Muscular DystrophiesPhase 1NCT00494195
Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
A Phase 1 study testing rAAV1.tMCK.human-alpha-sarcoglycan- First cohort and Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort in people with Muscular Dystrophies, run by Nationwide Children's Hospital at 1 site in United States.
Trial summary
Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Muscular Dystrophies
- What is being tested?
- rAAV1.tMCK.human-alpha-sarcoglycan- First cohort and Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2008to Aug 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Six LGMD2D subjects ages 5 and older based on the clinical degree of involvement (impaired muscle function/weakness, sufficient muscle preservation)
- Preservation of EDB muscle or another muscle if judged more favorable because of adequate muscle mass for gene transfer
- Males and females of any ethnic group
- Established mutations of an -SG gene on both alleles
- Ability to cooperate for testing
- Sexually active patients must be willing to practice a reliable method of contraception during the study
Who cannot join
- Active viral infection (symptoms listed in section 9.0 of the protocol)
- LGMD2D subjects without weakness or functional loss
- Cardiomyopathy based on clinical exam and ECHO with ejection fraction less than 40%
- HIV infected
- Hepatitis A, B, or C infected
- Autoimmune diseases and immunosuppressive drugs (other than pulse methylprednisolone at time of gene transfer)
Show all 11Show fewer
- Persistent leucopenia or leucocytosis (WBC less than or equal to 3.5 K/cu mm or at least 20.0 K/ cu mm) or neutrophils less than 1.5 K/ cu mm
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the Principal Investigator creates unnecessary risks for gene transfer
- Pregnancy
- Abnormal laboratory values considered clinically significant
- Alcoholism (CAGE questionnaire), and laboratory tests such as GGT and MCV
Trial locations and contacts
1 site in 1 country.
United States1 site
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: Phase I Gene Transfer of rAAV1.tMCK.Human-alpha-sarcoglycan for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating children and adults with LGMD2D.
Read the detailed description
The primary objective of this study is the assessment of the safety of intramuscular administration to alpha-sarcoglycan deficient subjects of recombinant adeno-associated virus serotype 1 (rAAV1)-human alpha-sarcoglycan gene (hαSG) vector under control of a skeletal muscle creatine kinase promoter. The secondary objective is to determine the dose of rAAV1.tMCK.hαSG vector required to achieve a detectable level of alpha-sarcoglycan in muscle of subjects with this disorder.
A recombinant virus vector constructed from AAV1 has been altered to carry the human alpha-sarcoglycan gene expressed from a tMCK promoter. The construct has been shown to initiate the production of a functional alpha-sarcoglycan protein in laboratory animals. This construct can reverse the dystrophic phenotype in the alpha-sarcoglycan knock out mouse, a laboratory animal model for the clinical disorder. Intramuscular injection of rAAV1 restores muscle histology to normal and increases muscle strength to levels exceeding control knock out mice but not to the same degree as wild-type mice.
The proposed human clinical trial is a phase I, double-blind randomized protocol with injection of rAAV1.tMCK.hαSG gene vector into muscle. Two cohorts of subjects with LGMD2D(alpha-sarcoglycan deficiency), each with proven mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each cohort. The first cohort will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate considering the individual patient) with a dose of 3.25 X 10 to the 11 vg in 1.5 ml. The anatomical midline point of the muscle will be identified on the skin and 2 to 6 vector injections will be distributed in the direction of an X. The second cohort will receive the same dose delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. On the day of the vector infusion, 4 hours before gene transfer, patients will receive intravenous methylprednisolone 2.0 mg/kg (not to exceed 1 gm total), with repeat doses on two consecutive mornings. The methylprednisolone is specifically given to diminish the immediate inflammation from the needle injection, which is known to arouse an inflammatory reaction and could contribute to bringing antigen presenting cells to the site of vector delivery. We have previously demonstrated that this treatment enhances gene expression by at least 2-fold (Included as part of BB-IND-12936 for minidystrophin gene transfer).
Safety endpoints to be assessed include inflammatory reaction to the vector, as evaluated by muscle biopsy, and changes in hematology, serum chemistry, urinalysis, immunologic responses to rAAV1 and alpha-sarcoglycan, and reported history and observations of symptoms. The patient will have 10 to 12 follow-up visits for the next 2 years after the initial infusion.
- Conditions
- Muscular Dystrophies
- Being tested
- rAAV1.tMCK.human-alpha-sarcoglycan- First cohort, Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort
- Keywords
- limb girdle muscular dystrophy type 2D, alpha-sarcoglycanopathy, LGMD, LGMD2D, gene therapy, gene transfer, muscular dystrophy, SGCA, sarcoglycan, limb girdle, adeno-associated virus, AAV, AAV1
- Medical subject headings
- Muscular Dystrophies, Sarcoglycanopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rAAV1.tMCK.human-alpha-sarcoglycan- First cohortGenetic
Also called LGMD2D Gene therapy-First cohort
The first cohort of subjects with LGMD2D (alpha-sarcoglycan deficiency) and proven mutations will undergo gene transfer with a minimum of three subjects enrolled into this cohort and will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate upon considering the individual patient. The dose will be 3.25 X 10 to the 11 vg in 1.5 ml.
In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo.
Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohortGenetic
Also called LGMD2D Gene therapy- Second cohort
The second cohort will receive the same dosis of 3.25 X 10 to the 11 vg in 1.5 ml delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | The first cohort will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate upon considering the individual patient. The dose will be 3.25 X 10 to the 11 vg in 1.5 ml. The anatomical midline point of the muscle will be identified on the skin and two to six vector injections will be distributed in the direction of an X. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. Receives: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- First cohort |
| 2 | Experimental | The second cohort will receive the same dose of 3.25 X 10 to the 11 vg in 1.5 ml delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. Receives: Genetic: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of AAV1.tMCK.human-alpha-sarcoglycan gene transfer via intramuscular injection to the EDB muscle
Measured: Measured throughout the study
Secondary outcomes (2)
Human-alpha-sarcoglycan gene expression at the site of gene transfer via muscle biopsy
Measured: First cohort: Measured 45 days for two patients and at 90 days after gene transfer for one patient; Second cohort: Measured at 6 months after gene transfer for the three patients
Muscle strength of the gene transferred muscle via maximal volume isometric contraction testing (MVICT) if selected muscle is suitable for strenght testing
Measured: Measured 45 or 90 days after gene transfer in the first cohort, or 6 months post-gene transfer in second cohort depending on muscle biopsy date
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jun 2007 (estimated)
- Study startMar 2008
- Primary completionJun 2011
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Muscular Dystrophy Association
- Responsible party
- Principal investigator, Jerry R. Mendell, Nationwide Children's Hospital
- Investigators
- Jerry R. Mendell, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00494195
- Sponsor's protocol ID
- 5U54AR050733
- Other IDs
- U54AR050733, 5U54AR050733, IRB07-00329
- Status last verified
- Feb 2013
- First submitted
- 27 Jun 2007
- Last updated
- 5 Feb 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Allamand V, Donahue KM, Straub V, Davisson RL, Davidson BL, Campbell KP. Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. Gene Ther. 2000 Aug;7(16):1385-91. doi: 10.1038/sj.gt.3301247.
PubMed 10981665Buning H, Nicklin SA, Perabo L, Hallek M, Baker AH. AAV-based gene transfer. Curr Opin Mol Ther. 2003 Aug;5(4):367-75.
PubMed 14513679Bueno MR, Moreira ES, Vainzof M, Chamberlain J, Marie SK, Pereira L, Akiyama J, Roberds SL, Campbell KP, Zatz M. A common missense mutation in the adhalin gene in three unrelated Brazilian families with a relatively mild form of autosomal recessive limb-girdle muscular dystrophy. Hum Mol Genet. 1995 Jul;4(7):1163-7. doi: 10.1093/hmg/4.7.1163.
PubMed 8528203Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology. 2008 Jul 22;71(4):240-7. doi: 10.1212/01.wnl.0000306309.85301.e2. Epub 2008 Jun 4.
PubMed 18525034Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.
PubMed 21031578Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732.
PubMed 19798725
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I Gene Transfer of rAAV1.tMCK.Human-alpha-sarcoglycan for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D)
- ClinicalTrials.gov ID
- NCT00494195
- Acronym / sponsor ID
- 5U54AR050733
- Conditions
- Muscular Dystrophies
- Keywords
- limb girdle muscular dystrophy type 2D, alpha-sarcoglycanopathy, LGMD, LGMD2D, gene therapy, gene transfer, muscular dystrophy, SGCA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS), Muscular Dystrophy Association
- Study start
- Mar 2008
- Primary completion
- Jun 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 29 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Limb girdle muscular dystrophy type 2D (LGMD2D) is a genetic disease that affects skeletal muscle. Insufficient levels of the protein alpha-sarcoglycan result in muscle weakness that worsens over time. The purpose of this study is to evaluate the safety and effectiveness of gene therapy in treating children and adults with LGMD2D.
Detailed description
The primary objective of this study is the assessment of the safety of intramuscular administration to alpha-sarcoglycan deficient subjects of recombinant adeno-associated virus serotype 1 (rAAV1)-human alpha-sarcoglycan gene (hαSG) vector under control of a skeletal muscle creatine kinase promoter. The secondary objective is to determine the dose of rAAV1.tMCK.hαSG vector required to achieve a detectable level of alpha-sarcoglycan in muscle of subjects with this disorder.
A recombinant virus vector constructed from AAV1 has been altered to carry the human alpha-sarcoglycan gene expressed from a tMCK promoter. The construct has been shown to initiate the production of a functional alpha-sarcoglycan protein in laboratory animals. This construct can reverse the dystrophic phenotype in the alpha-sarcoglycan knock out mouse, a laboratory animal model for the clinical disorder. Intramuscular injection of rAAV1 restores muscle histology to normal and increases muscle strength to levels exceeding control knock out mice but not to the same degree as wild-type mice.
The proposed human clinical trial is a phase I, double-blind randomized protocol with injection of rAAV1.tMCK.hαSG gene vector into muscle. Two cohorts of subjects with LGMD2D(alpha-sarcoglycan deficiency), each with proven mutations will undergo gene transfer. A minimum of three subjects will be enrolled into each cohort. The first cohort will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate considering the individual patient) with a dose of 3.25 X 10 to the 11 vg in 1.5 ml. The anatomical midline point of the muscle will be identified on the skin and 2 to 6 vector injections will be distributed in the direction of an X. The second cohort will receive the same dose delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. On the day of the vector infusion, 4 hours before gene transfer, patients will receive intravenous methylprednisolone 2.0 mg/kg (not to exceed 1 gm total), with repeat doses on two consecutive mornings. The methylprednisolone is specifically given to diminish the immediate inflammation from the needle injection, which is known to arouse an inflammatory reaction and could contribute to bringing antigen presenting cells to the site of vector delivery. We have previously demonstrated that this treatment enhances gene expression by at least 2-fold (Included as part of BB-IND-12936 for minidystrophin gene transfer).
Safety endpoints to be assessed include inflammatory reaction to the vector, as evaluated by muscle biopsy, and changes in hematology, serum chemistry, urinalysis, immunologic responses to rAAV1 and alpha-sarcoglycan, and reported history and observations of symptoms. The patient will have 10 to 12 follow-up visits for the next 2 years after the initial infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- First cohortThe first cohort will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate upon considering the individual patient. The dose will be 3.25 X 10 to the 11 vg in 1.5 ml. The anatomical midline point of the muscle will be identified on the skin and two to six vector injections will be distributed in the direction of an X. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. |
| 2 | Experimental | Genetic: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohortThe second cohort will receive the same dose of 3.25 X 10 to the 11 vg in 1.5 ml delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Six LGMD2D subjects ages 5 and older based on the clinical degree of involvement (impaired muscle function/weakness, sufficient muscle preservation)
- Preservation of EDB muscle or another muscle if judged more favorable because of adequate muscle mass for gene transfer
- Males and females of any ethnic group
- Established mutations of an -SG gene on both alleles
- Ability to cooperate for testing
Key exclusion criteria (11 listed)
- Active viral infection (symptoms listed in section 9.0 of the protocol)
- LGMD2D subjects without weakness or functional loss
- Cardiomyopathy based on clinical exam and ECHO with ejection fraction less than 40%
- HIV infected
- Hepatitis A, B, or C infected
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rAAV1.tMCK.human-alpha-sarcoglycan- First cohortAlso: LGMD2D Gene therapy-First cohortThe first cohort of subjects with LGMD2D (alpha-sarcoglycan deficiency) and proven mutations will undergo gene transfer with a minimum of three subjects enrolled into this cohort and will receive a total of 1.5 ml volume of study agent in two to six separate injections into the selected muscle (extensor digitorum brevis) or other muscle if more appropriate upon considering the individual patient. The dose will be 3.25 X 10 to the 11 vg in 1.5 ml. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. | Genetic | 1 |
| Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohortAlso: LGMD2D Gene therapy- Second cohortThe second cohort will receive the same dosis of 3.25 X 10 to the 11 vg in 1.5 ml delivered to muscle according to the same paradigm. In each cohort, only one extremity will receive vector with transgene while the opposite extremity will be injected with placebo. | Genetic | 2 |
Primary outcome
Safety of AAV1.tMCK.human-alpha-sarcoglycan gene transfer via intramuscular injection to the EDB muscle
Time frame: Measured throughout the study
2 secondary outcomes
Human-alpha-sarcoglycan gene expression at the site of gene transfer via muscle biopsy
Time frame: First cohort: Measured 45 days for two patients and at 90 days after gene transfer for one patient; Second cohort: Measured at 6 months after gene transfer for the three patients
Muscle strength of the gene transferred muscle via maximal volume isometric contraction testing (MVICT) if selected muscle is suitable for strenght testing
Time frame: Measured 45 or 90 days after gene transfer in the first cohort, or 6 months post-gene transfer in second cohort depending on muscle biopsy date
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2013.
- Background: PubMed 10981665Allamand V, Donahue KM, Straub V, Davisson RL, Davidson BL, Campbell KP. Early adenovirus-mediated gene transfer effectively prevents muscular dystrophy in alpha-sarcoglycan-deficient mice. Gene Ther. 2000 Aug;7(16):1385-91. doi: 10.1038/sj.gt.3301247.
- Background: PubMed 14513679Buning H, Nicklin SA, Perabo L, Hallek M, Baker AH. AAV-based gene transfer. Curr Opin Mol Ther. 2003 Aug;5(4):367-75.
- Background: PubMed 8528203Bueno MR, Moreira ES, Vainzof M, Chamberlain J, Marie SK, Pereira L, Akiyama J, Roberds SL, Campbell KP, Zatz M. A common missense mutation in the adhalin gene in three unrelated Brazilian families with a relatively mild form of autosomal recessive limb-girdle muscular dystrophy. Hum Mol Genet. 1995 Jul;4(7):1163-7. doi: 10.1093/hmg/4.7.1163.
- Background: PubMed 18525034Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR. Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology. 2008 Jul 22;71(4):240-7. doi: 10.1212/01.wnl.0000306309.85301.e2. Epub 2008 Jun 4.
- Results publication: PubMed 21031578Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.
- Results publication: PubMed 19798725Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol. 2009 Sep;66(3):290-7. doi: 10.1002/ana.21732.
- Click here for the Nationwide Children's Hospital Web siteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jerry R. Mendell, MD principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing rAAV1.tMCK.human-alpha-sarcoglycan- First cohort and Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort in people with Muscular Dystrophies, run by Nationwide Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Feb 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of AAV1.tMCK.human-alpha-sarcoglycan gene transfer via intramuscular injection to the EDB muscle (time frame: Measured throughout the study). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00494195, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2013.