally continue on open-label therapy for up to three additional consecutive 24-week treatment periods (e.g., Treatment Period 2, Treatment Period 3, Treatment Period 4) up to a maximum duration of ninety-six (96) weeks.
At treatment discontinuation or following the end of the participant's final Treatment Period, participants will complete an end of study (EOS) assessment 4-weeks following discontinuation of the investigational drug product. Select visit assessments may be collected remotely, via tele-visit with site staff.
|Study Type :||Expanded Access|
|Expanded Access Type :||Intermediate-size Population|
|See clinical trials of the intervention/treatment in this expanded access record.|
|Official Title:||An Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8|
- Drug: CNM-Au8
CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered USP water with a concentration of up to 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose HDPE containers.Other Name: Nanocrystalline gold
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04081714
|Contact: Robert Glanzman, MD||+1 (801) firstname.lastname@example.org|
|Contact: Austin Rynders, BS||+1 (801) email@example.com|
|United States, Massachusetts|
|Massachusetts General Hospital||Available|
|Boston, Massachusetts, United States, 02114|
|Contact: Judith Carney, RN 617-724-8995 firstname.lastname@example.org|
|Principal Investigator: Merit Cudkowicz, MD|