RTA 901 for Diabetic Neuropathy

(CYPRESS Trial)

Recruiting at 1 trial location

Overseen By Louise A. Taber MD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Reata, a wholly owned subsidiary of Biogen

Disqualifiers: Non-diabetic neuropathy, Fibromyalgia, Diabetic foot ulcer, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

384 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called RTA 901 for individuals with Diabetic Peripheral Neuropathic Pain (DPNP), a painful nerve damage caused by diabetes. The study aims to determine the safety and effectiveness of RTA 901 by comparing it to a placebo (a harmless pill with no active drug). Participants will be randomly assigned to one of three groups, each receiving a different dose of RTA 901 or the placebo. The trial seeks individuals who have had either type 1 or type 2 diabetes for at least a year and experience painful symptoms in their legs, such as burning, tingling, or electric shock-like pain. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that RTA 901 was tested for safety in earlier studies. In these studies, doses of 10 mg and 80 mg were safe and tolerable for participants. People who took these doses did not experience serious side effects, and the treatment was generally well-received. Although these studies did not demonstrate efficacy for the treatment's main purpose, the focus was on safety, and the results were positive in that regard. To date, no major safety concerns have been reported about RTA 901 in humans. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike most treatments for diabetic neuropathy, which typically focus on managing symptoms with pain relievers or anticonvulsants like pregabalin and duloxetine, RTA 901 works differently by targeting the underlying cellular stress response. This drug is a selective heat shock protein 90 (Hsp90) modulator, which helps protect nerve cells from damage and might slow the progression of neuropathy. Researchers are excited about RTA 901 because it offers a potential new mechanism to address nerve cell damage directly, rather than just alleviating pain symptoms. This could lead to more effective long-term management of diabetic neuropathy.

What evidence suggests that this trial's treatments could be effective for diabetic neuropathy?

Research suggests that RTA 901, a treatment under study in this trial, could help alleviate pain from diabetic nerve damage. This treatment protects nerve cells, potentially reducing pain. Earlier studies tested various doses of RTA 901 and found it safe and tolerable, though they did not clearly demonstrate pain reduction. While the safety results are promising, further research is needed to confirm its effectiveness in reducing pain for people with diabetic neuropathy. Participants in this trial will receive either RTA 901 or a placebo to further investigate its effectiveness. ^{1 2 3 4 5}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

Adults over 18 with type 1 or type 2 diabetes for at least a year and suffering from diabetic nerve pain in the lower extremities can join. They must have a certain level of pain despite taking one standard pain medication. Pregnant women, those not using birth control, people with recent severe liver issues, cancer history, frequent hypoglycemia needing medical help, or other major health problems are excluded.

Inclusion Criteria

Your pain score is 4 or higher on a scale of 0 to 10 at the screening.

I am on a stable dose of one standard pain medication for my nerve pain.

I have diabetes-related nerve pain in my legs or feet that feels burning or electric.

Exclusion Criteria

I have neuropathy not caused by type 1 or type 2 diabetes.

I have had a diabetic foot ulcer or infection in the last 90 days.

You needed help from a doctor for low blood sugar more than 3 times in the last 90 days.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2 weeks

Run-in

Participants receive study drug once daily to assess initial response and eligibility for randomization

2 weeks

Treatment

Participants receive randomized treatment with either RTA 901 or placebo once daily

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• RTA 901

Trial Overview The trial is testing RTA 901 against a placebo to see if it's safe and effective for diabetic nerve pain. Participants will be randomly assigned to receive either RTA 901 at two different doses or a placebo during the study which lasts about 20 weeks including screening, treatment period and follow-up.

How Is the Trial Designed?

Group I: Part 2 RTA 901 Dose 2 Experimental Treatment 1 Intervention

Participants will receive study drug, QD, during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901, QD, for a 12-week treatment duration.

Group II: Part 2 RTA 901 Dose 1 Experimental Treatment 1 Intervention

Participants will receive study drug, QD, during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901, QD, for a 12-week treatment duration.

Group III: Part 1 RTA 901 Dose 2 Experimental Treatment 1 Intervention

Participants will receive study drug, QD, during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901, QD, for a 12-week treatment duration.

Group IV: Part 1 RTA 901 Dose 1 Experimental Treatment 1 Intervention

Participants will receive study drug, once daily (QD), during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901, QD, for a 12-week treatment duration.

Group V: Part 1 RTA 901-Matching Placebo Placebo Group 1 Intervention

Participants will receive study drug, QD, during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901-matching placebo, QD, for a 12-week treatment duration.

Group VI: Part 2 RTA 901-Matching Placebo Placebo Group 1 Intervention

Participants will receive study drug, QD, during the 2-week Run-in Period. Following randomization, the participants will receive a dose of RTA 901-matching placebo, QD, for a 12-week treatment duration.

Who Is Running the Clinical Trial?

Reata, a wholly owned subsidiary of Biogen

Lead Sponsor

Trials

52

Recruited

7,400+

Biogen

Lead Sponsor

Trials

655

Recruited

468,000+

Daniel Quirk

Biogen

Chief Medical Officer

MD

Christopher A. Viehbacher

Biogen

Chief Executive Officer since 2022

Graduated from Queen's University, Kingston, Ontario, Canada

Reata Pharmaceuticals, Inc.

Lead Sponsor

Trials

52

Recruited

6,900+

Published Research Related to This Trial

In a 52-week open-label extension study involving 214 Asian patients with diabetic peripheral neuropathic pain (DPNP), mirogabalin demonstrated long-term safety and efficacy, with most patients experiencing a decrease in pain scores over time.

The most common side effects were mild to moderate, including nasopharyngitis and dizziness, with a treatment discontinuation rate of 13.1% due to adverse events, indicating that mirogabalin is generally well-tolerated in this patient population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term safety and efficacy of mirogabalin in Asian patients with diabetic peripheral neuropathic pain. Baba, M., Matsui, N., Kuroha, M., et al.[2022]

Around 50% of diabetic patients develop painful peripheral neuropathy within 25 years, primarily due to metabolic and vascular factors that damage nerves, making tight glycemic control essential for management.

While there is currently no drug to reverse diabetic neuropathy, treatments like tricyclic antidepressants, SNRIs, and certain anticonvulsants have shown efficacy in symptom relief, with ongoing research into newer therapies such as NMDA antagonists and aldose reductase inhibitors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Painful diabetic neuropathy: an update. Kaur, S., Pandhi, P., Dutta, P.[2022]

In a systematic review of 21 studies involving 2425 patients with painful diabetic neuropathy, the average placebo response showed a significant reduction in pain intensity, with a moderate effect size of 0.72, indicating that many patients experienced relief even without active treatment.

The study found that over half (53.3%) of patients in the placebo groups reported adverse events, with 5.1% dropping out due to these events, highlighting the importance of understanding both the benefits and potential risks associated with placebo treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Placebo and nocebo responses in painful diabetic neuropathy: systematic review and meta-analysis. Frisaldi, E., Vollert, J., Al Sultani, H., et al.[2023]

At least 25% of diabetic patients suffer from distal symmetric polyneuropathy, which can cause severe pain and significantly reduce quality of life, highlighting the need for effective treatment strategies.

Current treatment options include managing blood sugar levels, using available medications like alpha-lipoic acid and newer analgesics such as duloxetine and pregabalin, but challenges remain in effectively managing chronic pain associated with diabetic neuropathy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of diabetic neuropathy and neuropathic pain: how far have we come? Ziegler, D.[2022]

Strict blood glucose control is essential for managing diabetic neuropathy, but it can lead to complications like hypoglycemia; thus, a multifaceted approach to treatment is necessary.

Current evidence supports the use of tricyclic antidepressants and anticonvulsants for short-term pain relief, with combination therapies showing better efficacy than single treatments, alongside promising non-pharmacological options like electrical nerve stimulation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Painful diabetic neuropathy management. Khalil, H.[2013]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05895552?intr=cemdomespib&rank=1

A Phase 2 Study of RTA 901 (BIIB143) in Participants With ... This is a 2-part, randomized, placebo-controlled, double-blind, Phase 2 study to evaluate the safety, tolerability, efficacy, and pharmacokinetics (PK) of ...

2. biogentriallink.com biogentriallink.com/content/dam/global-development/general/biogen-trial-link/educational/en-us/pdf/csr/NCT05895552-Synopsis.pdf

A Phase 2 Study to Evaluate the Safety and Efficacy of RTA ... Overall, the 10 mg and 80 mg doses of RTA 901 tested in Part 1 were safe and tolerable, but the study did not meet its primary efficacy endpoint (treatment ...

3. bwh.trialstoday.org bwh.trialstoday.org/trial/NCT05895552

A Phase 2 Study of RTA 901 (BIIB143) in Participants With ... This is a 2-part, randomized, placebo-controlled, double-blind, Phase 2 study to evaluate the safety, tolerability, efficacy, and pharmacokinetics (PK) of RTA ...

4. delta.larvol.com delta.larvol.com/Products/?ProductId=a308b5e1-b8e1-4b50-ad08-35bb26deef36

cemdomespib (RTA 901) / Biogen A Phase 2 Study of RTA 901 (BIIB143) in Participants With Diabetic ... data • CNS Disorders • Diabetic Neuropathy • Neuralgia • Pain • Peripheral Neuropathic Pain.

5. centerwatch.com centerwatch.com/clinical-trials/listings/NCT05895552/a-phase-2-study-of-rta-901-biib143-in-participants-with-diabetic-peripheral-neuropathic-pain-cypress

A Phase 2 Study of RTA 901 (BIIB143) in Participants With ... This is a 2-part, randomized, placebo-controlled, double-blind, Phase 2 study to evaluate the safety, tolerability, efficacy, and pharmacokinetics (PK) of RTA ...