Texas Trials, Clinical Research — it carries the weight of real work, doesn't it? For those of us living with rare autoimmune diseases, clinical research isn't abstract. It's the needles, the follow-up visits, the hope threaded through every protocol. Those of us who've carried the burden of experimental medicine know: every trial participant is someone's neighbor, someone's friend, someone betting on a cure.

Here's the truth: Texas is running hotter than ever in rare disease research. From CIDP and ALS to SMA, Angelman syndrome, and leukodystrophies, our state's medical institutions are advancing treatments that matter.

Read on. Get educated. And know that the work—your work, your participation, your courage—matters.

ADHERE Transition (Phase 4): evaluates switching stable CIDP patients from IVIg to efgartigimod PH20 SC without requiring clinical worsening—important for people who want to move from infusions to subcutaneous at-home dosing. (NCT06637072) (ClinicalTrials.gov)
Argenx’s AAN 2025 materials outline the design: start efgartigimod one week after the last IVIg, then follow safety and persistence over 12 weeks. (argenxmedical.com)

CAPTIVATE (DNTH103 / Phase 2–3): a classical complement C1s inhibitor designed to reduce demyelinating damage while sparing much of the immune system’s other functions. Ongoing development includes late-stage evaluation; recent updates indicate program progression beyond early trials. (GBS/CIDP Foundation International)

MIgGRATE (TAK-881 / Phase 3): higher-concentration subcutaneous IgG intended to reduce volume and time compared with existing options like HYQVIA®; PK comparability vs HYQVIA is a core study goal. (GBS/CIDP Foundation International)

NVG-2089 (Phase 2): an IV IgG-mimetic Fc-fusion approach now dosing CIDP participants; open-label study tracks safety and early efficacy. (NCT07027111) (Nuvig Therapeutics)

ARISE (Phase 3): evaluates an investigational therapy in adults with active CIDP; prescreen and contact options available via national study portals. (Autoimmune Association)

Angelman syndrome: Texas A&M announced human clinical testing of GTX-102 in 2025—an antisense therapy aimed at the genetic cause, not just symptoms. Texas-based preclinical leadership and recent updates keep the program on families’ radar. (Texas A&M Stories)

ALS: Houston Methodist’s neuromuscular program lists multiple ALS studies and coordinated clinical support; HEALEY ALS Platform Trial sites remain a key national access point patients can search for. (Houston Methodist)

SMA: CHRISTUS Children’s (San Antonio) runs an international SMA program providing advanced therapies and travel coordination—useful for families outside major metros. (CHRISTUS Health)

Leukodystrophies: The NIH-funded GLIA-CTN network (with Texas Children’s/Baylor investigators) standardizes real-world natural-history methods that directly inform trial design and endpoints. (PubMed)

“Can I switch from IVIg to a self-administered option?”
Yes—two active approaches are being studied: FcRn blockade (efgartigimod PH20 SC) and higher-concentration SC IgG (TAK-881). Talk to your neuro team about ADHERE (transition) and MIgGRATE (SC IgG). (ClinicalTrials.gov)

“Are there Texas trials outside CIDP?”
Yes—ALS (Methodist), SMA (CHRISTUS Children’s), Angelman (Texas A&M collaboration), and leukodystrophy natural-history work (GLIA-CTN) are active touchpoints. (Houston Methodist)

“How do I vet a study quickly?”
Grab the NCT number, verify on ClinicalTrials.gov, and ask about costs, travel, and who to call for prescreening. (ClinicalTrials.gov)

FcRn Inhibitor – A medicine that helps lower harmful antibodies in the bloodstream, potentially reducing nerve inflammation.

Complement Inhibitor – A drug that blocks part of the immune system (“complement pathway”) that contributes to nerve damage.

Antisense Therapy – A genetic approach that targets the root cause of diseases by blocking faulty gene instructions.

Phase 1–4 Trials – Clinical trial stages:

Open-Label vs. Randomized –

NCT Number – The unique ID used to find a specific study on ClinicalTrials.gov.

Natural-History Study – Research that tracks how a disease progresses over time without changing treatment—helps design better future trials.

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MEDICAL DISCLAIMER: This newsletter provides educational information reviewed by our medical advisory board. Content is not intended as medical advice. Always consult your healthcare provider before making treatment decisions. Clinical trial information is provided for educational purposes - eligibility and enrollment should be discussed with qualified medical professionals.

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