Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients with Myasthenia Gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

NMD Pharma A/S

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

19 Jul 2024 → ongoing

Decision date (initial)

2024-06-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others

To assess dose response of NMD670 on clinical efficacy as rated by clinicians

Conditions and MedDRA coding

Myasthenia Gravis

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 1. Participant must be a male or female being ≥18 years, at the time of signing the informed consent.
2. 2. Diagnosis of MG, MGFA class II, III or IV
3. 3. Documented positive AChR or MuSK antibody test.
4. 4. Participant must be able to swallow tablets
5. 5. Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg
6. 6. Female participants who are WOCBP and male participants with partners who are WOCBP must agree to use a highly effective contraception method during the study. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
7. 7. Participant is capable of and has given signed informed consent.

Exclusion criteria 8

1. 1. Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient’s full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study.
2. 2. Active or untreated thymoma, a history of thymic carcinoma or thymic malignancy (unless deemed cured by adequate treatment with no evidence of recurrence for 5 years or more before screening), a history of thymectomy in the 6 months before screening.
3. 3. Participant with a clinical diagnosis of gout, or with serum uric acid >upper limit of normal (ULN) at screening.
4. 4. Participant with any of the following: a. Abnormal liver function test defined as total bilirubin >1.5× ULN (except for participants with known Gilbert’s syndrome). b. Abnormal liver transaminase levels at baseline and confirmed current or chronic history of liver disease including (but is not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson’s disease, α 1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease considered clinically significant by the Investigator. c. Known hepatic or biliary abnormalities (with the exception of Gilbert’s syndrome or asymptomatic gallstones). d. Any renal impairment or kidney abnormalities considered clinically significant by the Investigator at screening.
5. 5. Participant with laboratory test abnormalities at screening considered clinically significant by the Investigator.
6. 6. Participants received treatment with IMP within 30 days (or 5 half-lives of the medication, whichever is longer) prior to day 1.
7. 7. Participants with history of poor compliance with relevant MG therapy.
8. 8. Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score for NMD670 vs placebo

Investigational products

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

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

NMD Pharma A/S

Sponsor organisation

NMD Pharma A/S

Address

Palle Juul-Jensens Boulevard 82

City

Aarhus N

Postcode

8200

Country

Denmark

Scientific contact point

Organisation

NMD Pharma A/S

Contact name

Vera Kiyasova

Public contact point

Organisation

NMD Pharma A/S

Contact name

Thomas Breuer

Third parties 6

Locations

7 EU/EEA countries · 27 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 246 files

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

Application history

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