Nomlabofusp: Dosing Protocols
Dosing guidelines, reconstitution, and administration information
๐TL;DR
- โข1 dosing protocols documented
- โขReconstitution instructions included
- โขStorage: Store refrigerated at 2-8 degrees C. Protect from light. Do not freeze. As a recombinant protein, nomlabofusp requires cold chain storage to maintain stability and bioactivity.
Protocol Quick-Reference
Friedreich ataxia frataxin replacement
Dosing
Amount
25 mg daily (with 5 mg test dose on initial administration)
Frequency
Once daily
Duration
Ongoing
Step-wise Titration
Administration
Route
SCSchedule
Once daily
Timing
Initial dose under medical observation due to anaphylaxis risk
โ Rotate injection sites
Cycle
Duration
Ongoing
Repeatable
Yes
Preparation & Storage
Storage: Refrigerate at 2-8 degrees C. Protect from light. Do not freeze.
โ๏ธ Suggested Bloodwork (4 tests)
When:
Why:
When:
Why:
When:
Why:
When:
Why:
๐ก Key Considerations
- โInvestigational biologic - not yet approved by any regulatory authority
- โAnaphylaxis risk requires initial dosing under medical observation
- โDaily subcutaneous injection required for sustained frataxin restoration
- โTreatment interruption leads to decline in frataxin levels
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| Purpose | Dose | Frequency | Duration | Notes |
|---|---|---|---|---|
| Friedreich Ataxia (Frataxin Replacement) | 25 mg subcutaneously once daily. Initial administration uses a modified regimen with 5 mg test dose followed by 25 mg one hour later under observation. Doses of 25-100 mg have been studied. | Once daily subcutaneous injection | Ongoing (continuous treatment required for sustained frataxin levels) | Modified starting regimen implemented to mitigate anaphylaxis risk. Phase 2 studied 25 mg and 50 mg cohorts. Open-label extension uses 25 mg daily. Dose-dependent frataxin increases observed across 25-100 mg range. |
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๐Reconstitution Instructions
Nomlabofusp is a recombinant fusion protein supplied in a formulation for subcutaneous injection. Specific reconstitution details depend on the clinical trial formulation. As a protein therapeutic, proper cold chain handling is essential.
Recommended Injection Sites
- โAbdomen
- โThigh
๐งStorage Requirements
Store refrigerated at 2-8 degrees C. Protect from light. Do not freeze. As a recombinant protein, nomlabofusp requires cold chain storage to maintain stability and bioactivity.
Community Dosing Protocols
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Before You Begin
Review safety warnings and contraindications before starting any protocol.
Investigational Status#
Nomlabofusp (CTI-1601) is an investigational biologic that has not received regulatory approval. All dosing information is derived from clinical trial protocols and published study results. This information is provided for research reference only.
Clinical Trial Dosing#
Phase 1 Dose Exploration (SAD/MAD)#
Single Ascending Dose (SAD):
- Doses: 10 mg, 25 mg, 50 mg, and 100 mg subcutaneous
- Single injection per dose level
- Peak plasma concentrations within 15 minutes
Multiple Ascending Dose (MAD):
- Up to 100 mg daily for 13 days
- Subcutaneous injection
- Linear dose-proportional pharmacokinetics
- Carrier-equivalent frataxin levels achieved at 50-100 mg
Phase 2 Dose Exploration#
Two dose cohorts evaluated:
25 mg Cohort (n=13):
- 25 mg SC daily for 14 days, then every other day to day 28
- Median frataxin increase: 0.56 pg/mcg (buccal), 2.81 pg/mcg (skin)
50 mg Cohort:
- 50 mg SC daily for 14 days, then every other day to day 28
- Median frataxin increase: 0.72 pg/mcg (buccal), 5.57 pg/mcg (skin)
- 100% of patients achieved skin FXN > 33% of healthy volunteer average
Open-Label Extension (Current Protocol)#
The current dosing protocol includes a modified starting regimen:
Initial Dose (Under Medical Observation):
- Administer 5 mg SC test dose
- Observe for 1 hour for allergic reaction
- If no reaction, administer 25 mg SC
- Continue observation period
Subsequent Daily Dosing:
- 25 mg SC once daily
- Self-administered at home after initial observation period
- Rotate injection sites (abdomen, thigh)
Pharmacokinetic Profile#
| Parameter | Value |
|---|---|
| Peak plasma concentration (Tmax) | ~15 minutes post-SC injection |
| Dose proportionality | Linear across 10-100 mg range |
| Frataxin increase onset | Within days of daily dosing |
| Time to carrier-equivalent FXN | ~90 days at 25 mg daily |
| Full carrier equivalence | 6 months (100% of participants, n=10) |
Monitoring Requirements#
Efficacy Monitoring#
- Skin frataxin levels: Surrogate endpoint being used for accelerated approval; measured by punch biopsy
- Buccal cell frataxin: Less invasive biomarker; measured by cheek swab
- mFARS: Modified Friedreich Ataxia Rating Scale for clinical outcome assessment
- Functional tests: Nine-hole peg test (9-HPT), timed 25-foot walk
Safety Monitoring#
- Anaphylaxis observation: Initial doses must be administered under medical supervision
- Injection site monitoring: Assess for reactions (typically mild, resolving in hours)
- Anti-drug antibodies: Monitor for immunogenicity with long-term exposure
- Cardiac assessment: Echocardiography for cardiomyopathy monitoring (per FA standard of care)
Treatment Continuity#
Nomlabofusp is a protein replacement therapy that requires ongoing administration:
- Frataxin levels increase with daily dosing and plateau at carrier-equivalent levels
- Treatment interruption leads to decline in frataxin levels
- Sustained daily dosing is required for continued clinical benefit
- The open-label extension demonstrates maintained frataxin levels and clinical outcomes for over 1 year of continuous dosing
Storage and Handling#
As a recombinant fusion protein, nomlabofusp requires careful handling:
- Refrigerated storage: 2-8 degrees C
- Protection from light
- Do not freeze
- Cold chain must be maintained during shipping and storage
- Use within specified time after removal from refrigeration
Related Reading#
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Medical Disclaimer
This website is for educational and informational purposes only. The information provided is not intended to diagnose, treat, cure, or prevent any disease. Always consult with a qualified healthcare professional before using any peptide or supplement.