A Multicenter, Double-Blind Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With ALS
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| ClinicalTrials.gov Identifier: NCT00561366 |
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Recruitment Status :
Withdrawn
(Not moving forward with program.)
First Posted : November 20, 2007
Last Update Posted : February 9, 2012
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis | Drug: Placebo Drug: Arimoclomol | Phase 2 |
This is a Phase 2b double-blind, randomized, placebo-controlled parallel-group study evaluating the safety and efficacy of arimoclomol (400 mg t.i.d.) compared to placebo. A safety lead-in phase will be employed to ensure the safety of all study volunteers.
Tier I (Safety Lead-in): During the enrollment period for the safety lead-in phase, 24 volunteers meeting inclusion/exclusion criteria will be randomized at 4 investigative sites. These volunteers will have weekly visits during the first 4 weeks after starting treatment. Pharmacokinetics (PK) will be performed at various timepoints throughout these 4 weeks. After the initial 4 weeks of treatment, visits will continue at 4-week intervals up to Week 36, subsequently visits will occur every 8 weeks up to Week 68. A final visit will occur at Week 72. There will be a 28-day post study medication Follow-Up Telephone Call to assess medical status and adverse events.
Tier II: After the Tier I volunteers finish 4 weeks of treatment, their data will be reviewed by the IDMC and, if no serious safety issues are identified, the recommendation will be made to start the second enrollment period (Tier II). During Tier II enrollment, volunteers recruited from approximately 30 to 40 centers in the US and Canada will be randomized. After screening and randomization, volunteers will be followed every 4 weeks for 9 months. Subsequently visits will occur every 8 weeks up to Week 68, with interim Follow-Up Telephone Calls at Weeks 16, 24, and 32 and a final visit at Week 72. A Week 76 Follow-Up Telephone Call to assess medical status and adverse events will occur at 28 days post last dose of study medication.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 0 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Multicenter, Double-Blind, Placebo-Controlled, Phase 2b Study to Investigate the Safety and Efficacy of Arimoclomol in Volunteers With Amyotrophic Lateral Sclerosis (ALS) |
| Arm | Intervention/treatment |
|---|---|
| Placebo Comparator: 1 |
Drug: Placebo
Placebo t.i.d. |
| Experimental: 2 |
Drug: Arimoclomol
capsule, 400 mg t.i.d. |
- ALSFRS-R [ Time Frame: 9 months ]
- ALSFRS-R [ Time Frame: 18 months ]
- Survival [ Time Frame: 18 months ]
- Muscle strength [ Time Frame: 9 and 18 months ]
- Pulmonary function [ Time Frame: 9 and 18 months ]
- MUNE [ Time Frame: 9 and 18 months ]
- Quality of Life [ Time Frame: 9 and 18 months ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Familial or sporadic ALS.
- Diagnosed with laboratory-supported probable, probable or definite ALS according to the World Federation of Neurology El Escorial criteria for less than or equal to 36 months' duration prior to the Screening Visit.
- Vital capacity (VC) equal to or greater than 70% predicted value for gender, height and age at the Screening Visit.
- Geographic accessibility to the study site.
- Ability to take oral medication at the Screening Visit, based on verbal report.
- Fluency in English, Spanish or Canadian French.
Exclusion Criteria:
- History of known sensitivity or intolerability to arimoclomol or to any other related compound.
- Prior exposure to arimoclomol through a clinical trial or physician-sponsored IND.
- Exposure to any investigational agent within 30 days of the Screening Visit.
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Presence of any of the following clinical conditions:
- Substance abuse within the past year
- Unstable cardiac, pulmonary, renal, hepatic, endocrine, hematologic, or active malignancy or infectious disease
- AIDS or AIDS-related complex
- Unstable psychiatric illness defined as psychosis (hallucinations or delusions), untreated major depression within 90 days of the Screening Visit.
- Laboratory values: Screening serum creatinine greater than or equal to 1.5 mg/dL, creatinine clearance less than 70 cc/min, alanine aminotransferase (ALT) greater than 3.0 times the upper limit of normal, total bilirubin greater than 1.5 times the upper limit of normal, white blood cell (WBC) count less than 3,500/mm3, platelet concentration of <100,000/ul, hematocrit level of less than 33 % for female or less than 35 % for male, or coagulation tests (PT, PTT) greater than or equal to 1.5 times upper limit of normal.
- Female volunteers who are breast-feeding.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00561366
Show 35 study locations
| Principal Investigator: | Merit Cudkowicz, MD, MSc | Massachusetts General Hospital | |
| Principal Investigator: | Jeremy Shefner, MD, PhD | State University of New York - Upstate Medical University |
| Responsible Party: | CytRx |
| ClinicalTrials.gov Identifier: | NCT00561366 |
| Other Study ID Numbers: |
AALS-003 |
| First Posted: | November 20, 2007 Key Record Dates |
| Last Update Posted: | February 9, 2012 |
| Last Verified: | February 2012 |
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ALS Lou Gehrig's disease |
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Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |