Intermediate Expanded Access Protocol for ALS

• ClinicalTrials.gov Identifier: NCT04081714
• Expanded Access Status: Available
• First Posted: September 9, 2019
• Last Update Posted: April 3, 2023
• Sponsor: Clene Nanomedicine
• Collaborator: Massachusetts General Hospital
• Information provided by (Responsible Party): Clene Nanomedicine

Study Description

Brief Summary:

This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 40 participants diagnosed with ALS.

Condition or disease	Intervention/treatment
Amyotrophic Lateral Sclerosis	Drug: CNM-Au8

Detailed Description:

This is a single-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to forty (40) 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 three study periods:

1. A screening period over up to four (4) weeks (Screening Period);

2. Initial treatment period of twenty-four (24) weeks (Treatment Period 1);

   a. Additional consecutive optional follow-on treatment periods of twenty-four (24) weeks duration may be offered at the discretion of the Investigator and Sponsor (e.g., Treatment periods 2, 3, 4, 5, 6+)

3. 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, Treatment Period 5, Treatment Period 6 (n), Treatment Period n+1).

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 Design

• 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

Interventions

Intervention Details:

• Drug: CNM-Au8
  CNM-Au8 is an aqueous suspension of clean surfaced faceted nanocrystals consisting of gold atoms self-organized into crystals of various geometrical shapes (hexagonal bi-pyramid, pentagonal bipyramid, tetrahedron, decahedron, planar spheroids). Highly pure elemental Au nanocrystals are suspended in USP purified deionized water buffered with 0.546 mg/mL (6.5 mM) sodium bicarbonate (NaHCO3) nominally concentrated to 0.5 mg/L (500 ppm). CNM-Au8 will be administered orally in volumes of 60 mL from single-dose HDPE containers. Participants will orally receive 30 mg of CNM-Au8 once daily.
  Other Name: Nanocrystalline gold

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All

Criteria

Inclusion Criteria:

1. Able to understand and give written informed consent.
2. Male or female participants aged 18 years or greater (inclusive) at the time of ALS diagnosis.
3. Participants whose conditions are defined as "definite ALS" or "probable ALS" or "possible ALS" diagnostic criteria by the revised El Escorial Criteria as determined by a neurologist specializing in ALS (e.g., the Principal Investigator or subinvestigator at the site).
4. Participant is able to daily consume 60 mL of the investigational drug suspension without substantial dysphagia, OR can intake the drug through a gastrostomy tube.
5. In the judgement of the Investigator, the participant's expected survival is greater than six- months.
6. Participants who have established care with a neurologist at the specialized ALS center involved in the study and will maintain this clinical care throughout the duration of the EAP.

Exclusion Criteria:

1. Participant is eligible for a clinical study with CNM-Au8 for the treatment of ALS.
2. Participant with a history of any clinically significant or unstable medical condition based on the Investigator's judgment that may interfere with assessment of the study objectives.
3. Based on the investigator's judgment, participants who may have difficulty complying with the protocol and/or any study procedures.
4. Participant with clinically significant abnormalities in hematology, blood chemistry, ECG, or physical examination not resolved by the Baseline visit which according to Investigator can interfere with EAP participation1.
5. Participants with clinically significant hepatic or renal dysfunction or clinical laboratory findings that would limit the interpretability of change in liver or kidney function, or those with low platelet counts (< 150 x 109 per liter) or eosinophilia (absolute eosinophil count of ≥ 500 eosinophils per microliter) at Screening.
6. Participants with a prior history of, or positive serological assay for the presence of HIV infection, or laboratory evidence of active or chronic infection with hepatitis C (HCV) or hepatitis B (HBV). Note, participants who have been vaccinated for HBV and have detectable HB antibodies are not excluded unless positive for surface antigen (HBsAg).
7. Participant has participated in any other investigational drug trial (within 4-weeks prior to screening or at least five-half lives of the investigational product), unless waived at the discretion of the Sponsor (e.g., for other investigational drug trials with no known interaction with CNM-Au8).
8. Females who are pregnant or nursing or who plan to get pregnant during the course of this clinical trial or within 6 months of the end of this trial.
9. Females of child-bearing potential, or men, who are unwilling or unable to use accepted methods of birth control.
10. History of gold allergy.

Contacts and Locations

Contacts

Contact: Austin Rynders, BS; +1 (801) 676-9695; info@clene.com

Contact: Jeremy Evan, PA-C; +1 (801) 676-9695; info@clene.com

Locations

United States, Massachusetts

Massachusetts General Hospital; Available

Boston, Massachusetts, United States, 02114

Contact: Judith Carney, RN 617-724-8995 jcarey8@partners.org

Principal Investigator: Merit Cudkowicz, MD

Sponsors and Collaborators

Clene Nanomedicine

Massachusetts General Hospital

More Information

• Responsible Party: Clene Nanomedicine
• ClinicalTrials.gov Identifier: NCT04081714
• Other Study ID Numbers: CNMAu8.EAP01
• First Posted: September 9, 2019
• Last Update Posted: April 3, 2023
• Last Verified: March 2023

Keywords provided by Clene Nanomedicine:

ALS; Gold; Nanoparticle; Expanded access; Au8; CNMAu8

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis; Pathologic Processes; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases