October 10, 2025
Welcome to the fourth issue of the MAGNITUDE-2 newsletter, containing important study-related information, updates, and data. Please file it in your Investigator Site File (ISF) binder because it is a study correspondence. If you have any comments or suggestions, contact your CRA, your Medpace regulatory submissions coordinator, or the study team.
There are currently 15 sites active in 7 countries.
• Countries with all sites activated: Argentina, Australia, Brazil, New Zealand, Singapore, and Taiwan.
• Countries with active and non-active sites: Thailand
• Countries pending site activation: Mexico
We are ahead of our enrollment projections. Thanks to all sites that have contributed to enrollment so far! Below you will see an overview of current enrollment status and recognition of our highest enrolling sites to date. Well done all, and we look forward to additional sites making their contributions!
Brazil 07606 / PSEG Centro de Pesquisa Clínica S.A. | Paulo Sgobbi de Souza / Leslye Donato Pinto: 5 Randomizations
Brazil 07601 / HCFMRP - Hospital das Clinicas da Faculdade de Medicina de Ribeirao Preto | Wilson Marques Jr. / Victoria Maria dos Santos: 5 Randomizations
Taiwan 15803 / National Taiwan University Hospital |Chi-Chao Chao / Chia-Hua Hsu 5 Randomizations
Brazil 07607 / Hospital das Clinicas da Universidade Estadual de Campinas (UNICAMP) | Marcondes Cavalcante França Jr. / Adriana Mendes Vinagre: 5 Randomizations
Taiwan 15804 / Chang Gung Medical Foundation - Linkou Branch Long-Sun Ro / Hui-Wen Hung 4Singapore 70203 / National University Hospital - National University Health System | Kay Wei Ping Ng / Amara Htet Myat Noe Oo: 4 Randomizations
Brazil 07609 / Instituto de Educação, Pesquisa e Gestão em Saúde | Marcia Waddington Cruz / Lorena Caroline Santos Silva: 4 Randomizations
Taiwan 15802 / Taipei Veterans General Hospital | Yi-Chung Lee / Sharon Chou: 3 Randomizations
Thailand 76401 / Siriraj Hospital | Pannathat Soontrapa / Thanyalak Ganjiang: 3 Randomizations
Argentina 03206 / Hospital Británico de Buenos Aires | Ricardo Reisin / Elizabeth Varela: 2 Randomizations
Argentina 03205 / Hospital El Cruce |Cristian Calandra / Cintia Amato 2 Randomizations
Singapore 70204 / Singapore General Hospital |Kaavya Narasimhalu / Erinn Jade Yi Choong: 1 Randomization
New Zealand 55401 / New Zealand Clinical Research |Ed Gane / Noosheen Ahdi: 0 Randomizations
Australia 03602 / Westmead Hospital | Matthew Silsby / Helen Knight: 0 Randomizations
Argentina 03203 / Hospital Italiano de Buenos Aires | Marcelo Rugiero / Mariela Lovelle: 0 Randomizations
Screened 69
In screening 7
Randomized 43
Screen failed 19
Please see below for a high-level summary of enrolled participants’ characteristics:
Median Age (range) 60 (22-76)
Gender Male: 31 Female:12
Mutation Type: A97S: 14 (32.5%) V30M late onset: 9 (21%) V30M early onset: 11 (25.5%) Others: 9 (21%)
• Record all relevant medical conditions and surgeries that occurred within the past 3 years from signing informed consent. If a condition is clinically relevant or related to the participant’s underlying disease but has a start date earlier than 3 years, please enter the condition.
• Provide as many details as possible, including body systems related to the medical history (i.e., Left, Right, or Bilateral) or specifics associated with the condition (i.e., Allergies—seasonal, animal dander, latex, etc.).
• Spell out the condition, i.e., do not use medical abbreviations.
• Report corresponding medical conditions (if known) for surgeries.
• For female participants: record history of menopause and/or sterilization procedures.
• For all participants: record entry of ATTR polyneuropathy (and ATTR cardiomyopathy, if applicable).
• Only conditions which started prior to signing of informed consent are to be reported as medical history.
If you have questions about entering medical history information, please refer to page 58 of the eCCGs (V3) or contact your CRA.
Medication Name
• Record one medication per record.
• For combination medications, do not split the entries. Record the brand name (if available).
• Record premedications, Vitamin A supplementation, and ATTR treatments on this eCRF.
Indication
• Provide the specific condition/diagnosis. Do not enter the type of drug.
• For medications taken as preventative, describe the condition being treated prophylactically.
• Medications to treat symptoms related to TTR (e.g., diarrhea, pain, tingling, etc.) should be listed as concomitant medication and not TTR-specific medication. Link them to the corresponding medical history.
Reason
• Select the applicable option from the drop-down.
• Record medications taken for multiple reasons as separate entries.
• For example, two entries are expected for ongoing medications taken as needed (PRN) for a medical history condition and for treatment of an AE.
• Only select “TTR Specific Medication” for medications approved for treatment of ATTR, including: acoramidis, diflunisal, doxycycline, inotersen, patisiran, tafamidis, tauroursodeoxycholic, vutrisiran, and vyndamax. Once selected, Reason for Starting will appear.
• Select Prophylaxis for medications administered in preparation for a procedure or exam that is not considered treatment for an AE or medical history condition (e.g., lidocaine for a biopsy, dilation medications for eye exams).
• Only select Premedication for medications required prior to study drug administration as described in the protocol.
• Corresponding Medical History (MH)
If “Reason” is Medical History, select the applicable condition. This field is a Dynamic Search List (see Section 3.9). The condition must be entered on the Medical History eCRF (with at least the condition and start date) prior to medication entry. If you have any questions about entering concomitant medications, please refer to page 66 of the eCCGs (V3) or contact your CRA.
Please reference the mNIS+7 upload instructions below and on page 77 of the eCCGs (V3).
1. Upload each assessment individually. Assessment materials should never be uploaded as one file. There should be an individual upload of the Clinical Neuropathy Assessment (CNA), Smart Somatotopic Quantitative Sensory Testing (QST), Postural Hypotension (PHT), and Nerve Conduction Studies (NCS) assessment at each mNIS+7 Uploads CRF. NCS wave forms should be included in the same PDF as the NCS worksheet.
2. Required file naming convention:
a. [Site-Subject]_[Visit]_[Date]_[Component]_Initial
b. 12345-0001_Screening-1_13Oct2024_CNA_Initial
3. Updated files/uploads: For each updated file upload, add “_Query Response and date (DDMMMYYYY)” to the end of the file naming convention described above. Always upload the full set of pages of the assessment that had a correction performed, even if the correction is only on one page. Any clarification should be written in the worksheet instead of answered directly in EDC as a response to a query.
a. [Site-Subject]_[Visit]_[Date]_[Component]_Query Response [Date]
b. 12345-0001_Screening-1_13Oct2024_CNA_Query Response 15Oct2024
If the EDC system is down, please reach out to your CRA so an alternative, compliant system can be used in the meantime.
Remember to enter data within 5 business days of the participant’s visit and resolve any queries within 5 business days of issuance. If you have any questions or concerns, please contact your CRA.
Please inform your CRA about the dates that mNIS+7 assessments were scheduled at your site as soon as possible. This will facilitate the review turnaround time and help avoid unnecessary window deviations
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