A Phase 1b/2 Study to Evaluate NB-4746 in Participants with Amyotrophic Lateral Sclerosis.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Nura Bio Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-06-03

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Nura Bio

External identifiers

EU CT number

2025-523939-18-00

ClinicalTrials.gov

NCT07369076

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Safety, Pharmacokinetic

For Phase 1b: to assess the safety profile of NB-4746 out of 28 days in participants with Amyotrophic Lateral Sclerosis (ALS)
For Phase 2 and Open label Extension (OLE): to assess the safety profile of NB-4746 in participants with (ALS)

Secondary objectives 2

1. Phase II: To demonstrate the effect of NB-4746 on NfL concentration.
2. Open Label Extention: To evaluate the effect of NB-4746 on NfL concentrations.

Conditions and MedDRA coding

Amyotrofic Lateral Sclerosis (ALS)

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

IPD plan description

na

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. ALS Criteria for Phase 1b: 1-Participants in Phase 1b must have: a. Diagnosis of ALS per Gold Coast Criteria and b. Symptom onset ≤48 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.
2. Capable of giving signed informed consent, as described in the protocol, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.
3. ALS Criteria for Phase 2: Participants in Phase 2 must have: a. Diagnosis of ALS per Gold Coast Criteria and b. Symptom onset ≤24 months prior to randomization on Day 1. Date of first ALS symptom (any ALS symptom) onset is defined as the onset of weakness in the limbs, bulbar region, or trunk. Weakness in the bulbar region includes dysarthria and dysphagia.
4. Male or female participants aged ≥18 years and ≤80 years at the time of signing informed consent.
5. Score of at least 2 on the swallowing function of the ALSFRS-R.
6. Slow vital capacity (SVC) ≥60% of predicted at Screening.
7. If taking riluzole, participant must be on a stable dose for ≥60 days prior to the screening lab blood draw
8. Screening laboratory test values within normal ranges. If taking riluzole, participant must be on a stable dose for ≥60 days prior to Screening lab blood draw. If taking edaravone, participant must have completed at least 1 cycle of edaravone prior to the Screening lab sample.
9. Willing to adhere to contraceptive requirements during the study period as described in the protocol

Exclusion criteria 9

1. Presence of tracheostomy or permanent assisted ventilation; defined as > 22 hours daily of mechanical ventilation for more than 1 week (7 days).
2. Inability to swallow or tolerate oral medications at Screening.
3. Current participation in any other investigational drug study or receipt of an investigational drug within 30 days or 5 half-lives (whichever is longer) before Screening.
4. Known sensitivity to the NB-4746 study drug or any of the formulation components.
5. History or presence of clinically significant uncontrolled medical conditions (other than ALS) that include cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study drugs; or interfering with the interpretation of data as determined by the Investigator.
6. Any other reason that, in the opinion of the Investigator, would confound the conduct of this study or the interpretation of the results or that could compromise the participant’s safety.
7. Female participants who have a positive serum pregnancy test at Screening, positive urine pregnancy test on Day 1, or who are breastfeeding on Day 1
8. Currently taking or planning to receive tofersen for the treatment of ALS.
9. International Normalized Ratio (INR) >1.4, platelet count <50,000/µL, or use of warfarin, heparin, or direct oral anticoagulants.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. Phase Ib: Number of treatment emergent adverse events (TEAEs) and number of serious adverse events (SAEs).
2. Phase II: TEAEs, and changes in vital signs, clinical laboratory tests, and 12-lead ECGs.
3. Open label Extention: TEAEs, and changes in vital signs, clinical laboratory tests, and 12-lead ECGs.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Nura Bio Inc.

Sponsor organisation

Nura Bio Inc.

Address

171 Oyster Point Boulevard Suite 401

City

South San Francisco

Postcode

94080-1936

Country

United States

Scientific contact point

Organisation

Nura Bio Inc.

Contact name

Lahar Mehta

Public contact point

Organisation

Nura Bio Inc.

Contact name

Lahar Mehta

Third parties 8

Locations

2 EU/EEA countries · 4 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 27 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications